Final Program - American Society of Gene & Cell Therapy
Final Program - American Society of Gene & Cell Therapy
Final Program - American Society of Gene & Cell Therapy
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ASGCT Officers and Board <strong>of</strong> Directors<br />
President<br />
Xandra O. Breakeield, PhD<br />
President-Elect<br />
David W. Russell, MD, PhD<br />
Vice President<br />
Harry L. Malech, MD<br />
Secretary<br />
John J. Rossi, PhD<br />
Treasurer<br />
David V. Schaffer, PhD<br />
Board Members<br />
Chiara Bonini, MD<br />
Barry J. Byrne, MD, PhD<br />
Fabio Candotti, MD<br />
Roberto Cattaneo, PhD<br />
Marina Cavazzana-Calvo, MD, PhD<br />
Jeffrey Chamberlain, PhD<br />
Brendan Lee, MD, PhD<br />
John J. Nemunaitis, MD<br />
Xiao Xiao, PhD<br />
Ex-Oficio Board Members<br />
Malcolm K. Brenner, MD, PhD<br />
Katherine P. Ponder, MD<br />
Table <strong>of</strong> Contents<br />
A Welcome from Xandra O. Breakeield, PhD ................2<br />
Annual Meeting Exhibitors ....................................................3<br />
Annual Meeting Supporters ..................................................4<br />
Committee Meeting Schedule and Rosters ....................6<br />
Abstract Planning Committee & Reviewers ................ 12<br />
Excellence in Research Awards<br />
& Travel Grant Recipients .................................................. 14<br />
Award Recipients .................................................................... 15<br />
Faculty List ................................................................................ 16<br />
<strong>Gene</strong>ral Meeting Information ............................................ 22<br />
Hotel Information and Area Map ..................................... 26<br />
Convention Center Plans...................................................... 27<br />
Schedule-at-a-Glance ............................................................. 30<br />
<strong>Program</strong> Schedule ................................................................... 31<br />
Exhibit Hall Floor Plan .......................................................... 78<br />
Exhibitor Descriptions .......................................................... 79<br />
Exhibitor Information ........................................................... 86<br />
ASGCT Executive Office<br />
555 East Wells Street, Suite 1100<br />
Milwaukee, Wisconsin 53202<br />
Phone: 414.278.1341<br />
Fax: 414.276.3349<br />
Email: info@asgct.org<br />
Website: www.asgct.org<br />
Abstract Directory .................................................................. 87<br />
Abstract Author Index ........................................................ 139<br />
Mary Dean, JD, CAE<br />
Executive Director<br />
Ken Janowski<br />
Education & Meetings Manager<br />
Shaun Kramer<br />
Marketing & Outreach Manager<br />
Tara Withington, CAE<br />
Consulting Partner<br />
David Wood<br />
Senior <strong>Program</strong> Manager<br />
Rebecca Zimmer<br />
Membership & Administrative Coordinator<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013<br />
1
A Welcome from Xandra O. Breakefield, PhD<br />
Dear Colleagues,<br />
Welcome to the <strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> (ASGCT) 16th Annual<br />
Meeting! Salt Lake City’s rich history in the study <strong>of</strong> genetics and genealogy<br />
makes it a perfect city to feature this year’s latest advancements in our vibrant<br />
ield. The environs are also rich in majestic state parks and I hope you will<br />
take advantage <strong>of</strong> the chance to visit them, including Antelope Island State<br />
Park, as well as a guided tour <strong>of</strong> Temple Square on May 14 as part <strong>of</strong> an ASGCT<br />
networking adventure series.<br />
The program this year relects the increasing focus on reaching the<br />
commercialization phase <strong>of</strong> gene and cell therapeutics. On Wednesday evening,<br />
the Outreach Committee and BioIndustry Liaison Committee developed a two<br />
hour workshop on commercialization to discuss the low <strong>of</strong> funding in pipeline<br />
development along with some <strong>of</strong> the key conditions that must be in place in<br />
order to insure appropriate and eficient utilization <strong>of</strong> resources. On Saturday,<br />
the Translational Science & Product Development Committee will explore the<br />
unique issues we face in manufacturing biologics. Lastly, the BioIndustry Liaison<br />
Committee has organized a symposium on Friday comprising a series <strong>of</strong> talks<br />
and panel discussion aimed towards the latter steps <strong>of</strong> commercialization.<br />
In addition, we will continue to focus on our historic strengths in novel<br />
technologies in delivery platforms for oligonucleotide, gene and cell<br />
therapies, as well as advances in preclinical and clinical trials, with important<br />
breakthroughs on many fronts. I hope you will be able to stay for the full four<br />
days <strong>of</strong> programming, including the series <strong>of</strong> evening symposia sponsored by<br />
our foundation partners – the Foundation Fighting Blindness, the Muscular<br />
Dystrophy Association, the French Muscular Dystrophy Association, and the<br />
Alliance for Cancer <strong>Gene</strong> <strong>Therapy</strong>.<br />
My special thanks to all the ASGCT members, exhibitors, sponsors and attendees<br />
who are making this such an exciting meeting!<br />
Sincerely,<br />
Xandra O. Breakeield, PhD<br />
President, ASGCT<br />
2<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013
Annual Meeting Exhibitors – as <strong>of</strong> April 2013<br />
The <strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> would like to acknowledge and thank the following exhibitors for their<br />
participation at the ASGCT 16 th Annual Meeting. ASGCT greatly appreciates their support and encourages all attendees to<br />
make time each day to visit each <strong>of</strong> these exhibitors in Hall C/D.<br />
Aldevron<br />
AltheaDx<br />
<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong><br />
Biomiga, Inc.<br />
BTX/Harvard Apparatus<br />
<strong>Cell</strong>Genix GmbH<br />
Charles River<br />
Children’s Hospital <strong>of</strong> Philadelphia<br />
Cincinnati Children’s Hospital Medical Center<br />
First Biotech Inc.<br />
Florida Biologix<br />
GE Healthcare<br />
GenScript USA Inc<br />
HypOxygen<br />
Lovelace Respiratory Research Institute<br />
Mary Ann Liebert, Inc.<br />
Miltenyi Biotec<br />
MPI Research<br />
NanoSight<br />
National <strong>Gene</strong> Vector Biorepository (NGVB)<br />
Nature Publishing Group<br />
Nature Technology Corporation<br />
Nexcelom Bioscience<br />
NHLBI <strong>Gene</strong> <strong>Therapy</strong> Resource <strong>Program</strong><br />
NIH Ofice <strong>of</strong> Biotechnology Activities<br />
Panasonic Healthcare Company <strong>of</strong> North America<br />
Penn Vector Core<br />
PerkinElmer<br />
PolyPlus-Transfection<br />
REGENX Biosciences, LLC<br />
SAFC<br />
St. Jude Children’s Research Hospital<br />
The Baker Company<br />
TriLink BioTechnologies, Inc.<br />
UAB Vector Production Facility<br />
UNC <strong>Gene</strong> <strong>Therapy</strong> Vector Core<br />
University <strong>of</strong> Iowa/<strong>Gene</strong> Transfer Vector Core<br />
University <strong>of</strong> Utah Blood & Marrow Transplant <strong>Program</strong> –<br />
<strong>Cell</strong> <strong>Therapy</strong> & Regenerative Medicine Facility<br />
VGXI, Inc.<br />
Waisman Biomanufacturing<br />
Wilson Wolf Corporation<br />
Thank you 16 th Annual Meeting Exhibitors!<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013<br />
3
Annual Meeting Supporters – as <strong>of</strong> April 2013<br />
The <strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> is honored to acknowledge the following organizations for their support <strong>of</strong> the<br />
16th Annual Meeting:<br />
Partner Level Support<br />
Printing <strong>of</strong> the Abstract Supplement<br />
Registration Bags<br />
Contributor Level Support<br />
Foundation Symposium<br />
Foundation Symposium<br />
Patron Level Support<br />
Conference Grant Support<br />
Foundation Symposium<br />
Excellence in Research Award (1)<br />
Conference Grant Support<br />
Outstanding New Investigator Award (1)<br />
Travel Grant (5)<br />
Excellence in Research Awards (2)<br />
Travel Grant (1)<br />
Travel Grant (4)<br />
Travel Grant (1)<br />
Travel Grant (2)<br />
Excellence in Research Award (1)<br />
Excellence in Research Award (1)<br />
Travel Grant (1)<br />
4<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013
Annual Meeting Supporters<br />
Patron Level Support, continued<br />
Excellence in Research Award (1)<br />
Outstanding New Investigator Award (1)<br />
Conference Grant Support<br />
Supported in part by March <strong>of</strong> Dimes<br />
Travel C<strong>of</strong>fee Mugs Travel Grant (1)<br />
Foundation Grant No. 4-FY13-234<br />
Foundation Symposium<br />
Travel Grant (2)<br />
Outstanding New Investigator Award (1)<br />
<strong>Gene</strong>ral Meeting Support<br />
Travel Grant (3)<br />
Outstanding New Investigator Award (1)<br />
Travel Grants (10)<br />
Travel Grants (4)<br />
Travel Grants (5)<br />
Shenzhen SiBono <strong>Gene</strong> Tech<br />
Conference Grant Support<br />
Travel Grant (1) Travel Grants (5)<br />
National Institutes <strong>of</strong> Health Grant Support<br />
This conference was supported by Grant Number 1R13NS083180-01<br />
from the National Institute <strong>of</strong> Neurological Disorders and Stroke.<br />
The content is solely the responsibility <strong>of</strong> the authors and does not<br />
necessarily represent the <strong>of</strong>icial views <strong>of</strong> the National Institute <strong>of</strong><br />
Neurological Disorders and Stroke or the National Institutes <strong>of</strong> Health.<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013<br />
5
Committee Meeting Schedule – as <strong>of</strong> April 2013<br />
Wednesday – May 15<br />
12:00 pm – 1:15 pm Advisory Council 250D<br />
12:00 pm – 1:15 pm Translational Science & Product Development Committee 250E<br />
12:00 pm – 1:15 pm Host Committee 251A<br />
12:00 pm – 1:15 pm Membership Committee 251B<br />
7:30 pm – 9:00 pm Molecular <strong>Therapy</strong> Editorial Board Meeting Room: Canyon BC (Hilton<br />
Salt Lake City Center)<br />
Thursday – May 16<br />
12:30 pm – 2:00 pm Publications Committee 250D<br />
12:30 pm – 2:00 pm Education Committee 250E<br />
12:30 pm – 2:00 pm Exhibitor Relations Committee 251A<br />
12:30 pm – 2:00 pm Clinical Trials & Regulatory Affairs Committee 251E<br />
8:00 pm – 9:30 pm Molecular <strong>Therapy</strong>: Nucleic Acids Editorial Board Meeting Room: Salon 3 (Hilton<br />
Salt Lake City Center)<br />
Friday – May 17<br />
11:45 am – 1:00 pm Cancer <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> Committee 250D<br />
11:45 am – 1:00 pm Development Committee 250E<br />
11:45 am – 1:00 pm Bio-Industry Liaison Committee 251A<br />
11:45 am – 1:00 pm New Investigator Committee 251B<br />
11:45 am – 1:00 pm Neurologic & Ophthalmic <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> Committee 251D<br />
11:45 am – 1:00 pm Outreach Committee 251E<br />
Saturday – May 18<br />
7:30 am – 8:00 am ASGCT Annual Business Meeting 150ABC<br />
12:15 pm – 1:45 pm<br />
Scientiic Committee Meetings:<br />
Cardiovascular <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>;<br />
Chemical <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>;<br />
<strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> <strong>of</strong> <strong>Gene</strong>tic and Metabolic Diseases;<br />
Hematologic & Immunologic <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>;<br />
Immune Responses to <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>;<br />
Infectious Diseases & Vaccines;<br />
Musculo-Skeletal <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>;<br />
Oligonucleotide and RNAi Therapeutics;<br />
Physical <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> & Vectorology;<br />
Respiratory Tract <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>;<br />
Stem <strong>Cell</strong>;<br />
Viral <strong>Gene</strong> Transfer Vectors Committee<br />
255EF<br />
12:15 pm – 1:45 pm Board <strong>of</strong> Directors Meeting 250D<br />
<strong>Society</strong> Annual Business Meeting<br />
The ASGCT Annual Business Meeting will be conducted on Saturday, May 18, 7:30 am – 8:00 am. A continental breakfast will<br />
be served at 7:15 am in room 150ABC <strong>of</strong> the Convention Center.<br />
All ASGCT Members are welcome to attend. Election results will be announced, new <strong>of</strong>icers will be installed, and the<br />
<strong>Society</strong>’s inances and membership statistics will be reviewed. We welcome your input and participation!<br />
6<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013
Committee Rosters<br />
Advisory Council<br />
Chair<br />
Terence R. Flotte, MD (2013 - 2014)<br />
Katherine P. Ponder, MD (2011 - 2013)<br />
Members<br />
David M. Bodine, PhD (2009 - 2014)<br />
Xandra O. Breakeield, PhD (2013 - 2018)<br />
Barrie J. Carter, PhD (2011 - 2016)<br />
Kenneth Cornetta, MD (2010 - 2015)<br />
Beverly L. Davidson, PhD (2012 - 2017)<br />
Terence R. Flotte, MD (2011 - 2016)<br />
Carl H. June, MD (2010 - 2015)<br />
Luigi M. Naldini, MD, PhD (2008 - 2013)<br />
Arthur W. Nienhuis, MD (2008 - 2013)<br />
Katherine P. Ponder, MD (2009 - 2014)<br />
R. Jude Samulski, PhD (2012 - 2017)<br />
Bio Industry Liaison Committee<br />
Chair<br />
Estuardo Aguilar-Cordova (2012 - 2014)<br />
Members<br />
Dale G. Ando, MD (2010 - 2016)<br />
Joseph T. Bruder, PhD (2010 - 2013)<br />
Sunil Chada, PhD (2012 - 2015)<br />
Anthony T. Cheung, PhD (2008 - 2014)<br />
Olivier Danos, PhD (2013 - 2016)<br />
Mitchell H. Finer, PhD (2013 - 2016)<br />
Douglas J. Jolly, PhD (2013 - 2016)<br />
William F. Kaemmerer, PhD (2010 - 2016)<br />
David Kirn, MD (2012 - 2015)<br />
Karen Kozarsky, PhD (2007 - 2013)<br />
Timothy K. MacLachlan, PhD (2012 - 2015)<br />
Gabor M. Rubanyi, MD, PhD (2010 - 2013)<br />
Sean M. Sullivan, PhD (2010 - 2013)<br />
Thomas Tillett (2010 - 2013)<br />
Bruce Martin Wentworth, PhD (2013 - 2016)<br />
Cancer <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> Committee<br />
Chair<br />
Chiara Bonini, MD (2012 - 2014)<br />
Members<br />
Karen S. Aboody, MD (2011 - 2014)<br />
Margaret E. Black, PhD (2012 - 2013)<br />
Renier J. Brentjens, MD, PhD (2010 - 2013)<br />
Evanthia Galanis, MD, DSc (2011 - 2014)<br />
Stephen Gottschalk, MD (2007 - 2013)<br />
Paola Grandi, PhD (2011 - 2014)<br />
Akseli Hemminki, MD, PhD (2012 - 2015)<br />
Scott Lonning, DVM, PhD (2012 - 2015)<br />
J. Michael Mathis, PhD (2013 - 2016)<br />
John J. Nemunaitis, MD (2008 - 2014)<br />
Selvarangan Ponnazhagan, PhD (2012 - 2015)<br />
Barbara Savoldo, MD, PhD (2013 - 2016)<br />
Bakhos A. Tannous, PhD (2011 - 2014)<br />
Savio L.C. Woo, PhD (2010 - 2013)<br />
Masato Yamamoto, MD, PhD (2013 - 2016)<br />
Cardiovascular <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> Committee<br />
Chair<br />
David A. Dichek, MD (2012 - 2014)<br />
Members<br />
Michael P. Murphy, MD (2009 - 2015)<br />
Timothy O’Brien, MD, PhD (2009 - 2015)<br />
Michael J. Passineau, PhD (2010 - 2016)<br />
Lucio Pastore, MD, PhD (2013 - 2016)<br />
Gabor M. Rubanyi, MD, PhD (2010 - 2016)<br />
Matthew L. Springer, PhD (2007 - 2013)<br />
Ba-Bie Teng, PhD (2013 - 2016)<br />
Karen A. Vincent, PhD (2008 - 2014)<br />
Chemical <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> Committee<br />
Chair<br />
Ian MacLachlan, PhD (2011 - 2013)<br />
Kevin G. Rice, PhD (2013 - 2014)<br />
Members<br />
Stephen L. Hart, PhD (2011 - 2014)<br />
Philip L. Leopold, PhD (2012 - 2015)<br />
Ian MacLachlan, PhD (2010 - 2013)<br />
Suzie H. Pun, PhD (2007 - 2013)<br />
Kevin G. Rice, PhD (2010 - 2013)<br />
Cyrus R. Sainya, PhD (2010 - 2013)<br />
David B. Weiner, PhD (2010 - 2013)<br />
Clinical Trials and Regulatory Affairs Committee<br />
Chair<br />
Barry J. Byrne, MD, PhD (2012 - 2014)<br />
Members<br />
Dale G. Ando, MD (2012 - 2013)<br />
Wilson W. Bryan, MD (2012 - 2015)<br />
Jacqueline Corrigan-Curay, MD, JD (2008 - 2014)<br />
Katherine A. High, MD (2012 - 2015)<br />
David Kirn, MD (2012 - 2015)<br />
Maritza C. McIntyre, PhD (2013 - 2016)<br />
Peter K. Working, PhD, DABT (2010 - 2013)<br />
Development Committee<br />
Chair<br />
Barrie J. Carter, PhD (2012 - 2013)<br />
James M. Wilson, MD, PhD (2013 - 2014)<br />
Members<br />
Claudio Bordignon, MD (2012 - 2015)<br />
Malcolm K. Brenner, MD, PhD (2012 - 2015)<br />
Barrie J. Carter, PhD (2011 - 2014)<br />
E. Antonio Chiocca, MD, PhD (2012 - 2015)<br />
Terence R. Flotte, MD (2013 - 2016)<br />
Ryszard Kole, PhD (2012 - 2015)<br />
Brendan Lee, MD, PhD (2013 - 2016)<br />
Arthur W. Nienhuis, MD (2012 - 2015)<br />
John J. Rossi, PhD (2012 - 2015)<br />
James M. Wilson, MD, PhD (2012 - 2014)<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013<br />
7
Committee Rosters<br />
Education Committee<br />
Co-Chairs<br />
Hans-Peter Kiem, MD, FACP (2013 - 2014)<br />
Sonia I. Skarlatos, PhD (2011 - 2013)<br />
Luk H. Vandenberghe, PhD (2012 - 2014)<br />
Members<br />
Aravind Asokan, PhD (2013 - 2016)<br />
Terry R. Bishop, PhD (2013 - 2016)<br />
Brian D. Brown, PhD (2011 - 2014)<br />
Nathalie Cartier-Lacave, MD (2011 - 2014)<br />
William F. Goins, PhD (2007 - 2013)<br />
Dirk Grimm, PhD (2011 - 2014)<br />
Richard P. Harbottle, PhD (2013 - 2016)<br />
Scott Q. Harper, PhD (2010 - 2013)<br />
Tal Kafri, MD, PhD (2007 - 2013)<br />
Elizabeth Kang, MD (2011 - 2014)<br />
Hans-Peter Kiem, MD, FACP (2011 - 2013)<br />
Harry L. Malech, MD (2010 - 2013)<br />
Matthew H. Porteus, MD, PhD (2010 - 2016)<br />
Sonia I. Skarlatos, PhD (2011 - 2014)<br />
James Yockman, PhD (2012 - 2015)<br />
Ethics Committee<br />
Chair<br />
Theodore Friedmann, MD (2012 - 2013)<br />
Members<br />
R. Michael Blaese, MD (2010 - 2013)<br />
Theodore Friedmann, MD (2008 - 2014)<br />
Dieter C. Gruenert, PhD (2010 - 2013)<br />
Nancy M.P. King, JD (2012 - 2015)<br />
Bruce E. Torbett, PhD, MSPH (2012 - 2015)<br />
Nelson A. Wivel, MD (2010 - 2016)<br />
Exhibitor Relations Committee<br />
Chair<br />
Larry A. Couture, PhD (2013 - 2014)<br />
Members<br />
Janet Benson, PhD, DABT (2013 - 2016)<br />
Linda B. Couto, PhD (2013 - 2016)<br />
J. David Enloe, Jr., BBA CPA (2013 - 2016)<br />
Clague Hodgson, PhD (2013 - 2016)<br />
Ian Johnston, PhD (2013 - 2016)<br />
Karen Kozarsky, PhD (2013 - 2016)<br />
Financial Affairs Committee<br />
Chair<br />
Xandra O. Breakeield, PhD (2012 - 2013)<br />
Members<br />
Harry L. Malech, MD (2012 - 2015)<br />
John J. Rossi, PhD (2010 - 2013)<br />
David W. Russell, MD, PhD (2011 - 2014)<br />
David V. Schaffer, PhD (2011 - 2014)<br />
<strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> <strong>of</strong> <strong>Gene</strong>tic & Metabolic<br />
Diseases and Diabetes Committee<br />
Chair<br />
Lawrence C. Chan, MD (2012 - 2014)<br />
Members<br />
Valder R. Arruda, MD, PhD (2012 - 2013)<br />
Alessandra Bifi, MD (2011 - 2014)<br />
Fatima Bosch, PhD (2010 - 2016)<br />
Nicola Brunetti-Pierri, MD (2012 - 2015)<br />
Randy J. Chandler, PhD, MB (2012 - 2015)<br />
Seng H. Cheng, PhD (2013 - 2016)<br />
Stephanie Cherqui, PhD (2011 - 2014)<br />
Andrew M. David<strong>of</strong>f, MD (2010 - 2016)<br />
Dwight Koeberl, MD, PhD (2013 - 2016)<br />
Federico Mingozzi, PhD (2012 - 2015)<br />
Philip Ng, PhD (2013 - 2016)<br />
Sihong Song, PhD (2011 - 2014)<br />
Peter M. Thule, MD (2013 - 2016)<br />
Charles P. Venditti, MD, PhD (2008 - 2014)<br />
<strong>Gene</strong>tic Vaccines Committee<br />
Chair<br />
Jeffrey J. Molldrem, MD (2011 - 2013)<br />
Members<br />
Austin J. Barrett, MD (2010 - 2013)<br />
William J. Bowers, PhD (2012 - 2013)<br />
J. Keith Joung, MD, PhD (2012 - 2013)<br />
Sattva S. Neelapu, MD (2010 - 2013)<br />
Hematologic and Immunologic <strong>Gene</strong> and <strong>Cell</strong><br />
<strong>Therapy</strong> Committee<br />
Chair<br />
Alessandro Aiuti, MD, PhD (2013 - 2014)<br />
Cynthia Dunbar, MD (2012 - 2013)<br />
Members<br />
Alessandro Aiuti, MD, PhD (2008 - 2013)<br />
Fabio Candotti, MD (2013 - 2016)<br />
Gianpietro Dotti, MD (2010 - 2013)<br />
John T. Gray, PhD (2011 - 2014)<br />
Helen E. Heslop, MD (2011 - 2014)<br />
Dennis D. Hickstein, MD (2013 - 2016)<br />
J. Keith Joung, MD, PhD (2012 - 2015)<br />
Punam Malik, MD, PhD (2013 - 2016)<br />
Paris Margaritis, DPhil (2011 - 2014)<br />
Derek A. Persons, MD, PhD (2007 - 2013)<br />
Pankaj Qasba, PhD (2013 - 2016)<br />
Barbara Savoldo, MD, PhD (2010 - 2013)<br />
Evangelia Yannaki, MD (2012 - 2015)<br />
8<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013
Committee Rosters<br />
Host Committee<br />
Chair<br />
Xandra O. Breakeield, PhD (2011 - 2013)<br />
David W. Russell, MD, PhD (2013 - 2014)<br />
Members<br />
Janet Benson, PhD, DABT (2011 - 2013)<br />
James Brown, PhD (2013 – 2014)<br />
Xavier Danthinne, PhD (2011 - 2013)<br />
Giovanni Di Pasquale, PhD (2013 - 2014)<br />
Boro Dropulic, PhD (2013 - 2014)<br />
Howard J. Feder<strong>of</strong>f, MD, PhD (2013 - 2014)<br />
David T. Harris, PhD (2011 - 2013)<br />
Eric H<strong>of</strong>fman, PhD (2013 - 2014)<br />
Sung Wan Kim, PhD (2011 - 2013)<br />
Catherine McKeon, PhD (2013 - 2014)<br />
Paul Muhlrad, PhD (2011 - 2013)<br />
Immune Responses to <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong><br />
Chair<br />
Shulin Li, PhD (2012 - 2014)<br />
Members<br />
Catherine M. Bollard, MBChB, FRACP, FRCPA (2011 - 2014)<br />
Andrew P. Byrnes, PhD (2010 - 2016)<br />
Vincenzo Cerullo, PhD (2013 - 2016)<br />
Jonathan D. Finn, PhD (2013 - 2016)<br />
Roland W. Herzog, PhD (2010 - 2016)<br />
Song Li, MD, PhD (2012 - 2015)<br />
Gerald S. Lipshutz, MD (2010 - 2016)<br />
Pedro Lowenstein, MD, PhD (2012 - 2013)<br />
J. Michael Mathis, PhD (2013 - 2016)<br />
John D. Mountz, MD, PhD (2007 - 2013)<br />
Alistair Ramsay, PhD (2012 - 2015)<br />
Denise Sabatino, PhD (2008 - 2014)<br />
Renata Stripecke, PhD (2013 - 2016)<br />
Yiping Yang, MD, PhD (2010 - 2016)<br />
Xianzheng Zhou, MD, PhD (2012 - 2015)<br />
Infectious Diseases and Vaccines Committee<br />
Chair<br />
Margaret A. Liu, MD (2011 - 2013)<br />
Eric Poeschla, MD (2013 - 2014)<br />
Members<br />
William J. Bowers, PhD (2012 - 2015)<br />
Thomas W. Dubensky, Jr., PhD (2012 - 2015)<br />
Philip R. Johnson, MD (2010 - 2016)<br />
David Klatzmann, MD, PhD (2012 - 2015)<br />
Margaret A. Liu, MD (2011 - 2014)<br />
Jeffrey J. Molldrem, MD (2011 - 2014)<br />
Sattva S. Neelapu, MD (2010 - 2016)<br />
Eric Poeschla, MD (2010 - 2013)<br />
Steven Polyak, MD (2010 - 2013)<br />
Alain Rolland, PharmD, PhD (2012 - 2015)<br />
James M. Wilson, MD, PhD (2007 - 2013)<br />
International Committee<br />
Chair<br />
Bobby Gaspar, MD, PhD (2013 - 2014)<br />
Thierry C. VandenDriessche, PhD (2011 - 2013)<br />
Members<br />
Alberto Auricchio, MD (2010 - 2013)<br />
Etiena Basner-Tschakarjan, MD, PhD (2013 - 2016)<br />
Christopher Baum, MD (2010 - 2013)<br />
John C. Bell, PhD (2012 - 2015)<br />
Bobby Gaspar, MD, PhD (2008 - 2013)<br />
Salima Hacein-Bey Abina, PharmD, PhD (2008 - 2014)<br />
Yasufumi Kaneda, MD, PhD (2012 - 2015)<br />
Kazunori Kataoka, PhD (2012 - 2015)<br />
Paula Y. P. Lam, PhD (2013 - 2016)<br />
Kyriacos A. Mitrophanous, PhD (2012 - 2015)<br />
Amit Nathwani, MD, PhD (2011 - 2014)<br />
Thierry C. VandenDriessche, PhD (2010 - 2014)<br />
Chae-Ok Yun, PhD (2012 - 2015)<br />
Membership Committee<br />
Chair<br />
Guangping Gao, PhD (2011 - 2013)<br />
Thomas Conlon, PhD (2013 - 2014)<br />
Members<br />
Donna Armentano, PhD (2011 - 2014)<br />
Alberto Auricchio, MD (2011 - 2014)<br />
Margaret E. Black, PhD (2010 - 2013)<br />
Michele P. Calos, PhD (2010 - 2013)<br />
Resy Cavallesco Wharton, PhD (2013 - 2016)<br />
Thomas Conlon, PhD (2012 - 2015)<br />
Guangping Gao, PhD (2011 - 2014)<br />
David Kirn, MD (2011 - 2014)<br />
Toya Ohashi, MD (2008 - 2014)<br />
Keiya Ozawa, MD, PhD (2011 - 2014)<br />
Ex-Oficio<br />
Robert M. Frederickson, PhD (2005 - 2014)<br />
Musculo-Skeletal <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> Committee<br />
Chair<br />
Kathryn R. Wagner, MD, PhD (2012 - 2014)<br />
Members<br />
Jeffrey Chamberlain, PhD (2010 - 2013)<br />
Joel R. Chamberlain, PhD (2013 - 2016)<br />
Charles A. Gersbach, PhD (2012 - 2015)<br />
Richard J. Gregory, PhD (2010 - 2013)<br />
Dwight Koeberl, MD, PhD (2010 - 2016)<br />
Gloria Matthews, DVM, PhD,DACVS (2013 - 2016)<br />
Jerry R. Mendell, MD (2012 - 2013)<br />
Alan J. Nixon, DVM, MS (2007 - 2013)<br />
Shin’ichi Takeda, MD, PhD (2010 - 2016)<br />
Bing Wang, MD, PhD (2010 - 2016)<br />
Xiao Xiao, PhD (2012 - 2015)<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013<br />
9
Committee Rosters<br />
Neurologic & Ophthalmic <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong><br />
Committee<br />
Chair<br />
Anne Messer, PhD (2012 - 2014)<br />
Members<br />
Robin R. Ali, PhD (2010 - 2016)<br />
Alessandra Bifi, MD (2011 - 2014)<br />
Thomas W. Chalberg, Jr., PhD (2011 - 2014)<br />
Florian Eichler, MD (2011 - 2014)<br />
David J. Fink, MD (2010 - 2016)<br />
William W. Hauswirth, PhD (2010 - 2016)<br />
Alfred S. Lewin, PhD (2012 - 2015)<br />
Jacques JB Mallet, PhD (2012 - 2015)<br />
Jodi L. McBride, PhD (2013 – 2016)<br />
Seshidhar Reddy Police, DVM, MS, PhD (2011 - 2014)<br />
Miguel Sena-Esteves, PhD (2012 - 2015)<br />
Mark H. Tuszynski, MD, PhD (2012 - 2013)<br />
John H. Wolfe, VMD, PhD (2010 - 2016)<br />
New Investigator Committee<br />
Chair<br />
Matthew D. Weitzman, PhD (2011 - 2014)<br />
Members<br />
Jennifer E. Adair, PhD (2011 - 2014)<br />
F. Charles Brunicardi, MD (2011 - 2014)<br />
Paul Gadue, PhD (2011 - 2014)<br />
Scott Q. Harper, PhD (2011 - 2014)<br />
Brian K. Kaspar, PhD (2011 - 2014)<br />
Maria P. Limberis, PhD (2012 - 2015)<br />
Casey A. Maguire, PhD (2012 - 2015)<br />
Eirini P. Papapetrou, MD, PhD (2011 - 2014)<br />
Miguel Sena-Esteves, PhD (2011 - 2014)<br />
Johan Skog, PhD (2012 - 2015)<br />
Luk H. Vandenberghe, PhD (2011 - 2014)<br />
Andy Wilber, PhD (2011 - 2014)<br />
Nominating Committee<br />
Chair<br />
Kenneth Cornetta, MD (2012 - 2014)<br />
Members<br />
Dale G. Ando, MD (2012 - 2013)<br />
Helen E. Heslop, MD (2012 - 2013)<br />
Brendan Lee, MD, PhD (2011 - 2013)<br />
Kevin V. Morris, PhD (2012 - 2013)<br />
Oligonucleotide and RNAi Therapeutics Committee<br />
Chair<br />
Paloma H. Giangrande, PhD (2013 - 2014)<br />
Kevin V. Morris, PhD (2011 - 2013)<br />
Members<br />
C. Frank Bennett, PhD (2010 - 2016)<br />
Brian D. Brown, PhD (2010 - 2013)<br />
Paloma H. Giangrande, PhD (2012 - 2013)<br />
Dirk Grimm, PhD (2010 - 2013)<br />
Scott Q. Harper, PhD (2013 - 2016)<br />
Ryszard Kole, PhD (2011 - 2014)<br />
Matthew Levy, PhD (2011 - 2014)<br />
Paul B. McCray, Jr., MD (2008 - 2014)<br />
Nuria Morral, PhD (2012 - 2015)<br />
Kevin V. Morris, PhD (2011 - 2014)<br />
Marco S. Weinberg, PhD (2010 - 2016)<br />
Outreach Committee<br />
Chair<br />
Rachel Salzman, DVM (2013 - 2014)<br />
Savio L.C. Woo, PhD (2011 - 2013)<br />
Members<br />
Timothy R. Cote, MD, MPH (2011 - 2014)<br />
Joseph C. Glorioso, III, PhD (2011 - 2014)<br />
Jane S. Lebkowski, PhD (2011 - 2014)<br />
Brendan Lee, MD, PhD (2012 - 2013)<br />
Harry L. Malech, MD (2011 - 2014)<br />
Andra E. Miller, PhD (2011 - 2014)<br />
Rachel Salzman, DVM (2011 - 2014)<br />
James M. Wilson, MD, PhD (2012 - 2015)<br />
Savio L.C. Woo, PhD (2011 - 2014)<br />
Physical <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> and Vectorology<br />
Committee<br />
Chair<br />
Richard Heller, PhD (2011 - 2013)<br />
R. Scott McIvor, PhD (2013 - 2014)<br />
Members<br />
Michele P. Calos, PhD (2007 - 2013)<br />
David A. Dean, PhD (2013 - 2016)<br />
Julie Gehl, MD, Dr.Med.Sci. (2013 - 2016)<br />
James G. Hecker, MD, PhD (2013 - 2016)<br />
Richard Heller, PhD (2011 - 2014)<br />
Dexi Liu, PhD (2013 - 2016)<br />
R. Scott McIvor, PhD (2009 - 2015)<br />
Carol H. Miao, PhD (2009 - 2015)<br />
Sean M. Sullivan, PhD (2010 - 2013)<br />
Ernst Wagner, PhD (2012 - 2015)<br />
10<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013
Committee Rosters<br />
<strong>Program</strong> Committee<br />
Co-Chairs<br />
Daniel G. Anderson, PhD (2012 - 2013)<br />
Xandra O. Breakeield, PhD (2012 - 2013)<br />
Maria G. Castro, PhD (2011 - 2013)<br />
Gay Crooks, MD (2011 - 2013)<br />
R. Scott McIvor, PhD (2013 – 2015)<br />
John J. Nemunaitis, MD (2013 - 2015)<br />
David W. Russell, MD, PhD (2013 - 2014)<br />
Sonia I. Skarlatos, PhD (2013 - 2015)<br />
Publications Committee<br />
Chair<br />
Cynthia Dunbar, MD (2013 - 2014)<br />
Francis C. Szoka, Jr., PhD (2011 - 2013)<br />
Members<br />
Hans-Peter Kiem, MD, FACP (2007 - 2013)<br />
Arthur W. Nienhuis, MD (2010 - 2016)<br />
Stephen J. Russell, MD, PhD (2012 - 2015)<br />
Michel Sadelain, MD, PhD (2007 - 2013)<br />
Rachel Salzman, DVM (2008 - 2014)<br />
Francis C. Szoka, Jr., PhD (2011 - 2014)<br />
Respiratory Tract <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> Committee<br />
Chair<br />
John F. Engelhardt, PhD (2013 - 2014)<br />
Terence R. Flotte, MD (2011 - 2013)<br />
Members<br />
Mark J. Cooper, MD (2012 - 2015)<br />
David A. Dean, PhD (2010 - 2013)<br />
John F. Engelhardt, PhD (2010 - 2013)<br />
Terence R. Flotte, MD (2011 - 2014)<br />
Uta Griesenbach, PhD (2010 - 2013)<br />
Dieter C. Gruenert, PhD (2013 - 2016)<br />
William B. Guggino, PhD (2013 - 2016)<br />
Jay K. Kolls, MD (2007 - 2013)<br />
Maria P. Limberis, PhD (2013 - 2016)<br />
Christian Mueller, PhD (2012 - 2015)<br />
David W. Parsons, PhD (2007 - 2013)<br />
Stem <strong>Cell</strong> Committee<br />
Chair<br />
Karen S. Aboody, MD (2012 - 2014)<br />
Members<br />
James Ellis, PhD (2009 - 2015)<br />
Paul Gadue, PhD (2013 - 2016)<br />
Jane S. Lebkowski, PhD (2010 - 2016)<br />
Carolyn Lutzko, PhD (2013 - 2016)<br />
Eirini P. Papapetrou, MD, PhD (2013 - 2016)<br />
Alice F. Tarantal, PhD (2009 - 2015)<br />
Translational Science and Product Development<br />
Committee<br />
Chair<br />
Boro Dropulic, PhD (2012 - 2014)<br />
Members<br />
Joy Cavagnaro, PhD, DABT, ATS (2010 - 2016)<br />
Douglas J. Jolly, PhD (2010 - 2016)<br />
<strong>Gene</strong> Liau, PhD (2012 - 2015)<br />
Dexi Liu, PhD (2013 - 2016)<br />
Ian MacLachlan, PhD (2010 - 2013)<br />
Phillip B. Maples, PhD (2013 - 2016)<br />
Maritza C. McIntyre, PhD (2012 - 2013)<br />
Alain Rolland, PharmD, PhD (2010 - 2016)<br />
Richard O. Snyder, PhD (2010 - 2016)<br />
Ge<strong>of</strong>f P. Symonds, PhD (2012 - 2015)<br />
Dale VanderPutten, MBA, PhD (2010 - 2013)<br />
Gabor Veres, PhD (2012 - 2015)<br />
Liaison<br />
Jakob Reiser, PhD (2011 - 2014)<br />
Viral <strong>Gene</strong> Transfer Vectors Committee<br />
Chair<br />
John T. Gray, PhD (2012 - 2014)<br />
Members<br />
Mavis Agbandje-McKenna, PhD (2010 - 2016)<br />
Ian E. Alexander, MBBS, PhD (2011 - 2014)<br />
Jeffrey S. Bartlett, PhD (2013 - 2016)<br />
Haifeng Chen, PhD (2013 - 2016)<br />
John A. Chiorini, PhD (2010 - 2016)<br />
Kevin D. Foust, PhD (2012 - 2015)<br />
Guangping Gao, PhD (2013 - 2016)<br />
Stephen J. Russell, MD, PhD (2010 - 2016)<br />
Dmitry M. Shayakhmetov, PhD (2009 - 2015)<br />
Jackie Sheng, PhD (2011 - 2014)<br />
H. Trent Spencer, PhD (2012 - 2015)<br />
Weidong Xiao, PhD (2012 - 2015)<br />
To learn how to serve on an ASGCT Committee, visit the ASGCT Membership Booth!<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013<br />
11
Abstract Planning Committee<br />
Each year this committee, which is made up <strong>of</strong> the ASGCT President, President-Elect, Abstract Review Chair, <strong>Program</strong><br />
Committee Co-Chairs and two invited members, reviews abstracts that are suggested by the various categories for the<br />
Presidential Symposium and the Clinical Oral Abstract Session.<br />
Daniel G. Anderson, PhD<br />
Massachusetts Institute <strong>of</strong> Technology<br />
Cambridge, MA<br />
Xandra O. Breakefield, PhD<br />
Massachusetts <strong>Gene</strong>ral Hospital and<br />
Harvard Medical School<br />
Charlestown, MA<br />
Abstract Reviewers<br />
Abstract Committee Chair<br />
Kenneth Cornetta, MD<br />
Kenneth Cornetta, MD<br />
Indiana University<br />
Indianapolis, IN<br />
Pedro Lowenstein, MD, PhD<br />
University <strong>of</strong> Michigan School <strong>of</strong><br />
Medicine<br />
Ann Arbor, MI<br />
David W. Russell, MD, PhD<br />
University <strong>of</strong> Washington<br />
Seattle, WA<br />
Michel Sadelain, MD, PhD<br />
Memorial Sloan-Kettering Cancer<br />
Center<br />
New York, NY<br />
RNA Virus Vectors<br />
Abstract Review Chair<br />
Dorothee von Laer, MD<br />
Reviewers<br />
Jennifer Adair, PhD*<br />
Brian D. Brown, PhD<br />
Cynthia Dunbar, MD<br />
Rik Gijsbers, PhD*<br />
AAV Vectors<br />
Abstract Review Chair<br />
Aravind Asokan, PhD<br />
Reviewers<br />
Mavis Agbandje-McKenna, PhD<br />
Casey A. Maguire, PhD*<br />
Hiroyuki Nakai, MD, PhD<br />
David V. Schaffer, PhD<br />
Adenovirus and<br />
Other DNA Virus Vectors<br />
Abstract Review Chair<br />
James M. Wilson, MD, PhD<br />
Reviewers<br />
Soumitra Roy, MD, PhD*<br />
Miguel Sena-Esteves, PhD<br />
Prem Seth, PhD*<br />
William S.M. Wold, PhD<br />
DNA Vectorology & <strong>Gene</strong><br />
Targeting<br />
Abstract Review Chair<br />
Matthew H. Porteus, MD, PhD<br />
Reviewers<br />
Matthew Hirsch, PhD*<br />
Carol Miao, PhD<br />
Shondra Miller, PhD*<br />
Andy Wilber, PhD<br />
Physical Methods <strong>of</strong> Delivery<br />
Abstract Review Chair<br />
Kevin G. Rice, PhD<br />
Reviewers<br />
Todd Giorgio, PhD<br />
Richard Heller, PhD<br />
King F. Kwong, MD*<br />
Sean M. Sullivan, PhD<br />
Chemical and Molecular<br />
Conjugates<br />
Abstract Review Chair<br />
Young Jik Kwon, PhD<br />
Reviewers<br />
Hai-Quan Mao, PhD<br />
Tatiana Segura, PhD<br />
Lonnie D. Shea, PhD<br />
Chun Wang, PhD<br />
<strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> <strong>of</strong> Diabetes<br />
and Metabolic and <strong>Gene</strong>tic<br />
Diseases<br />
Abstract Review Chair<br />
Nuria Morral, PhD<br />
Reviewers<br />
Nicola Brunetti-Pierri, MD<br />
Stephanie Cherqui, PhD*<br />
Gerald S. Lipshutz, MD*<br />
Sihong Song, PhD*<br />
Cardiovascular <strong>Gene</strong> & <strong>Cell</strong><br />
<strong>Therapy</strong><br />
Abstract Review Chair<br />
Thomas Weber, PhD<br />
Reviewers<br />
Michael A. Barry, PhD<br />
Dongsheng Duan, PhD<br />
Katherine P. Ponder, MD<br />
Joseph E. Rabinowitz, PhD<br />
Neurologic <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong><br />
Abstract Review Chair<br />
Nicholas Boulis, MD<br />
Reviewers<br />
Mimoun Azzouz, PhD*<br />
David J. Fink, MD<br />
Brian Kaspar, PhD<br />
Scott McPhee, PhD*<br />
Musculo-skeletal <strong>Gene</strong> & <strong>Cell</strong><br />
<strong>Therapy</strong><br />
Abstract Review Chair<br />
Charles A. Gersbach, PhD<br />
Reviewers<br />
Annemieke Aartsma-Rus, PhD*<br />
Louise Rodino-Klapac, PhD*<br />
Jacques Tremblay, PhD<br />
Xiao Xiao, PhD<br />
Infectious Diseases and Vaccines<br />
Abstract Review Chair<br />
Grant D. Trobridge, PhD<br />
Reviewers<br />
Dong Sung An, MD, PhD*<br />
K. Reed Clark, PhD<br />
Bruce L. Levine, PhD<br />
Renata Stripecke, PhD*<br />
12<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013
Abstract Reviewers<br />
Cancer - Immunotherapy<br />
Abstract Review Chair<br />
Richard G. Vile, PhD<br />
Reviewers<br />
Steven M. Albelda, MD*<br />
Vincenzo Cerullo, PhD<br />
Jeffrey J. Molldrem, MD<br />
Michael I. Nishimura, PhD*<br />
Cancer – Oncolytic Viruses<br />
Abstract Review Chair<br />
Grant McFadden, PhD<br />
Reviewers<br />
John C. Bell, PhD*<br />
David Kirn, MD<br />
Brian Lichty, PhD*<br />
Stephen J. Russell, MD, PhD<br />
Cancer - Targeted <strong>Gene</strong> & <strong>Cell</strong><br />
<strong>Therapy</strong><br />
Abstract Review Chair<br />
Evanthia Galanis, MD, DSc<br />
Reviewers<br />
Karen S. Aboody, MD<br />
Helen E. Heslop, MD<br />
Noriyuki Kasahara, MD, PhD<br />
Janaiah Kota, PhD*<br />
Hematologic and Immunologic<br />
<strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong><br />
Abstract Review Chair<br />
Fabio Candotti, MD<br />
Reviewers<br />
Marina Cavazzana-Calvo, MD, PhD<br />
Bobby Gaspar, MD, PhD<br />
Arnab Ghosh, MD, PhD*<br />
David A. Wilcox, PhD*<br />
<strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> for<br />
Pulmonary Diseases<br />
Abstract Review Chair<br />
Uta Griesenbach, PhD<br />
Reviewers<br />
Katherine J. Exc<strong>of</strong>fon, PhD*<br />
Stephen L. Hart, PhD<br />
Jim Hu, PhD<br />
Maria P. Limberis, PhD<br />
Immunologic & Host Responses<br />
in <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong><br />
Abstract Review Chair<br />
Pedro Lowenstein, MD, PhD<br />
Reviewers<br />
Renier J. Brentjens, MD, PhD<br />
Antonia Follenzi, MD, PhD*<br />
Maciej S. Lesniak, MD, MHCM, FACS<br />
Federico Mingozzi, PhD*<br />
Sensory (Ophthalmic and<br />
Auditory) <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong><br />
Abstract Review Chair<br />
Jean Bennett, MD, PhD<br />
Reviewers<br />
Alberto Auricchio, MD<br />
Rajendra Kumar-Singh, PhD*<br />
Tonia Rex, PhD*<br />
Luk H. Vandenberghe, PhD<br />
<strong>Gene</strong> Regulation<br />
Abstract Review Chair<br />
David W. Emery, PhD<br />
Reviewers<br />
Donna Armentano, PhD<br />
Michelle P. Calos, PhD<br />
Punam Malik, MD, PhD<br />
David M. Spencer, PhD<br />
Oligonucleotide & RNAi<br />
Therapeutics<br />
Abstract Review Chair<br />
Paloma H. Giangrande, PhD<br />
Reviewers<br />
Scott Q. Harper, PhD<br />
Shawn E. Lupold, PhD*<br />
M. Corinna Palanca-Wessels, MD, PhD*<br />
Laura Sepp-Lorenzino, PhD<br />
Stem <strong>Cell</strong> Therapies<br />
Abstract Review Chair<br />
Michel Sadelain, MD, PhD<br />
Reviewers<br />
Paul Gadue, PhD<br />
Hans-Peter Kiem, MD, FACP<br />
Jan A. Nolta, PhD<br />
Darwin J. Prockop, MD, PhD<br />
<strong>Cell</strong> Processing and Vector<br />
Production<br />
Abstract Review Chair<br />
Adrian P. Gee, PhD<br />
Reviewers<br />
Gerhard Bauer, PhD<br />
Steven A. Feldman, PhD*<br />
Isabelle Riviere, PhD<br />
Sowmya Viswanathan, PhD*<br />
Special thanks to the abstract reviewers for their time and expertise!<br />
*Indicates irst time reviewer for ASGCT.<br />
To volunteer to review abstracts for the 17th Annual Meeting, visit the ASGCT Membership booth.<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013<br />
13
Excellence in Research Awards & Travel Grant Recipients<br />
Excellence in Research Awards – For Students and Postdoctoral Fellows<br />
Luca Biasco<br />
San Raffaele Telethon Institute for <strong>Gene</strong> <strong>Therapy</strong><br />
Comprehensive Study <strong>of</strong> Lentiviral [quot]Integrome[quot] in<br />
WAS <strong>Gene</strong> <strong>Therapy</strong> Patients<br />
Sourav Choudhury<br />
University <strong>of</strong> Massachusetts Medical School<br />
Identiication <strong>of</strong> Novel Vectors Capable <strong>of</strong> CNS Transduction<br />
in Adult Mice after Single Round Selection Using DNA Shufled<br />
AAV Capsid Library<br />
Drew Deniger<br />
University <strong>of</strong> Texas MD Anderson Cancer Center<br />
Clinical Implications <strong>of</strong> ROR1-Speciic T <strong>Cell</strong>s That Target<br />
B-<strong>Cell</strong> Leukemia<br />
Charles Lai<br />
Massachusetts <strong>Gene</strong>ral Hospital<br />
Non-Invasive In Vivo Imaging, Biodistribution and Clearance<br />
Analyses <strong>of</strong> Intravenously Administered Extracellular Vesicles<br />
Rajiv Sharma<br />
Children’s Hospital <strong>of</strong> Philadelphia<br />
In Vivo ZFN Mediated Targeting <strong>of</strong> Albumin as a Platform for<br />
Expression <strong>of</strong> Multiple Therapeutic <strong>Gene</strong>s<br />
Barbara Smith<br />
University <strong>of</strong> Florida<br />
Acid alpha-Glucosidase <strong>Gene</strong> Replacement <strong>Therapy</strong> to the<br />
Diaphragm in Ventilator-Dependent Pompe Disease: One-Year<br />
Respiratory Motor Outcomes<br />
Travel Grants – For Students and Postdoctoral Fellows<br />
Erik Abels<br />
Kei Adachi<br />
Seemin Ahmed<br />
Mahzad Akbarpour<br />
Andrea Annoni<br />
Usanarat Anurathapan<br />
Claudia Arambula<br />
Cynthia Bartholomae<br />
Luca Biasco<br />
Aaron Black<br />
Kathleen Boerner<br />
Nicolas Boisgerault<br />
Florie Borel<br />
Francesca Bortolotti<br />
Allison Bradbury<br />
Elizabeth Brooks<br />
Christopher Burtner<br />
Leah Byrne<br />
Andrea Calabria<br />
Hua Cao<br />
Michael Castle<br />
Anna Castleton<br />
Soo Kyung Cho<br />
Szu-Ting Chou<br />
Sourav Choudhury<br />
Julie Crudele<br />
Drew Deniger<br />
Mark Doyal<br />
Theodore Drivas<br />
Brett Dufour<br />
F. Jason Duncan<br />
Frank Dyka<br />
Lauriel Earley<br />
Salim El-Amouri<br />
Colin Exline<br />
Susan Faust<br />
Antonio Filareto<br />
Eli Fine<br />
Richard Gabriel<br />
Pietro Genovese<br />
Evelyne Gicquel<br />
Melissa Goddard<br />
Diane Golebiowski<br />
Sara Gombash<br />
German Gornalusse<br />
Yue Guan<br />
Dwijit GuhaSarkar<br />
Marie Reine Haddad<br />
Chady Hakim<br />
Jingfen Han<br />
Jayson Hardcastle<br />
Hiromi<br />
Hayashita-Kinoh<br />
Kristin Heller<br />
Luiza Hernandez<br />
Mari Hirvinen<br />
Eloise Hudry<br />
Eric Irons<br />
Alok Joglekar<br />
Ami Kabadi<br />
Christine Kaeppel<br />
Shuichi Kamijima<br />
Kerstin Kaufmann<br />
Omkar Kawalekar<br />
Christiaan King<br />
Nico Lachmann<br />
Charles Lai<br />
Hongtao Li<br />
Li Li<br />
Shaoyong Li<br />
Nick Llewellyn<br />
Chantale Maltais<br />
Victoria McCurdy<br />
Tanner Miest<br />
Yoshiaki Miura<br />
Shohei Miyamoto<br />
Giridhar Murlidharan<br />
Bartholomew<br />
Naughton<br />
Sarah Nicolson<br />
Kyla Ortved<br />
David Ousterout<br />
Nunzia Pastore<br />
Pasquale Piccolo<br />
Karin Pike-Overzet<br />
Eric Pozsgai<br />
Jan-Michael Prill<br />
Marco Ranzani<br />
Maximillian Richter<br />
Fabrizio Rinaldi<br />
Julie Riviere<br />
Zhechao Ruan<br />
Giulia Ruozi<br />
Anna Sacher<br />
Lluis Samaranch<br />
Dwaipayan Sen<br />
Julien Senac<br />
Anurag Sharma<br />
Rajiv Sharma<br />
Shen Shen<br />
Kahori Shimizu<br />
Barbara Smith<br />
Arati Sridharan<br />
Merel Stok<br />
Rui-Jun Su<br />
Colin Sweeney<br />
Mehran Taherian<br />
William Thiel<br />
Lisa Tobin<br />
Shengdar Tsai<br />
Mallory Turner<br />
Niek van Til<br />
Hiu Man Viecelli<br />
Olga Villamizar<br />
Monica Volpin<br />
Hongjie Wang<br />
Rachael Watson<br />
Korashon Watts<br />
Cara Weismann<br />
Anna-Sophia<br />
Wiekmeijer<br />
Jun Xie<br />
Sea Young Yoon<br />
Roma Yumul<br />
The Excellence in Research Awards and Travel Awards were made possible through grant support from:<br />
14<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013
Outstanding New Investigator Awards<br />
The following Outstanding New Investigator Award winners were selected by the ASGCT Advisory Council based on<br />
merit and their contributions to the ield <strong>of</strong> gene and cell therapy. The recipients will present their work at the special<br />
Outstanding New Investigator Symposium on Saturday, May 18th from 8:00 am – 10:00 am.<br />
Aravind Asokan, PhD<br />
University <strong>of</strong> North Carolina at Chapel Hill<br />
A Sweet Side to AAV Biology<br />
Michael A. Laflamme, MD, PhD<br />
University <strong>of</strong> Washington<br />
Cardiac Repair with Human Pluripotent Stem<br />
<strong>Cell</strong>s<br />
Paloma H. Giangrande, PhD<br />
University <strong>of</strong> Iowa<br />
Targeted-Image Guided RNA Therapies<br />
Ann M. Leen, PhD<br />
Baylor College <strong>of</strong> Medicine - CAGT<br />
T cell <strong>Therapy</strong> for Viruses and Cancer (TIGR)<br />
The Outstanding New Investigator Awards were made possible through grant support from:<br />
Distinguished Service Award<br />
The ASGCT Distinguished Service Award recognizes a person or group that has consistently fostered and enhanced<br />
the ield <strong>of</strong> genetic and cellular therapy.<br />
Sonia I. Skarlatos, PhD<br />
National Heart Lung Blood Institute<br />
National Institutes <strong>of</strong> Health<br />
Catherine McKeon, PhD<br />
National Institute <strong>of</strong> Diabetes and Digestive<br />
and Kidney Diseases<br />
National Institutes <strong>of</strong> Health<br />
Outstanding Achievement Award<br />
The Outstanding Achievement Award winner was selected<br />
by the ASGCT Advisory Council as an example <strong>of</strong> an ASGCT<br />
member who has achieved a pioneering research success<br />
through a lifetime <strong>of</strong> signiicant scientiic contributions to the<br />
ield <strong>of</strong> gene and cell therapy.<br />
Mark A. Kay, MD, PhD<br />
Stanford University School <strong>of</strong> Medicine<br />
Deciphering Vector and Non-coding RNA<br />
Biology: A New Frontier in Nucleic Acid based<br />
Therapies.<br />
Special Recognition Award<br />
The Special Recognition Award is given in acknowledgement<br />
and appreciation <strong>of</strong> an individual’s many scientiic<br />
contributions and dedication to not only the <strong>Society</strong> itself,<br />
but to the ield <strong>of</strong> gene and cell therapy.<br />
John J. Rossi, PhD<br />
Beckman Research Institute<br />
City <strong>of</strong> Hope<br />
The Distinguished Service Award, Special Recognition Award, and Outstanding Achievement Award Lecture will be presented<br />
on Friday, May 17 from 8:00 am – 9:00 am.<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013<br />
15
Faculty List<br />
Annemieke Aartsma-Rus, PhD<br />
Leiden University Medical Center<br />
Leiden, Netherlands<br />
Karen S. Aboody, MD<br />
Beckman Research Institute <strong>of</strong> the City<br />
<strong>of</strong> Hope<br />
Duarte, CA<br />
Jennifer E. Adair, PhD<br />
Fred Hutchinson Cancer Research<br />
Center<br />
Seattle, WA<br />
Mavis Agbandje-McKenna, PhD<br />
University <strong>of</strong> Florida<br />
Gainesville, FL<br />
Estuardo Aguilar-Cordova, PhD<br />
Advantagene, Inc.<br />
Auburndale, MA<br />
Alessandro Aiuti, MD, PhD<br />
San Raffaele-Telethon Institute for <strong>Gene</strong><br />
<strong>Therapy</strong><br />
Milano, Italy<br />
Ian E. Alexander, MBBS, PhD<br />
The Children’s Hospital at Westmead<br />
Sydney, Australia<br />
Daniel G. Anderson, PhD<br />
Massachusetts Institute <strong>of</strong> Technology<br />
Cambridge, MA<br />
Dale G. Ando, MD<br />
Sangamo BioSciences, Inc.<br />
Richmond, CA<br />
Khursheed Anwer, PhD<br />
Expression <strong>Gene</strong>tics, Inc.<br />
Huntsville, AL<br />
Aravind Asokan, PhD<br />
University <strong>of</strong> North Carolina at<br />
Chapel Hill<br />
Chapel Hill, NC<br />
Alberto Auricchio, MD<br />
Telethon Institute <strong>of</strong> <strong>Gene</strong>tics &<br />
Medicine<br />
Napoli, Italy<br />
You Han Bae, PhD<br />
University <strong>of</strong> Utah<br />
Salt Lake City, UT<br />
Andrew H. Baker, PhD<br />
University <strong>of</strong> Glasgow<br />
Glasgow, United Kingdom<br />
Alejandro B. Balazs, PhD<br />
California Institute <strong>of</strong> Technology<br />
Pasadena, CA<br />
Andrea Ballabio, MD<br />
Telethon Institute <strong>of</strong> <strong>Gene</strong>tics and<br />
Medicine<br />
Naples, Italy<br />
David Baltimore, PhD<br />
California Institute <strong>of</strong> Technology<br />
Pasadena, CA<br />
Kryst<strong>of</strong> Bankiewicz, MD, PhD<br />
University <strong>of</strong> California San Francisco<br />
San Francisco, CA<br />
Raymond T. Bartus, PhD<br />
Ceregene, Inc.<br />
San Diego, CA<br />
John C. Bell, PhD<br />
Ottawa Hospital Research Institute<br />
Ottawa, Canada<br />
C. Frank Bennett, PhD<br />
Isis Pharmaceuticals, Inc.<br />
Carlsbad, CA<br />
Janet Benson, PhD, DABT<br />
Lovelace Respiratory Research Institute<br />
Albuquerque, NM<br />
Alessandra Biffi, MD<br />
Telethon Institute <strong>of</strong> <strong>Gene</strong>tics and<br />
Medicine<br />
Milano, Italy<br />
Brian W. Bigger, PhD<br />
University <strong>of</strong> Manchester<br />
Manchester, United Kingdom<br />
Fabrizia Bignami, PhD<br />
GlaxoSmithKline<br />
Brentford, United Kingdom<br />
Nicholas Boulis, MD<br />
Emory University School <strong>of</strong> Medicine<br />
Atlanta, GA<br />
Shannon E. Boye, PhD<br />
University <strong>of</strong> Florida<br />
Gainesville, FL<br />
Xandra O. Breakefield, PhD<br />
Massachusetts <strong>Gene</strong>ral Hospital<br />
Harvard Medical School<br />
Charlestown, MA<br />
Malcolm K. Brenner, MD, PhD<br />
Baylor College <strong>of</strong> Medicine<br />
Houston, TX<br />
Renier J. Brentjens, MD, PhD<br />
Memorial Sloan-Kettering Cancer<br />
Center<br />
New York, NY<br />
Charles R. Bridges, MD, ScD<br />
Sanger Heart and Vascular Institute<br />
Charlotte, NC<br />
Philip J. Brooks, PhD<br />
National Center for Advancing<br />
Translational Sciences (NCATS)<br />
National Institutes <strong>of</strong> Health<br />
Bethesda, MD<br />
Brian D. Brown, PhD<br />
Mount Sinai School <strong>of</strong> Medicine<br />
New York, NY<br />
Nicola Brunetti-Pierri, MD<br />
Telethon Institute <strong>of</strong> <strong>Gene</strong>tics and<br />
Medicine<br />
Napoli, Italy<br />
Ana Buj Bello, MD, PhD<br />
<strong>Gene</strong>thon<br />
Evry, France<br />
Barry J. Byrne, MD, PhD<br />
University <strong>of</strong> Florida College <strong>of</strong><br />
Medicine<br />
Gainesville, FL<br />
Fabio Candotti, MD<br />
National Institutes <strong>of</strong> Health<br />
National Human Genome Research<br />
Institute<br />
Bethesda, MD<br />
Mario R. Capecchi, PhD<br />
University <strong>of</strong> Utah<br />
Salt Lake City, UT<br />
Drew E. Carlson, PhD<br />
National Institutes <strong>of</strong> Health<br />
National Heart, Lung, and Blood<br />
Institute<br />
Bethesda, MD<br />
Dana Carroll, PhD<br />
University <strong>of</strong> Utah School <strong>of</strong> Medicine<br />
Salt Lake City, UT<br />
Barrie J. Carter, PhD<br />
BioMarin Pharmaceutical<br />
Seattle, WA<br />
16<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013
Faculty List<br />
Thomas W. Chalberg, Jr., PhD<br />
Avalanche Biotechnologies, Inc.<br />
San Francisco, CA<br />
Jeffrey Chamberlain, PhD<br />
University <strong>of</strong> Washington School <strong>of</strong><br />
Medicine<br />
Seattle, WA<br />
Lawrence C. Chan, MD<br />
Baylor College <strong>of</strong> Medicine<br />
Houston, TX<br />
Randy J. Chandler, PhD, MB<br />
National Institutes <strong>of</strong> Health<br />
National Human Genome Research<br />
Institute<br />
Bethesda, MD<br />
Lieping Chen, MD, PhD<br />
Yale University <strong>of</strong> School <strong>of</strong> Medicine<br />
New Haven, CT<br />
Theresa Chen, PhD<br />
Food and Drug Administration<br />
Rockville, MD<br />
Jianjun Cheng, PhD<br />
University <strong>of</strong> Illinois at<br />
Urbana-Champaign<br />
Urbana, IL<br />
Stephanie Cherqui, PhD<br />
University <strong>of</strong> California, San Diego<br />
La Jolla, CA<br />
Anthony T. Cheung, PhD<br />
En<strong>Gene</strong>, Inc.<br />
Vancouver, Canada<br />
Ian Constable, AO<br />
Lions Eye Institute<br />
Nedlands, Australia<br />
Laurence JN Cooper, MD, PhD<br />
University <strong>of</strong> Texas M.D. Anderson<br />
Cancer Center<br />
Houston, TX<br />
Kenneth Cornetta, MD<br />
Indiana University<br />
Indianapolis, IN<br />
Ronald G. Crystal, MD<br />
Weill Medical College <strong>of</strong> Cornell<br />
University<br />
New York, NY<br />
Andrew M. David<strong>of</strong>f, MD<br />
St. Jude Children’s Research Hospital<br />
Memphis, TN<br />
Michele De Luca, MD<br />
University <strong>of</strong> Modena and Reggio<br />
Emilia<br />
Modena, Italy<br />
David A. Dean, PhD<br />
University <strong>of</strong> Rochester<br />
Rochester, NY<br />
Vojo Deretic, PhD<br />
University <strong>of</strong> New Mexico School <strong>of</strong><br />
Medicine<br />
Albuquerque, NM<br />
David A. Dichek, MD<br />
University <strong>of</strong> Washington<br />
Seattle, WA<br />
Gianpietro Dotti, MD<br />
Baylor College <strong>of</strong> Medicine<br />
Houston, TX<br />
Dongsheng Duan, PhD<br />
University <strong>of</strong> Missouri School <strong>of</strong><br />
Medicine<br />
Columbia, MO<br />
Cynthia Dunbar, MD<br />
National Institutes <strong>of</strong> Health<br />
National Heart Lung and Blood<br />
Institute<br />
Bethesda, MD<br />
Anja Ehrhardt, PhD<br />
Private University Witten/Herdecke<br />
Witten, Germany<br />
David W. Emery, PhD<br />
University <strong>of</strong> Washington<br />
Seattle, WA<br />
Christine Esau, PhD<br />
Regulus Therapeutics<br />
Carlsbad, CA<br />
Katherine J. Exc<strong>of</strong>fon, PhD<br />
Wright State University<br />
Dayton, OH<br />
Lukas Flatz, MD<br />
University Hospital <strong>of</strong> Lausanne<br />
Lausanne, Switzerland<br />
Leonard M. Fleck, PhD<br />
Michigan State University<br />
East Lansing, MI<br />
Terence R. Flotte, MD<br />
University <strong>of</strong> Massachusetts Medical<br />
School<br />
Worcester, MA<br />
Antonia Follenzi, MD, PhD<br />
University <strong>of</strong> Piemonte Orientale ‘A.<br />
Avogadro’<br />
Novara, Italy<br />
Zoe Fonseca-Kelly, PhD<br />
Harvard Medical School<br />
Boston, MA<br />
Robert M. Frederickson, PhD<br />
Molecular <strong>Therapy</strong><br />
Seattle, WA<br />
Svend O. Freytag, PhD<br />
Henry Ford Health System<br />
Detroit, MI<br />
Keith Gagnon, PhD<br />
University <strong>of</strong> Texas Southwestern<br />
Medical Center<br />
Dallas, TX<br />
Evanthia Galanis, MD, DSc<br />
Mayo Clinic<br />
Rochester, MN<br />
Daniel Gaudet, MD, PhD<br />
University <strong>of</strong> Montreal<br />
Montreal, Canada<br />
Andrew Geall, PhD<br />
Novartis<br />
Cambridge, MA<br />
Adrian P. Gee, PhD<br />
Baylor College <strong>of</strong> Medicine<br />
Houston, TX<br />
Charles A. Gersbach, PhD<br />
Duke University<br />
Durham, NC<br />
Hamid Ghandehari, PhD<br />
University <strong>of</strong> Utah<br />
Salt Lake City, UT<br />
Arnab Ghosh, MD, PhD<br />
Memorial Sloan-Kettering Cancer<br />
Center<br />
New York, NY<br />
Paloma H. Giangrande, PhD<br />
University <strong>of</strong> Iowa<br />
Iowa City, IA<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013<br />
17
Faculty List<br />
Harvinder Singh Gill, PhD<br />
Texas Tech University<br />
Lubbock, TX<br />
Joseph C. Glorioso, III, PhD<br />
University <strong>of</strong> Pittsburgh<br />
Pittsburgh, PA<br />
William F. Goins, PhD<br />
University <strong>of</strong> Pittsburgh School <strong>of</strong><br />
Medicine<br />
Pittsburgh, PA<br />
Stephen Gottschalk, MD<br />
Baylor College <strong>of</strong> Medicine<br />
Houston, TX<br />
John T. Gray, PhD<br />
St. Jude Children’s Research Hospital<br />
Memphis, TN<br />
Uta Griesenbach, PhD<br />
Imperial College Faculty <strong>of</strong> Medicine<br />
London, United Kingdom<br />
Dirk Grimm, PhD<br />
University <strong>of</strong> Heidelberg<br />
Heidelberg, Germany<br />
Markus Grompe, MD<br />
Oregon Health & Science University<br />
Portland, OR<br />
Farshid Guilak, PhD<br />
Duke University Medical Center<br />
Durham, NC<br />
Roger J. Hajjar, MD<br />
Mount Sinai School <strong>of</strong> Medicine<br />
New York, NY<br />
Joshua Hare, MD<br />
University <strong>of</strong> Miami Health System<br />
Miami, FL<br />
Scott Q. Harper, PhD<br />
Ohio State University & Nationwide<br />
Children’s Hospital<br />
Columbus, OH<br />
Richard Heller, PhD<br />
Old Dominion University<br />
Norfolk, VA<br />
Akseli Hemminki, MD, PhD<br />
University <strong>of</strong> Helsinki<br />
Helsinki, Finland<br />
Wim E. Hennink, PhD<br />
Utrecht Institute for Pharmaceutical<br />
Sciences<br />
Utrecht, Netherlands<br />
Roland W. Herzog, PhD<br />
University <strong>of</strong> Florida<br />
Gainesville, FL<br />
Helen E. Heslop, MD<br />
Baylor College <strong>of</strong> Medicine<br />
Houston, TX<br />
Matthew Hirsch, PhD<br />
University <strong>of</strong> North Carolina at<br />
Chapel Hill<br />
Chapel Hill, NC<br />
Jeffrey R. Holt, Ph.D.<br />
Boston Children’s Hospital<br />
Harvard Medical School<br />
Boston, MA<br />
Artur Isaev, PhD<br />
Human Stem <strong>Cell</strong>s Institute<br />
Moscow, Russia<br />
Brian Johnstone, PhD<br />
Oregon Health and Science University<br />
Portland, OR<br />
J. Keith Joung, MD, PhD<br />
Massachusetts <strong>Gene</strong>ral Hospital<br />
Charlestown, MA<br />
Carl H. June, MD<br />
University <strong>of</strong> Pennsylvania<br />
Philadelphia, PA<br />
Tal Kafri, MD, PhD<br />
University <strong>of</strong> North Carolina at<br />
Chapel Hill<br />
Chapel Hill, NC<br />
Noriyuki Kasahara, MD, PhD<br />
University <strong>of</strong> California, Los Angeles<br />
Los Angeles, CA<br />
Brian K. Kaspar, PhD<br />
The Research Institute at Nationwide<br />
Children’s Hospital<br />
Columbus, OH<br />
Kazunori Kataoka, PhD<br />
University <strong>of</strong> Tokyo<br />
Bunkyo-Ku, Japan<br />
Mark A. Kay, MD, PhD<br />
Stanford University School <strong>of</strong> Medicine<br />
Stanford, CA<br />
Hans-Peter Kiem, MD, FACP<br />
Fred Hutchinson Cancer Research<br />
Center<br />
Seattle, WA<br />
Sung Wan Kim, PhD<br />
University <strong>of</strong> Utah<br />
Salt Lake City, UT<br />
David Kirn, MD<br />
Jennerex, Inc.<br />
San Francisco, CA<br />
Thomas Knotts, PhD<br />
Brigham Young University<br />
Provo, UT<br />
Donald B. Kohn, MD<br />
University <strong>of</strong> California, Los Angeles<br />
Los Angeles, CA<br />
Peter Kolchinsky<br />
RA Capital<br />
Boston, MA<br />
Janaiah Kota, PhD<br />
Indiana University School <strong>of</strong> Medicine<br />
Indianapolis, IN<br />
Karen Kozarsky, PhD<br />
ReGenX Biosciences, LLC<br />
Washington, DC<br />
H. Teresa Ku, PhD<br />
City <strong>of</strong> Hope<br />
Duarte, CA<br />
Rajendra Kumar-Singh, PhD<br />
Tufts University School <strong>of</strong> Medicine<br />
Boston, MA<br />
Young Jik Kwon, PhD<br />
University <strong>of</strong> California, Irvine<br />
Irvine, CA<br />
King F. Kwong, MD<br />
National Institutes <strong>of</strong> Health<br />
National Cancer Institute<br />
Bethesda, MD<br />
Michael A. Laflamme, MD, PhD<br />
University <strong>of</strong> Washington<br />
Seattle, WA<br />
Jane S. Lebkowski, PhD<br />
Menlo Park, CA<br />
Brendan Lee, MD, PhD<br />
Baylor College <strong>of</strong> Medicine<br />
Howard Hughes Medical Institute<br />
Houston, TX<br />
18<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013
Faculty List<br />
Ann M. Leen, PhD<br />
Baylor College <strong>of</strong> Medicine<br />
Houston, TX<br />
Nick Leschly<br />
bluebird bio<br />
Cambridge, MA<br />
Bruce L. Levine, PhD<br />
University <strong>of</strong> Pennsylvania<br />
Philadelphia, PA<br />
Shulin Li, PhD<br />
MD Anderson Cancer Center<br />
Houston, TX<br />
<strong>Gene</strong> Liau, PhD<br />
Pizer WRD<br />
Cambridge, MA<br />
Judy Lieberman, MD, PhD<br />
Harvard Medical School<br />
Boston, MA<br />
Maria P. Limberis, PhD<br />
University <strong>of</strong> Pennsylvania<br />
Philadelphia, PA<br />
Gerald S. Lipshutz, MD<br />
University <strong>of</strong> California, Los Angeles<br />
Los Angeles, CA<br />
Cecilia Lo, PhD<br />
University <strong>of</strong> Pittsburgh<br />
Pittsburgh, PA<br />
Shan Lu, MD, PhD<br />
University <strong>of</strong> Massachusetts Medical<br />
School<br />
Worcester , MA<br />
Katarina Luptakova, MD<br />
Beth Israel Deaconess Medical Center<br />
Boston, MA<br />
Lawrence Lustig, MD<br />
University <strong>of</strong> California, San Francisco<br />
San Francisco, CA<br />
Ian MacLachlan, PhD<br />
Tekmira Pharmaceuticals Corp.<br />
Burnaby, Canada<br />
Casey A. Maguire, PhD<br />
Massachusetts <strong>Gene</strong>ral Hospital<br />
Charlestown, MA<br />
Ram I. Mahato, PhD<br />
University <strong>of</strong> Tennessee Health Science<br />
Center<br />
Memphis, TN<br />
Harry L. Malech, MD<br />
National Institutes <strong>of</strong> Health<br />
National Institute <strong>of</strong> Allergy and<br />
Infectious Diseases<br />
Bethesda, MD<br />
Punam Malik, MD, PhD<br />
Cincinnati Children’s Hospital Medical<br />
Center<br />
Cincinnati, OH<br />
Paris Margaritis, DPhil<br />
Children’s Hospital <strong>of</strong> Philadelphia<br />
Philadelphia, PA<br />
Jeffrey R. Martens, PhD<br />
University <strong>of</strong> Michigan<br />
Ann Arbor, MI<br />
Fulvio Mavilio, PhD<br />
Généthon<br />
Evry, France<br />
Jodi L. McBride, PhD<br />
Oregon National Primate Research<br />
Center<br />
Beaverton, OR<br />
Cheryl L. McDonald, MD<br />
National Institutes <strong>of</strong> Health<br />
National Heart, Lung, and Blood<br />
Institute<br />
Germantown, MD<br />
R. Scott McIvor, PhD<br />
University <strong>of</strong> Minnesota<br />
Minneapolis, MN<br />
Olivia Merkel, PhD<br />
Wayne State University<br />
Detroit, MI<br />
Anne Messer, PhD<br />
David Axelrod Institute/Wadsworth<br />
Center<br />
Albany, NY<br />
Carol H. Miao, PhD<br />
University <strong>of</strong> Washington<br />
Seattle, WA<br />
Jeffrey S. Miller, MD<br />
University <strong>of</strong> Minnesota<br />
Minneapolis, MN<br />
Shondra M. Miller, PhD<br />
Washington University<br />
St. Louis, MO<br />
Federico Mingozzi, PhD<br />
University Pierre Et Marie Curie-Paris 6<br />
<strong>Gene</strong>thon<br />
Paris, France<br />
Elizabeth A. Mittendorf, MD, PhD<br />
The University <strong>of</strong> Texas MD Anderson<br />
Cancer Center<br />
Houston, TX<br />
Kevin V. Morris, PhD<br />
Scripps Research Institute<br />
La Jolla, CA<br />
Christian Mueller, PhD<br />
University <strong>of</strong> Massachusetts Medical<br />
School<br />
Worcester, MA<br />
Thomas H. Murray, PhD<br />
The Hastings Center<br />
Garrison, NY<br />
Muna I. Naash, PhD<br />
University <strong>of</strong> Oklahoma Health Sciences<br />
Center<br />
Oklahoma City, OK<br />
Hiroyuki Nakai, MD, PhD<br />
Oregon Health & Science University<br />
School <strong>of</strong> Medicine<br />
Portland, OR<br />
Luigi M. Naldini, MD, PhD<br />
University Vita-Salute San Raffaele<br />
Milan, Italy<br />
Amit Nathwani, MD, PhD<br />
University College London Cancer<br />
Institute<br />
London, United Kingdom<br />
Yoichi Negishi, PhD<br />
Tokyo University <strong>of</strong> Pharmacy and Life<br />
Sciences<br />
Hachioji, Japan<br />
John J. Nemunaitis, MD<br />
Mary Crowley Cancer Research Centers<br />
Dallas, TX<br />
Philip Ng, PhD<br />
Baylor College <strong>of</strong> Medicine<br />
Houston, TX<br />
Arthur W. Nienhuis, MD<br />
St. Jude Children’s Research Hospital<br />
Memphis, TN<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013<br />
19
Faculty List<br />
Alan J. Nixon, DVM, MS<br />
Cornell University<br />
Ithaca, NY<br />
Ryan M. O’Connell, PhD<br />
University <strong>of</strong> Utah<br />
Salt Lake City, UT<br />
Maria C. Palanca-Wessels, MD, PhD<br />
Fred Hutchinson Cancer Research<br />
Center<br />
Seattle, WA<br />
Eirini P. Papapetrou, MD, PhD<br />
University <strong>of</strong> Washington<br />
Seattle, WA<br />
David W. Parsons, PhD<br />
Women’s & Children’s Hospital<br />
Adelaide, Australia<br />
Michael J. Passineau, PhD<br />
West Penn Allegheny Health System<br />
Pittsburgh, PA<br />
Marc Penn, MD, PhD<br />
Summa Health System<br />
Northeast Ohio Medical University<br />
Akron, OH<br />
Rita Perlingeiro, PhD<br />
University <strong>of</strong> Minnesota<br />
Minneapolis, MN<br />
Harold Petry, PhD<br />
UniQure, B.V.<br />
Amsterdam, Netherlands<br />
Eric A. Pierce, MD, PhD<br />
Massachusetts Eye and Ear<br />
Boston, MA<br />
Eric Poeschla, MD<br />
Mayo Clinic<br />
Rochester, MN<br />
Katherine P. Ponder, MD<br />
Washington University School <strong>of</strong><br />
Medicine<br />
St. Louis, MO<br />
David L. Porter, MD<br />
Hospital <strong>of</strong> the University <strong>of</strong><br />
Pennsylvania<br />
Philadelphia, PA<br />
Matthew H. Porteus, MD, PhD<br />
Stanford University School <strong>of</strong> Medicine<br />
Stanford, CA<br />
Pankaj Qasba, PhD<br />
National Institute <strong>of</strong> Health<br />
National Heart Lung and Blood<br />
Institute<br />
Bethesda, MD<br />
Joseph E. Rabinowitz, PhD<br />
Temple University School <strong>of</strong> Medicine<br />
Philadelphia, PA<br />
Daniel Rader, MD<br />
University <strong>of</strong> Pennsylvania Medical<br />
Center<br />
Philadelphia, PA<br />
Nicholas Restifo, MD<br />
National Institutes <strong>of</strong> Health<br />
National Cancer Institute<br />
Bethesda, MD<br />
Tonia S. Rex, PhD<br />
Vanderbilt University<br />
Nashville, TN<br />
Kevin G. Rice, PhD<br />
University <strong>of</strong> Iowa College <strong>of</strong> Pharmacy<br />
Iowa City, IA<br />
Isabelle Riviere, PhD<br />
Memorial Sloan-Kettering Cancer<br />
Center<br />
New York, NY<br />
Marcelo N. Rivolta, MD, PhD<br />
The University <strong>of</strong> Shefield<br />
Shefield, United Kingdom<br />
Scott H. Robbins, PhD<br />
Immune Design Corp.<br />
Seattle, WA<br />
Louise Rodino-Klapac, PhD<br />
Research Institute at Nationwide<br />
Children’s Hospital<br />
Columbus, OH<br />
Maria-Grazia Roncarolo, MD, PhD<br />
San Raffaele Scientiic Institute<br />
Milano, Italy<br />
Stephen M. Rose, PhD<br />
Foundation Fighting Blindness<br />
Columbia, MD<br />
Steven M. Rowe, MD<br />
University <strong>of</strong> Alabama at Birmingham<br />
Birmingham, AL<br />
David W. Russell, MD, PhD<br />
University <strong>of</strong> Washington<br />
Seattle, WA<br />
Stephen J. Russell, MD, PhD<br />
Mayo Clinic<br />
Rochester, MN<br />
Denise Sabatino, PhD<br />
The Children’s Hospital <strong>of</strong> Philadelphia<br />
Philadelphia, PA<br />
Aliasger K. Salem, PhD<br />
University <strong>of</strong> Iowa<br />
Iowa City, IA<br />
Robert A. Sandhaus, MD, PhD<br />
Alpha-1 Foundation<br />
Miami, FL<br />
David V. Schaffer, PhD<br />
University <strong>of</strong> California Berkeley<br />
Berkeley, CA<br />
Andrew M. Scharenberg, MD<br />
Seattle Children’s Research Institute<br />
Seattle, WA<br />
Daniel Scherman, PhD<br />
INSERM U1022 - CNRS UMR8151<br />
- ParisDescares University<br />
- ChimieParisTech<br />
Paris, France<br />
Manfred Schmidt, PhD<br />
Deutsches Krebsforschungszentrum<br />
Heidelberg<br />
National Center for Tumor Diseases<br />
Heidelberg, Germany<br />
Miguel Sena-Esteves, PhD<br />
University <strong>of</strong> Massachusetts Medical<br />
School<br />
Worcester, MA<br />
Susan B. Sepelak, MS, MSM<br />
Social and Scientiic Systems, Inc<br />
Silver Spring, MD<br />
Prem Seth, PhD<br />
NorthShore University HealthSystem<br />
Evanston, IL<br />
Dmitry M. Shayakhmetov, PhD<br />
University <strong>of</strong> Washington<br />
Seattle, WA<br />
Sonia I. Skarlatos, PhD<br />
National Institutes <strong>of</strong> Health<br />
National Heart, Lung, Blood Institute<br />
Bethesda, MD<br />
20<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013
Faculty List<br />
Evan Y. Snyder, MD PhD FAAP<br />
Sanford-Burnham Medical Research<br />
Institute<br />
La Jolla, CA<br />
Sihong Song, PhD<br />
University <strong>of</strong> Florida<br />
Gainesville, FL<br />
David M. Spencer, PhD<br />
Bellicum Pharmaceuticals, Inc.<br />
Houston, TX<br />
H. Trent Spencer, PhD<br />
Emory University School <strong>of</strong> Medicine<br />
Atlanta, GA<br />
Gary K. Steinberg, MD, PhD<br />
Stanford School <strong>of</strong> Medicine<br />
Stanford, CA<br />
Renata Stripecke, PhD<br />
Hannover Medical School<br />
Hannover, Germany<br />
Stephen C. Strom, PhD<br />
Karolinska Institutet<br />
Stockholm, Sweden<br />
Suzannah Sundby<br />
Smith, Gambrell & Russell, LLP<br />
Washington DC<br />
Kenzaburo Tani, MD, PhD<br />
Kyushu University<br />
Higashi-ku, Japan<br />
Doris A. Taylor, PhD<br />
Texas Heart Institute at St. Luke’s<br />
Episcopal Hospital<br />
Houston, TX<br />
David H. Thompson, PhD<br />
Purdue University<br />
West Lafayette, IN<br />
John F. Tisdale, MD<br />
National Institutes <strong>of</strong> Health<br />
National Heart, Lung, and Blood<br />
Institute<br />
Bethesda, MD<br />
Jacques P. Tremblay, PhD<br />
Centre de recherche du Centre<br />
hospitalier de l’Université Laval<br />
Sainte-Foy, Canada<br />
Grant D. Trobridge, PhD<br />
Washington State University<br />
Pullman, WA<br />
Rocky S. Tuan, PhD<br />
McGowan Institute for Regenerative<br />
Medicine<br />
Pittsburgh, PA<br />
Evan C. Unger, MD<br />
University <strong>of</strong> Arizona Cancer Center<br />
Tucson, AZ<br />
Ludovic Vallier, PhD<br />
Anne McLaren Laboratory for<br />
Regenerative Medicine<br />
Cambridge, United Kingdom<br />
Sander van Deventer, MD, PhD<br />
Forbion Capital Partners<br />
Naarden, Netherlands<br />
Luk H. Vandenberghe, PhD<br />
Harvard Medical School<br />
Boston, MA<br />
Thierry C. VandenDriessche, PhD<br />
University <strong>of</strong> Brussels<br />
Leuven, Belgium<br />
Juan F. Vera, MD<br />
Baylor College <strong>of</strong> Medicine<br />
Houston, TX<br />
Gabor Veres, PhD<br />
bluebird bio<br />
Cambridge, MA<br />
Richard G. Vile, PhD<br />
Mayo Clinic<br />
Rochester, MN<br />
Samuel C. Wadsworth, PhD<br />
Genzyme Corporation - a Sanoi<br />
Company<br />
Framingham, MA<br />
Kathryn R. Wagner, MD, PhD<br />
The Kennedy Krieger Institute<br />
The Johns Hopkins School <strong>of</strong> Medicine<br />
Baltimore, MD<br />
Claes Wahlestedt, MD, PhD<br />
University <strong>of</strong> Miami Miller School <strong>of</strong><br />
Medicine<br />
Jupiter, FL<br />
Bing Wang, MD, PhD<br />
University <strong>of</strong> Pittsburgh School <strong>of</strong><br />
Medicine<br />
Pittsburgh, PA<br />
Sue Washer, MBA<br />
Applied <strong>Gene</strong>tics Technologies,<br />
Corporation<br />
Alachua, FL<br />
Yu-Quan Wei, MD, PhD<br />
State Key Laboratory <strong>of</strong> Biotherapy<br />
Chengdu, Peoples Republic <strong>of</strong> China<br />
Marco S. Weinberg, PhD<br />
The Scripps Research Institute<br />
Johannesburg, South Africa<br />
Andy Wilber, PhD<br />
Southern Illinois University School <strong>of</strong><br />
Medicine<br />
Springield, IL<br />
James M. Wilson, MD, PhD<br />
University <strong>of</strong> Pennsylvania Perelman<br />
School <strong>of</strong> Medicine<br />
Philadelphia, PA<br />
Savio L.C. Woo, PhD<br />
Mount Sinai School <strong>of</strong> Medicine<br />
New York, NY<br />
J. Fraser Wright, PhD<br />
Children’s Hospital <strong>of</strong> Philadelphia<br />
University <strong>of</strong> Pennsylvania<br />
Philadelphia, PA<br />
Xiao Xiao, PhD<br />
University <strong>of</strong> North Carolina at Chapel<br />
Hill<br />
Chapel Hill, NC<br />
James Yockman, PhD<br />
University <strong>of</strong> Utah<br />
Salt Lake City, UT<br />
Michael Young, PhD<br />
Schepens Eye Research Institute<br />
Boston, MA<br />
Chae-Ok Yun, PhD<br />
Hanyang University<br />
Seoul, Korea<br />
Assem Ziady<br />
Emory University<br />
Atlanta, GA<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013<br />
21
<strong>Gene</strong>ral Meeting Information<br />
AMERICAN SOCIETY OF GENE & CELL THERAPY<br />
Mission and Vision<br />
ASGCT’s mission is to advance knowledge, awareness, and<br />
education leading to the discovery and clinical application <strong>of</strong><br />
genetic and cellular therapies to alleviate human disease.<br />
ASGCT’s strategic vision is to be a catalyst for bringing<br />
together scientists, physicians, patient advocates, and other<br />
stakeholders to transform the practice <strong>of</strong> medicine by<br />
incorporating the use <strong>of</strong> genetic and cellular therapies to<br />
control and cure human disease.<br />
Abstract Volume - Molecular <strong>Therapy</strong><br />
All abstracts, except Late Abstract Submissions, accepted for<br />
presentation at the ASGCT 16th Annual Meeting have been<br />
published in the May supplement <strong>of</strong> Molecular <strong>Therapy</strong>.<br />
Attendees will receive one copy <strong>of</strong> the supplement along<br />
with their registration materials. Late Abstract Submissions<br />
accepted for presentation have been printed in a booklet<br />
that will be distributed to each attendee. In addition, the<br />
Late Abstract Submissions will be published in the July 2013<br />
online issue <strong>of</strong> Molecular <strong>Therapy</strong>.<br />
Admission<br />
Oficial name badges will be required for admission to all<br />
ASGCT sessions. All Annual Meeting attendees receive a<br />
name badge with their registration bag. Name badges should<br />
be worn at all times inside the convention center, as badges<br />
will be used to control access to sessions and activities.<br />
Attendees are cautioned against wearing their name badges<br />
while away from the convention center as badges may draw<br />
unwanted attention to your status as visitors to Salt Lake<br />
City, Utah.<br />
Admission by Ticket Only<br />
Some sessions at the Annual Meeting will require an<br />
additional ticket to control access and capacity. The Meetthe-Investigator<br />
sessions scheduled at the 16th Annual<br />
Meeting require tickets for admittance, which are not<br />
included in the regular registration fees. These sessions<br />
are designed to provide an opportunity for participants<br />
to interact with experts and discuss topics related to their<br />
research. Sessions will begin promptly and a lunch will be<br />
served during the program, which will last for approximately<br />
one hour. Each ticket is $25.<br />
Please Note: Tickets for the Meet-the-Investigator sessions<br />
are limited to 30 participants and will be sold on a irstcome,<br />
irst-serve basis. You must have a ticket to gain<br />
entrance to a session. Ushers will be collecting tickets at<br />
the door <strong>of</strong> each session. Tickets may be obtained at the<br />
Registration Desk.<br />
Boxed Lunches<br />
Pre-ordered boxed lunches will be available for pick-up<br />
at the registration desk from 12:00 pm – 12:30 pm on<br />
Wednesday, 12:30 pm – 1:00 pm on Thursday, 11:45 am –<br />
12:15 pm on Friday, and 12:15 pm – 12:45 pm on Satur day.<br />
All boxed lunches must have been pre-ordered.<br />
Business Center<br />
There is a Business Center located in the Salt Palace<br />
Convention Center on the Second Level Upper Concourse,<br />
near Room 252. The Business Center is open from<br />
8:00 am – 5:00 pm. Phone: (385) 468-2228.<br />
Business Meeting<br />
Join the <strong>Society</strong>’s leadership for a business meeting and<br />
continental breakfast.<br />
Saturday, May 18, 2013<br />
7:30 am – 8:00 am<br />
A light continental breakfast will be <strong>of</strong>fered at 7:15 am<br />
Room 150 ABC, Level 1.<br />
All members are welcome. See page 6 for more details.<br />
Camera/Recording Policy<br />
It is the policy <strong>of</strong> ASGCT that no cameras are permitted in<br />
the meeting sessions, exhibit hall, or poster sessions, with<br />
the exception <strong>of</strong> the <strong>of</strong>icial ASGCT photographer assigned<br />
by ASGCT to capture images for use in <strong>Society</strong> archival and<br />
promotional materials. Please refrain from taking any photos<br />
in those locations. Audio and/or videotaping is strictly<br />
prohibited.<br />
Your attendance at ASGCT events implies your permission<br />
for images captured during these events to be used for the<br />
purposes <strong>of</strong> ASGCT archival and promotional material and<br />
publications and waives your rights for compensation or<br />
ownership <strong>of</strong> these images.<br />
<strong>Cell</strong> Phones & PDAs<br />
For the courtesy <strong>of</strong> all presenters and attendees, please<br />
ensure that cell phone and PDA ringers are turned <strong>of</strong>f or<br />
silenced during all sessions as they may interfere with<br />
session audio.<br />
Committee Meetings<br />
Most standing and scientiic committees will meet during<br />
the Annual Meeting. See page 6 for Committee Meeting<br />
information.<br />
Concierge Desk<br />
Salt Lake City <strong>of</strong>fers a wide variety <strong>of</strong> activities and<br />
restaurants to meet all tastes and budgets. A Concierge Desk<br />
will be located outside the Exhibit Hall <strong>of</strong> the Salt Palace<br />
Convention Center, staffed by the Salt Lake Convention &<br />
Visitors Bureau, who can help you with your city needs and<br />
reservations.<br />
Concierge Desk Hours<br />
Wednesday through Saturday 10:00 am – 6:00 pm<br />
22<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013
<strong>Gene</strong>ral Meeting Information<br />
Continuing Medical Education<br />
The ASGCT Board <strong>of</strong> Directors decided that the <strong>Society</strong> will<br />
not <strong>of</strong>fer CME credit for the 16th Annual Meeting due to<br />
rising costs.<br />
Disclosure<br />
In keeping with good practices, ASGCT requires faculty,<br />
planning committee and oral abstract presenters to disclose<br />
any relevant inancial interest or other relationship with<br />
the manufacturer(s) <strong>of</strong> any commercial product(s) and/or<br />
provider(s) <strong>of</strong> commercial services that are discussed in this<br />
educational activity.<br />
Educational Methods and Materials<br />
Lectures, Case Presentations, Panel Discussions, Question<br />
and Answer Sessions, Audio/Video Presentations, Abstracts,<br />
Posters<br />
Educational Objectives<br />
At the conclusion <strong>of</strong> the activity, the participant should be<br />
able to:<br />
• Provide advice to patients who inquire about the<br />
potential <strong>of</strong> gene and cell therapy or the availability <strong>of</strong><br />
open clinical trials, based on their exposure to the current<br />
clinical trials in gene and cell therapies.<br />
• Better instruct their students in medical school and other<br />
health venues using the state-<strong>of</strong>-the-art basic science and<br />
clinical trials data presented at the meeting.<br />
• Use the latest advances in gene and cell therapy to<br />
enhance their research mission, as physician scientists<br />
conducting basic and clinical research.<br />
• Demonstrate improved regulatory compliance in<br />
conducting gene and cell therapy clinical trials, through<br />
exposure to NIH and FDA faculty during the educational<br />
program.<br />
Evaluation Method<br />
Evaluation by questionnaire will address program content,<br />
presentation, and possible bias.<br />
Needs<br />
Clinical gene transfer has become increasingly complex<br />
due to ongoing developments in the ields <strong>of</strong> gene and cell<br />
therapy itself, together with bioethics, research integrity, and<br />
inancial conlicts, as well as federal mandates, regulations<br />
and guidelines. Oligonucleotide Therapies, Novel Vector<br />
Development, Host-Vector Interactions and Vaccine<br />
Therapies will be discussed as well as many other scientiic<br />
topics. This meeting will provide an educational forum for<br />
scientists and clinicians to expand their knowledge about the<br />
broad developments in these ields.<br />
Target Audience<br />
The target audience includes basic science and translational<br />
researchers, clinical investigators, physicians, postdoctoral<br />
fellows, graduate students, employees <strong>of</strong> federal government<br />
and regulatory agencies, and other healthcare pr<strong>of</strong>essionals<br />
with an interest in the latest advancements in the ield <strong>of</strong><br />
gene and cell therapy.<br />
Dates<br />
The ASGCT 16th Annual Meeting will begin on the morning<br />
<strong>of</strong> Wednesday, May 15 and continue through the evening <strong>of</strong><br />
Saturday, May 18, 2013. Exhibits will be open Wednesday,<br />
May 15 through Friday, May 17.<br />
Exhibits<br />
Exhibits will be located in Exhibit Hall C/D <strong>of</strong> the Salt Palace<br />
Convention Center. The companies, organizations and<br />
institutions exhibiting at the ASGCT Annual Meeting provide<br />
the latest information on products and services directly<br />
related to your pr<strong>of</strong>essional needs as well as recruit for open<br />
positions. An Exhibit Hall Social will be held on Wednesday<br />
evening and a c<strong>of</strong>fee social will be <strong>of</strong>fered on Thursday<br />
and Friday in the exhibit hall. All participants are urged to<br />
allow adequate time daily to visit the exhibits, as they are an<br />
integral part <strong>of</strong> the success <strong>of</strong> the meeting.<br />
Exhibit Hall Hours<br />
Exhibits will be open Wednesday, May 15 through Friday,<br />
May 17, 2013.<br />
Wednesday, May 15 5:15 pm – 7:15 pm<br />
Thursday, May 16 9:35 am – 10:30 am<br />
Thursday, May 16 4:00 pm – 6:00 pm<br />
Friday, May 17<br />
9:00 am – 9:45 am<br />
Friday, May 17<br />
5:30 pm – 7:30 pm<br />
Guest Attendance<br />
ASGCT asks registered attendees to refrain from taking<br />
children, spouses, or guests to any educational session or<br />
functions <strong>of</strong>fered at the 16th Annual Meeting.<br />
Internet Access & Policy<br />
Internet access will be available in public areas at the Salt<br />
Palace Convention Center to all meeting attendees. Limited<br />
computer stations will be provided in Exhibit Hall C/D.<br />
When others are waiting at the internet kiosks, please limit<br />
your Internet use to 15 minutes at a time so other attendees<br />
can access the internet as well.<br />
Job Bank<br />
At the 16th Annual Meeting, there will be a forum for<br />
academic institutions and companies to promote open<br />
positions to meeting attendees. If you have open positions,<br />
please bring multiple copies <strong>of</strong> your job posting to the<br />
meeting. Similarly, if you are seeking a job, please bring<br />
multiple copies <strong>of</strong> your résumé to submit. ASGCT will<br />
not take responsibility for résumés sent to the ASGCT<br />
<strong>of</strong>ice in response to a job posting. If you would like more<br />
information about this service, please contact Shaun Kramer<br />
at skramer@asgct.org. The Job exchange area will be located<br />
inside the Exhibit Hall.<br />
Lead Retrieval<br />
A lead retrieval system has been made available to all<br />
exhibitors <strong>of</strong> the ASGCT 16th Annual Meeting. Exhibitors<br />
may ask to scan attendee name badges with a hand held<br />
scanner in order to obtain attendee contact information.<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013<br />
23
<strong>Gene</strong>ral Meeting Information<br />
Location<br />
• The 16th Annual Meeting will be held at the Salt Palace<br />
Convention Center , 100 SW Temple, Salt Lake City, UT<br />
84101, USA<br />
• Exhibits and Posters will take place in Hall C/D.<br />
• Registration will be located on the East Registration Area<br />
– by the East Entrance on the 2nd loor <strong>of</strong> the Salt Palace<br />
Convention Center<br />
Materials Distribution<br />
Please refrain from distributing promotional materials<br />
throughout the meeting; these may only be distributed from<br />
an exhibit booth in Exhibit Hall C/D.<br />
No Smoking<br />
Smoking is prohibited at all 16th Annual Meeting sessions<br />
and events.<br />
Posters<br />
Abstract Posters will be on display in Exhibit Hall C/D.<br />
Thursday, May 16<br />
Abstract Poster Session I<br />
7:00 am – 12:30 pm Poster Setup by Authors<br />
12:30 pm – 6:00 pm Poster Viewing (authors present<br />
from 4:00 pm – 6:00 pm)<br />
4:00 pm – 6:00 pm Networking Reception, Exhibits, and<br />
Poster Session I<br />
6:00 pm – 6:30 pm Authors Remove Posters<br />
Friday, May 17<br />
Abstract Poster Session II<br />
7:00 am – 11:45 am Poster Setup by Authors<br />
11:45 am – 7:30 pm Poster Viewing (authors present<br />
from 5:30 pm – 7:30 pm)<br />
5:30 pm – 7:30 pm Networking Reception, Exhibits, and<br />
Poster Session II<br />
7:30 pm – 8:00 pm Authors Remove Posters<br />
Saturday, May 18<br />
Abstract Poster Session III<br />
7:00 am – 12:15 pm Poster Setup by Authors<br />
12:15 pm – 5:45 pm Poster Viewing (authors present<br />
from 3:45 pm – 5:45 pm)<br />
3:45 pm – 5:45 pm Poster Session III<br />
5:45 pm – 6:15 pm Authors Remove Posters<br />
Call4Posters ® Pick-up Schedule<br />
Once again this year the ASGCT arranged with Marathon<br />
Multimedia to provide poster authors with the opportunity<br />
to create their poster online using the Call4Posters® service.<br />
Poster presenters who took advantage <strong>of</strong> this service may<br />
pick-up their pre-ordered poster at the ASGCT Registration<br />
Desk during the following hours.<br />
Tuesday, May 14 5:00 pm – 7:00 pm<br />
Wednesday, May 15 7:00 am – 7:30 pm<br />
Thursday, May 16 7:00 am – 6:30 pm<br />
Friday, May 17 7:30 am – 7:30 pm<br />
Saturday, May 18 7:30 am – 4:00 pm<br />
Press<br />
Members <strong>of</strong> the working media may register for the 16th<br />
Annual Meeting in the Press Room – Room 252 AB. Interview<br />
space, computers and internet services are available for the<br />
convenience <strong>of</strong> media representatives covering the meeting.<br />
Press must register, provide credentials, and wear their<br />
press badge for admittance to ASGCT sessions. Assistance<br />
will be provided to members <strong>of</strong> the media that would like to<br />
schedule interviews.<br />
Press Room<br />
The Press Room is located in the Salt Palace Convention<br />
Center, 252 AB.<br />
Press Room Hours<br />
Wednesday, May 15 5:15 pm – 7:15 pm<br />
Thursday, May 16 4:00 pm – 6:00 pm<br />
Friday, May 17 5:30 pm – 7:30 pm<br />
Registration Desk<br />
Name badges, inal programs, and abstract supplements will<br />
be distributed at the registration desk located in the East<br />
Registration Area (Level 2) <strong>of</strong> the Salt Palace Convention<br />
Center, just inside the East Entrance.<br />
Registration Desk Hours<br />
Tuesday, May 14 5:00 pm – 7:00 pm<br />
Wednesday, May 15 7:00 am – 7:30 pm<br />
Thursday, May 16 7:00 am – 6:30 pm<br />
Friday, May 17 7:30 am – 7:30 pm<br />
Saturday, May 18 7:30 am – 4:00 pm<br />
Cancellation Refund Policy<br />
Refund requests should have been submitted in writing to<br />
the ASGCT Executive Ofice prior to May 3, 2013.<br />
Salt Lake City Information<br />
Please abide by the trafic signs and lights and utilize the<br />
designated crosswalks at all times. See Salt Lake Fun Facts<br />
on the next page for more information on Salt Lake City.<br />
Shuttle Bus<br />
ASGCT will not be providing shuttle transportation for the<br />
16th Annual Meeting.<br />
24<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013
<strong>Gene</strong>ral Meeting Information<br />
Speaker Ready Room<br />
There will be a Speaker Ready Room located in Room 250 A<br />
on the second level <strong>of</strong> the Salt Palace Convention Center, near<br />
the ASGCT Registration Desk.<br />
All speakers, including oral abstract presenters, must deliver<br />
their presentations to the Speaker Ready Room the day<br />
before their session or at least four hours prior to their<br />
presentations. Equipment is available for faculty to review<br />
their materials. Audiovisual personnel will be available for<br />
assistance. Please mark your materials (your name, session<br />
and speaker order) so the materials can be returned to<br />
you. The <strong>Society</strong> strongly encourages faculty to pre-load<br />
presentations in the Speaker Ready Room; those faculty who<br />
load presentations in the meeting rooms during the sessions<br />
will have that time deducted from their presentation time by<br />
the Chair.<br />
ASGCT strongly encourages faculty presenting on<br />
Wednesday <strong>of</strong> the Annual Meeting to check-in at the Speaker<br />
Ready Room on Tuesday, May 14 to avoid congestion.<br />
Avoid delays and check-in early!<br />
Speaker Ready Room Hours<br />
Tuesday, May 14 5:00 pm – 7:00 pm<br />
Wednesday, May 15 7:00 am – 7:30 pm<br />
Thursday, May 16 7:00 am – 6:30 pm<br />
Friday, May 17<br />
7:30 am – 7:30 pm<br />
Saturday, May 18 7:30 am – 4:00 pm<br />
Special Accessibility Needs<br />
If you require special accommodations under the ADA in<br />
order to fully participate in the meeting or the course, please<br />
visit the Registration Desk; ASGCT staff will be happy to<br />
assist you with your speciic needs.<br />
Salt Lake City & Utah “Fun Facts”<br />
• The State name Utah comes from the Native <strong>American</strong><br />
Ute Tribe meaning “people <strong>of</strong> the mountains”<br />
• The State nickname is the Beehive State – for the<br />
industriousness <strong>of</strong> the Utah Pioneer<br />
• Utah’s state motto is “Industry”<br />
• On December 3, 1933 Utah became the deciding US state<br />
to approve the repeal <strong>of</strong> prohibition<br />
• There are 21 National Parks within a day’s drive<br />
• The original Harman’s Kentucky Fried Chicken, the irst<br />
KFC franchise, still operates at 3900 South State Street<br />
• Utah is ranked #1 for happy people<br />
• Utah is ranked #1 for the best hiking State in the US<br />
• Utah is the number one per-capita consumer <strong>of</strong> Jell-O<br />
brand gelatin<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013<br />
25
Hotel Information and Area Map<br />
ASGCT 16 th Annual Meeting<br />
May 15-18, 2013<br />
1. Hilton Salt Lake City Center – HEADQUARTERS HOTEL<br />
2. Salt Lake Marriott Downtown at City Creek<br />
3. Sheraton Salt Lake City Hotel<br />
26<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013
Salt Palace Convention Center Floor Plan<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013<br />
27
Salt Palace Convention Center Floor Plan<br />
First Level — Exhibit Hall C/D and Meeting Rooms<br />
• Exhibits<br />
• Posters<br />
• Job Exchange<br />
• Internet Kiosks<br />
First Level<br />
• Plenary Lectures<br />
• Session Rooms<br />
(Education, Oral Abstracts,<br />
Scientiic Symposia)<br />
28<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013
Salt Palace Convention Center Floor Plan<br />
Second Level<br />
• Registration<br />
• Speaker Ready Room<br />
• Meet-the-Investigators<br />
• Committee Meetings<br />
• Receptions<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013<br />
29
Schedule-at-a-Glance (<strong>Program</strong> subject to change)<br />
Tuesday, May 14, 2013 (Pre-Meeting)<br />
11:00 am – 6:00 pm Salt Lake City Fun Day Activities<br />
4:00 pm – 6:00 pm Public Education Events<br />
Wednesday, May 15, 2013<br />
7:30 am – 8:00 am C<strong>of</strong>fee Break<br />
8:00 am – 10:00 am Scientiic Symposia Session I<br />
10:30 am – 12:00 pm Education Session I<br />
12:00 pm – 1:15 pm Lunch Break (Lunch Not Provided)<br />
1:15 pm – 2:45 pm Education Session II<br />
3:15 pm – 5:15 pm Oral Abstract Session I<br />
5:15 pm – 7:15 pm Exhibit Hall Welcome Reception<br />
7:30 pm – 8:30 pm New Member Welcome Reception<br />
7:30 pm – 9:30 pm Commercialization Workshop<br />
Thursday, May 16, 2013<br />
8:30 am – 9:35 am George Stamatoyannopoulos Lecture<br />
9:35 am – 10:30 am Exhibit Hall C<strong>of</strong>fee Social<br />
10:30 am – 12:30 pm Scientiic Symposia Session II<br />
12:30 pm – 2:00 pm Lunch Break (Lunch Not Provided)<br />
Meet-the-Investigator Session & Offsite Tour<br />
2:00 pm – 4:00 pm Oral Abstract Session II<br />
4:00 pm – 6:00 pm Exhibit Hall Open: Networking Reception & Poster Session I<br />
6:00 pm – 7:00 pm Canadian Attendee Reception<br />
6:00 pm – 8:00 pm Alliance for Cancer <strong>Gene</strong> <strong>Therapy</strong> Foundation Symposium<br />
Muscular Dystrophy Association & Association-Francaise Contre Les Myopathies Foundation<br />
Symposium<br />
Friday, May 17, 2013<br />
8:00 am – 9:00 am Outstanding Achievement Award Lecture with<br />
Distinguished Service Award and Special Recognition Award Presentations<br />
9:00 am – 9:45 am Exhibit Hall C<strong>of</strong>fee Social<br />
9:45 am – 11:45 am Scientiic Symposia Session III<br />
11:45 am – 1:00 pm Lunch Break (Lunch Not Provided)<br />
1:00 pm – 3:00 pm Oral Abstract Session III<br />
3:15 pm – 5:30 pm Presidential Symposium Lecture & Top Abstracts<br />
5:30 pm – 7:30 pm Exhibit Hall Open: Networking Reception & Poster Session II<br />
7:30 pm – 9:30 pm Foundation Fighting Blindness Foundation Symposium<br />
International Consortium for <strong>Gene</strong> and <strong>Cell</strong> <strong>Therapy</strong> Workshop<br />
New Investigator Town Hall & Mentoring Reception<br />
Saturday, May 18, 2013<br />
7:30 am – 8:00 am ASGCT Business Meeting<br />
8:00 am – 10:00 am Outstanding New Investigator Symposium<br />
10:15 am – 12:15 pm Oral Abstract Session IV<br />
12:15 pm – 1:45 pm Lunch Break (Lunch Not Provided)<br />
Meet-the-Investigator Session & Offsite Tour<br />
1:45 pm – 3:45 pm Scientiic Symposia Session IV<br />
3:45 pm – 5:45 pm Poster Session III & Reception<br />
5:45 pm Annual Meeting Concludes<br />
30<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013
<strong>Program</strong> Schedule, Wednesday, May 15, 2013<br />
C<strong>of</strong>fee Break<br />
7:30 am – 8:00 am<br />
Scientific Symposium 100<br />
8:00 am - 10:00 am<br />
ROOM: BALLROOM D<br />
Challenges and Success <strong>of</strong> <strong>Gene</strong> <strong>Therapy</strong> Product Approval<br />
CO-CHAIRS: Dale G. Ando, MD and Barry J. Byrne, MD, PhD<br />
SPEAKERS<br />
Dale G. Ando, MD<br />
Clinical Studies <strong>of</strong> the Infusion <strong>of</strong> ZFN CCR5 Modiied Autologous CD4 T cells (SB-728T) in HIV Subjects<br />
CCR5 is the second receptor for HIV in R5 trophic HIV strains and the CCR5Δ32 gene mutation confers resistance to HIV infection in<br />
humans. This resistance to HIV infection can be recreated in CD4 T cells by ex-vivo modiication <strong>of</strong> the CCR5 gene locus by speciic<br />
Zinc Finger Nucleases Clinical studies treating HIV subjects with autologous CD4 T cells modiied at CCR5 by ZFN (SB-728T) in<br />
order to protect against HIV have been performed. Clinical data on the pharmacology, immunologic effects and safety <strong>of</strong> these<br />
treatments will be presented.<br />
Wednesday, May 15, 2013<br />
Theresa Chen, PhD<br />
CBER Regulatory/Preclinical Considerations for AAV Products<br />
The presentation will provide an overview <strong>of</strong> the current CBER perspective regarding the preclinical evaluation <strong>of</strong> AAV-based gene<br />
therapy products and discuss regulatory and scientiic challenges in the safety and activity assessment <strong>of</strong> these investigational<br />
products. According to 21 CFR 312.23 (a)(8), adequate information derived from pharmacology and toxicology studies is needed<br />
to support a clinical investigation that is reasonably safe and scientiically feasible. Thus, the data from preclinical studies should<br />
provide a better understanding <strong>of</strong> the activity, safety, vector biodistribution, and transgene expression proiles following product<br />
administration in appropriate animal models. These data will then be used to guide aspects <strong>of</strong> the design <strong>of</strong> early phase clinical<br />
trials <strong>of</strong> AAV-based products, including: 1) determination <strong>of</strong> a potentially safe starting dose level and dose escalation scheme; 2) an<br />
optimal route <strong>of</strong> administration/anatomic site <strong>of</strong> delivery; 3) subject eligibility criteria; and 4) an adequate clinical monitoring plan.<br />
Barry J. Byrne, MD, PhD<br />
Next <strong>Gene</strong>ration Therapies with AAV<br />
Evan Y. Snyder, MD PhD FAAP<br />
The Rationale Use <strong>of</strong> <strong>Cell</strong>-based Therapies - Viewpoint from a Scientist who Advises Regulatory Agencies<br />
Although it is true that regulatory agencies, when assessing applications for cell-based therapies, tend to focus primarily on issues<br />
<strong>of</strong> safety, they are inevitably cognizant <strong>of</strong> the rationale behind the use <strong>of</strong> particular cell type in a particular clinical context in order<br />
to determine whether the risk is justiied. In other words, they do pay attention to whether a presumed mechanism-<strong>of</strong>-action<br />
is scientiically valid and supportable by the data. Certainly the scientists and physicians who advise such regulatory agencies<br />
scrutinize such data. This talk will discuss both the rational and irrational use <strong>of</strong> cell therapy, particularly for the neurological<br />
disorders, including a number <strong>of</strong> misconceptions.<br />
Scientific Symposium 101<br />
8:00 am - 10:00 am<br />
ROOM: BALLROOM C<br />
From Blood to the Brain: <strong>Gene</strong> and <strong>Cell</strong> <strong>Therapy</strong> <strong>of</strong> <strong>Gene</strong>tic Diseases and Inborn Errors <strong>of</strong><br />
Metabolism<br />
CO-CHAIRS: Randy J. Chandler, PhD, MB and Denise Sabatino, PhD<br />
SPEAKERS<br />
Ian E. Alexander, MBBS, PhD<br />
Pre-Clinical <strong>Gene</strong> <strong>Therapy</strong> for Urea Cycle Disorders<br />
A critically important function <strong>of</strong> the liver is the detoxiication <strong>of</strong> ammonia via the urea cycle. Infants with severe urea cycle<br />
enzyme deiciencies commonly present early in life with life-threatening hyperammonemia, progressing to coma and death in<br />
the absence <strong>of</strong> medical intervention. Effective management remains challenging and necessitates liver transplantation. Ornithine<br />
transcarbamylase (OTC) deiciency, the most common urea cycle defect, has long been recognized as an ideal model for livertargeted<br />
gene therapy. This presentation will explore contemporary efforts to treat OTC and other urea cycle defects, placing these<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013<br />
31
<strong>Program</strong> Schedule, Wednesday, May 15, 2013<br />
Wednesday, May 15, 2013<br />
in historical context. Robust life-long phenotype correction has been achieved in adult mice after single dose treatment with AAVbased<br />
vectors, and the translational challenges <strong>of</strong> moving towards clinical trials involving pediatric patients are currently being<br />
addressed. These challenges include targeting the developing liver where hepatocellular proliferation leads to loss <strong>of</strong> episomal AAV<br />
vector genomes and, depending on the therapeutic context, the possible need for vector re-administration to maintain effective<br />
levels <strong>of</strong> transgene expression.<br />
Stephen C. Strom, PhD<br />
Placental Stem <strong>Cell</strong> Treatment <strong>of</strong> Liver Disease<br />
Epithelial cells isolated from human amnion (hAE) have surface marker and gene expression proiles <strong>of</strong> pluripotent stem cells.<br />
Our previous studies have shown that hAE mature liver genes when transplanted into the liver <strong>of</strong> mice. Accordingly hAE were<br />
transplanted into the liver <strong>of</strong> iMSUD –mice, a useful model <strong>of</strong> the Maple Syrup Urine Disease. While untreated animals died at<br />
weaning, treated animals survived and thrived on a normal protein diet out to adulthood and showed signiicant correction <strong>of</strong><br />
amino acid and neurotransmitter proiles. These results suggest that hAE cells could be a useful cellular therapy for metabolic liver<br />
diseases.<br />
Alessandra Bifi, MD<br />
Phase I/II Clinical Trial <strong>of</strong> Hematopoietic Stem <strong>Cell</strong> <strong>Gene</strong> <strong>Therapy</strong> for the Treatment <strong>of</strong> Metachromatic<br />
Leukodystrophy<br />
Amit Nathwani, MD, PhD<br />
AAV Mediated <strong>Gene</strong> Transfer for Hemophilia A<br />
We have developed distinct approach for gene therapy <strong>of</strong> haemophilia A using adeno-associated virus (AAV) vectors which is based<br />
on our successful strategy for gene therapy <strong>of</strong> haemophilia B. Data from our preclinical evaluation will be presented together with<br />
an outline <strong>of</strong> Phase I/II trial design for Haemophilia A.<br />
Scientific Symposium 102<br />
8:00 am - 10:00 am<br />
ROOM: BALLROOM B<br />
<strong>Gene</strong> and <strong>Cell</strong> Therapies for Auditory and Olfactory Disorders<br />
CO-CHAIRS: Jodi L. McBride, PhD and Anne Messer, PhD<br />
SPEAKERS<br />
Jeffrey R. Holt, PhD<br />
<strong>Gene</strong> <strong>Therapy</strong> Strategies to Restore Sensory Function in the Inner Ear<br />
Sensory transduction in hair cells <strong>of</strong> the inner ear is the fundamental irst step that converts auditory information into electrical<br />
signals which are transmitted to the brain. Loss <strong>of</strong> sensory function in inner ear hair cells is the principle deicit in over sixty<br />
forms <strong>of</strong> genetic deafness. To investigate gene therapy as a strategy for restoration <strong>of</strong> auditory function our lab is focused on viral<br />
mediated delivery <strong>of</strong> wild-type Tmc1, mutations in which cause deafness in humans and mice. Tmc1 is expressed in hair cells and<br />
preliminary data indicated that AAV re-introduction <strong>of</strong> wild-type Tmc1 into hair cells <strong>of</strong> deaf mice can restore sensory transduction<br />
in vitro. Whether AAV-Tmc1 can restore sensory function in vivo is currently under investigation.<br />
Lawrence Lustig, MD<br />
AAV1-mediated Restoration <strong>of</strong> Hearing in the VGLUT3 Knockout Mouse<br />
Mice lacking the vesicular glutamate transporter-3 (VGLUT3) are congenitally deaf due to loss <strong>of</strong> glutamate release at the inner hair<br />
cell afferent synapse. Within two weeks <strong>of</strong> AAV1-VGLUT3 delivery, acoustic brainstem response (ABR) thresholds normalize, along<br />
with partial rescue <strong>of</strong> the startle response. Lastly, we demonstrate partial reversal <strong>of</strong> the morphologic changes seen within the<br />
afferent IHC ribbon synapse. These indings will be discussed and placed into the broader context <strong>of</strong> gene therapy for genetic forms<br />
<strong>of</strong> hearing loss.<br />
Jeffrey R. Martens, PhD<br />
<strong>Gene</strong> Therapeutic Rescue <strong>of</strong> an Olfactory Ciliopathy<br />
Olfactory dysfunction is a clinical manifestation <strong>of</strong> a growing class <strong>of</strong> human genetic diseases, termed ciliopathies. Despite<br />
substantial progress made in identifying genes that cause ciliopathies, therapies for these disorders are not yet available to patients.<br />
Here, we reported that odor detection can be restored to animals with a hypomorphic mutation in the gene encoding for the ciliary<br />
protein IFT88 that results in the loss <strong>of</strong> cilia on differentiated olfactory sensory neurons (OSNs). These studies represent the irst in<br />
vivo therapeutic treatment to reestablish cilia in a mammalian ciliopathy, and show that gene therapy is a viable curative option for<br />
functional rescue <strong>of</strong> the complex cilia organelle in established differentiated cells.<br />
32<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013
<strong>Program</strong> Schedule, Wednesday, May 15, 2013<br />
Marcelo N. Rivolta, MD, PhD<br />
Restoration <strong>of</strong> Auditory Evoked Responses by hESC-derived Otic Progenitors<br />
The presentation will discuss recent advances using stem cells in the search <strong>of</strong> a treatment for hearing loss. A method has been<br />
developed to generate ear sensory cells from human embryonic stem cells (hESCs). By activating FGF signalling on hESCs using<br />
ligands that induce the formation <strong>of</strong> the ear in vivo, we have generated otic progenitors that can produce sensory hair cell-like cells<br />
and auditory neurons. We have then taken the hESC-derived otic progenitor cells and explore if they could repair a deaf ear in a<br />
gerbil model <strong>of</strong> auditory neuropathy. When hESC-derived ear cells were transplanted into cochleae that have lost their auditory<br />
neurons the cells survived, engrafted and differentiated. Moreover, they sent projections making connections with the hair cells and<br />
with the brain and, more remarkably, they elicited a functional recovery represented by improved ABR thresholds. Related work,<br />
developing them as a platform for gene and drug discovery will also be reviewed.<br />
Scientific Symposium 103<br />
8:00 am - 10:00 am<br />
ROOM: 150 DEFG<br />
Novel Approaches to Cancer Immunotherapy<br />
CO-CHAIRS: Jennifer E. Adair, PhD and Elizabeth A. Mittendorf, MD, PhD<br />
SPEAKERS<br />
Wednesday, May 15, 2013<br />
Elizabeth A. Mittendorf, MD, PhD<br />
AE37: A Novel Hybrid Vaccine for Stimulating a Helper T <strong>Cell</strong> Response<br />
Our group has been leading the clinical trial efforts evaluating AE37, a novel vaccine that combines AE36, a native HER2-derived<br />
MHC class II epitope to the Ii-Key moiety. Linkage <strong>of</strong> the Ii-key moiety increases antigen presentation in vivo leading to increased<br />
potency <strong>of</strong> the vaccine. Phase I trials have demonstrated AE37 to be safe and effective in eliciting a HER2-speciic immune response.<br />
An ongoing phase II trial has shown evidence <strong>of</strong> clinical eficacy; at the time <strong>of</strong> a planned analysis at 24 month median follow-up,<br />
the disease-free survival rate was 90% in vaccinated patients versus 83% in non-vaccinated controls, a 41% relative risk reduction.<br />
This talk will focus on the preclinical and early clinical trial data available for the AE37 vaccine.<br />
John J. Nemunaitis, MD<br />
Clinical Update <strong>of</strong> bi-shRNA furin/GMCSF DNA Transfected Tumor Vaccine (FANG) in Cancer Patients<br />
Exciting the immune system to attack cancer involves 3 critical components: education <strong>of</strong> tumor antigens, enhancement <strong>of</strong> cancer<br />
targeting immune function, and blockade <strong>of</strong> pro-cancer immune inhibitors. FANG is a novel “triad” vaccine which utilizes both<br />
cellular and DNA technology to accomplish all 3 components. Speciically, autologous harvested tumor cells are transfected with a<br />
dual plasmid involving a GMCSF DNA cassette and a bifunctional RNA interference DNA cassette targeting furin, the proprotease<br />
converting enzyme <strong>of</strong> TGFß1 and TGFß2. Phase I trial involving 42 advanced cancer patients (Senzer et. al. Mol Ther 2012: 679-<br />
686) demonstrated patient safety, veriication <strong>of</strong> product mechanism and correlation <strong>of</strong> signiicant survival improvement with<br />
induction <strong>of</strong> activated T-cells as measured by ELISPOT response. Follow up results <strong>of</strong> the Phase I trial with demonstration <strong>of</strong> median<br />
survival advantage in comparison <strong>of</strong> FANG treated patients to FANG not treated patients <strong>of</strong> 562 days vs. 181 days (p
<strong>Program</strong> Schedule, Wednesday, May 15, 2013<br />
Wednesday, May 15, 2013<br />
Scientific Symposium 104<br />
8:00 am - 10:00 am<br />
ROOM: 151 ABCG<br />
Novel Structural and Functional Assessment <strong>of</strong> the Respiratory Tract: Potential New Endpoints<br />
for <strong>Gene</strong> and <strong>Cell</strong> <strong>Therapy</strong><br />
CO-CHAIRS: David A. Dean, PhD and Christian Mueller, PhD<br />
SPEAKERS<br />
David W. Parsons, PhD<br />
Developments in Functional X-ray Imaging: ASL, MCT and “Pinpoint Spirometry”<br />
Driven by a desire to rapidly, accurately and reliably measure the effect <strong>of</strong> airway gene therapy treatments for Cystic Fibrosis<br />
in mice, we have been developing several novel analytical options for airways using synchrotron phase contrast X-ray. The high<br />
lux and coherence <strong>of</strong> synchrotron X-rays underlie our ability to visualize mouse airway-surface anatomy and activity in-vivo, to<br />
sub-micron resolution; ultra-fast imaging <strong>of</strong> dynamic lung movement in 4-dimensions to reveal regional airlows is also enabled.<br />
Importantly, all <strong>of</strong> these new approaches are designed to be non-invasive and allow airway health and function in intact animals<br />
to be examined. Alterations in the depth <strong>of</strong> the airway surface liquid (ASL) and altered Mucociliary Transit (MCT) are the earliest<br />
physiological effects <strong>of</strong> CFTR activity that can produce visible change in airway surface structure and function, and are key markers<br />
<strong>of</strong> CF airway health to be improved by an effective gene therapy. The core methods for the image capture, analysis, and presentation<br />
<strong>of</strong> both ASL and MCT markers are rapidly developing to provide new endpoint tools. The application <strong>of</strong> velocimetry techniques to<br />
synchrotron CT data has created a powerful new tool for regional lung health assessment (”pinpoint spirometry”) that is likely to be<br />
<strong>of</strong> particular value in animal models, and is also well suited to translation into a clinically-applicable system.<br />
Cecilia Lo, PhD<br />
Respiratory Motile Cilia Dysfunction in Congenital Heart Disease Patients and Contribution to Worse<br />
Postsurgical Outcomes<br />
Steven M. Rowe, MD<br />
Functional Anatomic Imaging <strong>of</strong> the CF Airway<br />
A critical barrier causing a roadblock in our understanding <strong>of</strong> CF pathogenesis is the lack <strong>of</strong> tools available for visualizing the<br />
important microstructural, functional, and biomechanical features <strong>of</strong> the respiratory mucosa and mucus in vivo. Recently, our<br />
laboratories have developed an innovative technology, termed 1-µm resolution optical coherence tomography (µOCT), which<br />
enables real-time cross-sectional microscopy <strong>of</strong> the functional epithelial surface <strong>of</strong> living airways and can be readily combined with<br />
measures <strong>of</strong> ion transport in vivo. With µOCT, we have been able to simultaneously and quantitatively monitor airway surface liquid<br />
(ASL) and periciliary layer (PCL) depths, ciliary beating, and mucociliary transport (MCT) in situ, while also measuring mucus<br />
viscosity by native particle tracking techniques without the use <strong>of</strong> contrast dyes, exogenous microparticles, or other manipulations.<br />
Key manifestations <strong>of</strong> the underlying CF defect can readily be observed, and an in vivo probe is currently under development. In<br />
addition to answering important questions regarding CF pathophysiology, this technology provides a clear path towards the use <strong>of</strong><br />
µOCT for monitoring functional responses to novel therapeutics.<br />
Maria P. Limberis, PhD<br />
Development <strong>of</strong> an AAV-based Prophylactic Vaccine against Pandemic Inluenza<br />
We will discuss the development <strong>of</strong> an effective prophylactic vaccine against inluenza. This vaccine is based on AAV9 vector and<br />
expresses a broadly neutralizing antibody against inluenza that protects relevant animal models against challenge with H5N1 and<br />
H1N1.<br />
34<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013
<strong>Program</strong> Schedule, Wednesday, May 15, 2013<br />
Scientific Symposium 105<br />
8:00 am - 10:00 am<br />
ROOM: 150 ABC<br />
Polymeric <strong>Gene</strong> Delivery<br />
CHAIR: Sung Wan Kim, PhD<br />
SPEAKERS<br />
Wim E. Hennink, PhD<br />
Decationized Polyplexes: Novel Polymeric <strong>Gene</strong> Delivery System with Reduced Toxicity<br />
The clinical applicability <strong>of</strong> polymers as gene delivery systems depends not only on their eficiency, but also on their safety. The<br />
cytotoxicity <strong>of</strong> these systems remains a major issue, mainly due to their cationic nature. Therefore, it is highly preferable to have a<br />
system based on biocompatible neutral polymers, lacking polycations, without compromising the DNA condensing and protecting<br />
capacities. We introduce a concept to obtain a neutral polymeric gene delivery system, through a 3-step process to generate<br />
polyplexes with a core <strong>of</strong> disulide crosslinked poly(hydroxypropyl methacrylamide) (pHPMA) in which plasmid DNA (pDNA) is<br />
entrapped and a shell <strong>of</strong> poly(ethylene glycol) (PEG).<br />
Wednesday, May 15, 2013<br />
Ram I. Mahato, PhD<br />
Emerging Trends in Combination <strong>Therapy</strong> with RNAi<br />
Treatment strategies relying on a single small molecule drug, or silencing <strong>of</strong> a single gene <strong>of</strong>ten fail, while simultaneous targeting<br />
<strong>of</strong> multiple cellular pathways is more likely to succeed. RNA interference (RNAi) is a promising approach for treating diabetes and<br />
cancer. By pooling siRNAs targeting two different regions <strong>of</strong> the same target gene, or combining two siRNAs against two different<br />
genes, or even co-formulating small molecule drug with speciic siRNA or miRNA, we hope to improve islet transplantation and<br />
treat advanced prostate and pancreatic cancer. In this presentation, I will discuss our recent results on RNAi technologies for<br />
treating diabetes and cancer.<br />
Chae-Ok Yun, PhD<br />
Harnessing Adenovirus for Tumor Targeting by Nanocomplex<br />
A challenge to develop adenovirus (Ad)-mediated therapeutics has been issued to treat metastatic cancer via systemic<br />
administration. For effective gene therapeutics against primary and metastatic lesions, a systemically injectable tumor-targeting<br />
Ad vector system must be developed. Systemic delivery <strong>of</strong> Ad, however, is hampered by immune response against Ad, short half-life<br />
in the circulation, liver uptake, and low accumulation at target disease sites. Modiication <strong>of</strong> the Ad surface allows Ad to circulate<br />
in the bloodstream for a longer time, to be not targeted to the liver, and to passively accumulate in tumor sites via enhanced<br />
permeation and retention effects. The addition <strong>of</strong> afinity tags results in active targeting and high eficacy. Strategies including<br />
addition <strong>of</strong> polymer complexes, chemical modiications, and targeting moieties for the development <strong>of</strong> systemically injectable Ad<br />
gene therapeutic carriers will be discussed.<br />
Kazunori Kataoka, PhD<br />
Polymeric Micellar Nanocarriers for <strong>Gene</strong> and Oligonucleotide Delivery<br />
Focus <strong>of</strong> my talk will be on the use <strong>of</strong> self-assembly structures from multi-functional block copolymers, particularly polymeric<br />
micelles, for gene and oligonucleotide delivery. Importance <strong>of</strong> peptide ligands to facilitate extravasation as well as to control<br />
intracellular traficking <strong>of</strong> polymeric micelles will be discussed. Also, I will present our new strategy to delivery messenger RNA to<br />
target organ.<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013<br />
35
<strong>Program</strong> Schedule, Wednesday, May 15, 2013<br />
Wednesday, May 15, 2013<br />
Education Sessions<br />
The goal <strong>of</strong> the ASGCT Education <strong>Program</strong> is to provide participants with a broad background and introduction to topics relevant to the<br />
ield <strong>of</strong> gene and cell therapy. Education Sessions will be subdivided into two headings. “Topical Review” sessions will provide a broad<br />
perspective <strong>of</strong> an area <strong>of</strong> importance to the gene and cell therapy community such as major vector systems, speciic target organs or<br />
diseases, and funding/regulatory issues. “Emerging Field Review” sessions will provide a broad perspective <strong>of</strong> a technological or scientiic<br />
discipline that has recently become <strong>of</strong> great signiicance to ASGCT members.<br />
Education Session 110<br />
10:30 am - 12:00 pm<br />
ROOM: BALLROOM B<br />
Emerging Field Review: <strong>Gene</strong> Targeting<br />
<strong>Gene</strong> targeting, also known as genome editing, has become an increasingly feasible gene therapy strategy. In this education, three pioneers<br />
in the ield will describe different approaches to gene targeting. After this session, attendees should be able to understand the different<br />
approaches to gene targeting and which strategy might be best for their speciic gene therapy application.<br />
CHAIR: Matthew H. Porteus, MD, PhD<br />
SPEAKERS<br />
David W. Russell, MD, PhD<br />
AAV-Mediated <strong>Gene</strong> Targeting<br />
This presentation will describe the mechanism and applications <strong>of</strong> AAV-mediated gene targeting, which has been used to target<br />
over 100 genes in more than 50 different cell types. Sequence changes can be introduced at high frequencies, with up to 1% <strong>of</strong><br />
infected cells undergoing gene targeting. Insertions, deletions and substitutions can all be accurately introduced, and additional<br />
unwanted mutations are not found at targeted loci. Site-speciic nucleases are not required to obtain high targeting frequencies, so<br />
there is no risk <strong>of</strong> introducing DNA breaks at <strong>of</strong>f-target sites.<br />
J. Keith Joung, MD, PhD<br />
Engineered TALENs and CRISPR-Cas RNA-Guided Nucleases (RGNs) for Targeted Genome Editing<br />
Engineered site-speciic nucleases have emerged as a broadly applicable technology for eficiently inducing targeted alterations<br />
in the genomes <strong>of</strong> living cells and organisms. This talk will provide an introduction to two engineered nuclease platforms:<br />
transcription activator-like effector nucleases (TALENs) and CRISPR-Cas RNA-Guided Nucleases (RGNs). The strengths and<br />
limitations <strong>of</strong> each <strong>of</strong> these platforms will be discussed. In addition, publicly available methods and resources for practicing these<br />
technologies will be reviewed.<br />
Dana Carroll, PhD<br />
Nuclease-mediated <strong>Gene</strong> Targeting<br />
With new and rapidly developing technology, it is now possible to make knock-outs and knock-ins in virtually any gene in a wide<br />
range <strong>of</strong> cells and organisms. The secret is the development <strong>of</strong> nucleases that can be designed to cleave arbitrarily chosen genomic<br />
DNA sequences with high speciicity. <strong>Cell</strong>ular repair processes create localized mutations at the nuclease-induced break, or copy<br />
novel sequences from a user-provided template. This talk will cover two classes <strong>of</strong> targetable cleavage reagents, zinc-inger<br />
nucleases (ZFNs) and TALENs. The research and therapeutic potential <strong>of</strong> these reagents is enormous, and ZFNs are currently in<br />
clinical trials.<br />
Education Session 111<br />
10:30 am - 12:00 pm<br />
ROOM: BALLROOM C<br />
Emerging Field Review: Ocular <strong>Gene</strong> <strong>Therapy</strong>: Tools and <strong>Gene</strong>s<br />
Following the success <strong>of</strong> the clinical gene therapy trials for an early onset form <strong>of</strong> inherited blindness called LCA2, the momentum in the<br />
ield <strong>of</strong> ocular gene therapy is palpable. In this session, we try to capture this excitement by providing an in depth review into the genetics<br />
<strong>of</strong> vision and blindness and the technologies that are being developed to therapeutically intervene in the disease course using gene transfer.<br />
This educational session also will discuss disease gene discovery in the genomic era, genetic diagnosis for inherited forms <strong>of</strong> blindness, gene<br />
therapies for blindness under development, and AAV and nanoparticle vector systems for retinal targeting.<br />
36<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013
<strong>Program</strong> Schedule, Wednesday, May 15, 2013<br />
CHAIR: Luk H. Vandenberghe, PhD<br />
SPEAKERS<br />
Eric A. Pierce, MD, PhD<br />
<strong>Gene</strong> Discovery in Inherited Retinal Blindness<br />
IRDs important causes <strong>of</strong> blindness. Pro<strong>of</strong> <strong>of</strong> concept clinical trials gene therapy for RPE65 LCA suggest that these disorders<br />
are amendable to treatment with gene therapies. The genetic heterogeneity <strong>of</strong> these disorders present challenges for genetic<br />
diagnostic testing. The use <strong>of</strong> next-generation sequencing techniques for genetic diagnostic testing and disease gene discovery<br />
research for these disorders will be discussed.<br />
Shannon E. Boye, PhD<br />
AAV <strong>Gene</strong> Transfer to the Retina<br />
Clinical trials for RPE65-LCA (LCA2) have demonstrated the ability to target a subretinally- delivered therapeutic transgene to<br />
the retinal pigment epithelium (RPE) thereby restoring retinal function and visually-evoked behavior to these patients. However,<br />
because most retinal degenerations are caused by mutations in photoreceptor (PR)- speciic genes, there is a great need to develop<br />
PR-targeted gene therapies. This presentation will review and compare known PR-targeted AAV vectors, with emphasis placed on<br />
results recently obtained in higher order species following subretinal delivery. The future development <strong>of</strong> intravitreal-delivered<br />
vectors targeted to photoreceptors will also be discussed.<br />
Wednesday, May 15, 2013<br />
Muna I. Naash, PhD<br />
Nanoparticle-mediated Ocular <strong>Gene</strong> Delivery<br />
The eye is an organ that is well-suited for the development and testing <strong>of</strong> novel therapeutic approaches. It is easily accessible and<br />
allows local application <strong>of</strong> therapeutic agents with reduced risk <strong>of</strong> systemic effects. Need exists for the development <strong>of</strong> non-viral<br />
therapeutic approaches for ocular diseases. Our lab and others have investigated the potentials <strong>of</strong> nanotechnology for ocular<br />
delivery <strong>of</strong> therapeutic genes. Investigations thus far have highlighted great potentials for nanoparticles to be a successful approach<br />
for ocular gene delivery. Our group has focused on the eficacy <strong>of</strong> compacted DNA nanoparticles for the treatment <strong>of</strong> different<br />
diseases, particularly those associated with the retina and retinal pigment epithelium. We have shown that nanoparticle treatment<br />
leads to eficient transfection <strong>of</strong> ocular cells, long term gene expression, and exerts no toxic effects on the eye even after multiple<br />
injections. These nanoparticles mediate signiicant functional rescue in models <strong>of</strong> retinitis pigmentosa, Stargardt macular dystrophy<br />
and Leber’s congenital amaurosis. They have no limitations on the size <strong>of</strong> the genetic cargo and effective gene expression has been<br />
demonstrated with vectors up to 20 kb in the lung and 14 kb in the eye, making them an ideal complement to AAVs especially for<br />
delivery <strong>of</strong> large genes. Furthermore, in a side-by-side comparison study with AAV, we recently reported that nanoparticles can<br />
drive gene expression on a comparable scale and longevity to AAV.<br />
Education Session 112<br />
10:30 am - 12:00 pm<br />
ROOM: BALLROOM D<br />
Topical Review: A Primer on Non-Viral Delivery<br />
CO-CHAIRS: Ian MacLachlan, PhD and Kevin G. Rice, PhD<br />
SPEAKERS<br />
Ian MacLachlan, PhD<br />
Liposomal and Lipid Nanoparticle Formulations for the Delivery <strong>of</strong> siRNA<br />
David H. Thompson, PhD<br />
Non-Viral Vectors: Correlations Between Structure and Performance<br />
A vast array <strong>of</strong> materials have been screened as potential non-viral vectors, including cationic lipids, hyperbranched polymers,<br />
dendrimers, cyclodextrin polymers, and nanoparticle carriers, however, few <strong>of</strong> the materials developed for this application have<br />
been systematically evaluated for their structure-property relationships. This presentation will begin by briely reviewing the<br />
various strategies used before focusing on those systems where it has been possible to correlate biological performance with the<br />
structure and/or physical properties <strong>of</strong> the non-viral vector. <strong>Final</strong>ly, new directions in non-viral vector development will be covered<br />
with an emphasis on mechanism-based studies.<br />
Kevin G. Rice, PhD<br />
Principles <strong>of</strong> DNA Polyplex Design for In Vitro and In Vivo <strong>Gene</strong> Delivery<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013<br />
37
<strong>Program</strong> Schedule, Wednesday, May 15, 2013<br />
Wednesday, May 15, 2013<br />
Education Session 113<br />
10:30 am - 12:00 pm<br />
ROOM: 150 DEFG<br />
Topical Review: <strong>Gene</strong> and <strong>Cell</strong> <strong>Therapy</strong> Approaches to Hemoglobinopathies<br />
This session will introduce basic, translational and clinical efforts to provide therapeutic advances for the two major Hemoglobinopathies:<br />
sickle-cell anemia (SCD) and beta-thalassemia’s using gene therapy approaches. Elevation <strong>of</strong> HbF production has been a long-standing<br />
goal <strong>of</strong> therapy for ß-thalassemia and sickle cell disease (SCD) as its expression provides a potent attenuation <strong>of</strong> many <strong>of</strong> the most severe<br />
complications <strong>of</strong> disease phenotype. The central theme <strong>of</strong> this session will be improving gene transfer and gene correction in human<br />
hematopoietic stem and progenitor cells to provide more effective and safe therapies for blood cell disorders. In addition, efforts and the<br />
lessons learnt from gene therapy <strong>of</strong> Severe Combined Immunodeiciency (SCID) disease.<br />
CO-CHAIRS: Pankaj Qasba, PhD and Sonia I. Skarlatos, PhD<br />
SPEAKERS<br />
Punam Malik, MD, PhD<br />
<strong>Gene</strong> and <strong>Cell</strong> <strong>Therapy</strong> Approaches to Hemoglobinopathies<br />
Dr. Malik will present an overview <strong>of</strong> genetic therapies for hemoglobiniopathies, a review <strong>of</strong> the literature, the current state <strong>of</strong> the<br />
ield and future promising ways <strong>of</strong> gene editing and reprogramming.<br />
John F. Tisdale, MD<br />
Stem <strong>Cell</strong>s as <strong>Gene</strong> <strong>Therapy</strong> Vehicles for the Treatment <strong>of</strong> Disorders <strong>of</strong> Globin Synthesis<br />
Sickle cell disease (SCD) is a recessive genetic disease that results from the production <strong>of</strong> an abnormal hemoglobin that is prone<br />
to polymerization upon deoxygenation causing severe morbidity and early mortality. Although palliative therapies have been<br />
developed, conventional treatment remains unsatisfactory. Signiicant advances in cellular and molecular biology have enabled<br />
the development <strong>of</strong> a mounting armamentarium <strong>of</strong> tools to allow correction at the hematopoietic stem cell (HSC) level. Allogeneic<br />
HSC transplantation, a form <strong>of</strong> genetic therapy, is an established curative approach through replacement <strong>of</strong> the defective organ<br />
with that <strong>of</strong> a donor carrying the normal genotype; however, procedural toxicities limit this approach to only severely affected<br />
children. Nonmyeloablative approaches based upon basic and preclinical studies have now allowed for the successful application<br />
<strong>of</strong> this approach in adults with severe disease, with disease reversion in the majority <strong>of</strong> patients treated through the attainment <strong>of</strong><br />
stable mixed hematopoietic chimerism. Nonetheless, this approach remains limited by donor availability, and the development <strong>of</strong><br />
gene transfer directed at autologous HSCs remains a desirable alternative. Approaches using autologous transplantation <strong>of</strong> HSCs<br />
modiied by lentiviral vectors have demonstrated expression <strong>of</strong> the human beta-globin protein at clinically signiicant levels, and<br />
this approach is now being developed for clinical application in SCD. The progress toward the goal <strong>of</strong> these HSC based therapies will<br />
be reviewed.<br />
Donald B. Kohn, MD<br />
Lessons Learned from <strong>Gene</strong> <strong>Therapy</strong> for SCID<br />
The implementation and performance <strong>of</strong> clinical trials <strong>of</strong> gene therapy using hematopoietic stem cells is a complex process. After<br />
pro<strong>of</strong>-<strong>of</strong>-principle studies demonstrate potential disease modifying activity, preclinical activity and toxicology data need to be<br />
generated, along with clinical protocol, consent documents, etc. to support regulatory applications. Subjects are treated in the<br />
high acuity setting <strong>of</strong> hematopoietic stem cell transplantation (HSCT) which may include the use <strong>of</strong> myeloreductive conditioning,<br />
although no immune suppression pre- or post-transplant is needed, due to the use <strong>of</strong> autologous cells. Ultimately, clinical<br />
acceptance <strong>of</strong> gene therapy as a standard <strong>of</strong> care will depend on demonstrating equivalent or greater eficacy and improved<br />
outcomes or reduced costs, compared to allogeneic HSCT. Experience with gene therapy for severe combined immune deiciency<br />
may inform efforts to develop similar approaches to hemoglobinopathies; persistence and patience are needed.<br />
Education Session 114<br />
10:30 am - 12:00 pm<br />
ROOM: 151 ABCG<br />
Topical Review: Vector Design<br />
CO-CHAIRS: Alberto Auricchio, MD and Dirk Grimm, PhD<br />
38<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013
<strong>Program</strong> Schedule, Wednesday, May 15, 2013<br />
SPEAKERS<br />
Anja Ehrhardt, PhD<br />
Viral Hybrid-Vectors for Stable Tranduction <strong>of</strong> Mammalian <strong>Cell</strong>s: Molecular Design, Eficacy and Applications<br />
Viral hybrid-vectors combine the high transduction eficiencies <strong>of</strong> recombinant viruses with improved genetic elements for<br />
stabilized transgene expression. This tutorial provides a state-<strong>of</strong>-the-art overview <strong>of</strong> available viral hybrid-vector systems for<br />
random somatic integration (Sleeping Beauty transposase), targeted integration (designer nucleases) and episomal persistence<br />
<strong>of</strong> the transgene (plasmid replicons). Advantages but also limitations <strong>of</strong> each vector system will be discussed and safety issues<br />
addressed. Furthermore, recent in vivo applications will be mentioned.<br />
Alberto Auricchio, MD<br />
AAV - From the Genome to the Capsid<br />
Vectors based on the adeno-associated virus (AAV) are being tested in humans and the initial safety and eficacy results in several<br />
tissues, including liver, muscle, retina and central nervous system are extremely encouraging. The availability <strong>of</strong> hundreds <strong>of</strong><br />
naturally-occurring AAV serotypes, as well as <strong>of</strong> mutagenized variants with improved and different transduction proiles provide<br />
a theoretical endless source <strong>of</strong> vectors for in vivo gene therapy. Sophisticated strategies were developed to tailor transgene<br />
expression to speciic cell types within the AAV cargo capacity. In addition, efforts are being made to expand AAV cargo capacity<br />
for the expression <strong>of</strong> large transgenes. Improvements on both the outside (capsid) and inside (genome) <strong>of</strong> AAV vectors will be<br />
discussed in the context <strong>of</strong> the retina which is an excellent target tissue to challenge the versatile AAV platform.<br />
Wednesday, May 15, 2013<br />
Philip Ng, PhD<br />
Helper-dependent Adenovirus: Design, Production and Applications<br />
This education session will discuss optimal design, eficient production and novel applications <strong>of</strong> helper dependent adenovirus for<br />
gene and cell therapy.<br />
Lunch Break (On Own - Not Provided; Pre-ordered boxed lunches available for pick up at Registration Counter)<br />
12:00 pm - 1:15 pm<br />
Education Session 120<br />
1:15 pm - 2:45 pm<br />
ROOM: BALLROOM B<br />
Emerging Field Review: Therapeutic Antibody <strong>Gene</strong> Delivery<br />
Recent advances in monoclonal antibody discovery, isolation, and expression have created unprecedented opportunities to utilize this class<br />
<strong>of</strong> proteins for broad therapeutic beneit. A number <strong>of</strong> studies have highlighted several potential applications for the use <strong>of</strong> antibodies<br />
in vivo, ranging from the prevention <strong>of</strong> infectious diseases, to the treatment <strong>of</strong> drug addiction. This session will focus on several <strong>of</strong> these<br />
applications and will highlight strategies aimed at maximizing vector-based expression <strong>of</strong> antibodies in vivo.<br />
CO-CHAIRS: Alejandro B. Balazs, PhD and James M. Wilson, MD, PhD<br />
SPEAKERS<br />
James M. Wilson, MD, PhD<br />
Inluenza Antibodies and Airway Delivery<br />
Ronald G. Crystal, MD<br />
<strong>Gene</strong> Transfer Strategies to Treat Addiction<br />
Addiction to drugs is a major worldwide problem for which there is no effective therapy. Using cocaine and nicotine as examples<br />
<strong>of</strong> commonly used addictive drugs, we have developed two platform strategies to evoke high-titer antibodies speciic for the antiaddictive<br />
drug. In the irst strategy, based on the highly immunogenic nature <strong>of</strong> the adenovirus capsid, analogs <strong>of</strong> the addictive<br />
drug are bound to adenovirus capsid proteins to create active vaccines that generate high-titer anti-drug antibodies. In the second<br />
strategy, the gene coding sequences for monoclonals against the addictive drug are expressed by adeno-associated virus gene<br />
transefer vectors capable <strong>of</strong> generating, with a single administration, persistent high titers <strong>of</strong> anti-drug antibodies. In experimental<br />
animal models, both strategies are highly effective, providing new paradigms for anti-addictive molecular vaccines.<br />
Alejandro B. Balazs, PhD<br />
Expressing Antibodies in vivo via Adeno Associated Virus Vector for Vectored ImmunoProphylaxis<br />
Recent advances in vector-based antibody delivery have created the potential for engineering humoral immunity to address<br />
problems ranging from infectious disease to drug addiction. These efforts are <strong>of</strong>ten hampered by dificulties in achieving<br />
therapeutic levels <strong>of</strong> antibody expression. This presentation will focus on means <strong>of</strong> improving expression <strong>of</strong> antibodies in vivo and<br />
summarize the challenges <strong>of</strong> working with this novel application <strong>of</strong> gene transfer.<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013<br />
39
<strong>Program</strong> Schedule, Wednesday, May 15, 2013<br />
Wednesday, May 15, 2013<br />
Education Session 121<br />
1:15 pm - 2:45 pm<br />
ROOM: BALLROOM C<br />
Topical Review: Cancer <strong>Gene</strong> <strong>Therapy</strong><br />
The ield <strong>of</strong> Cancer <strong>Gene</strong> <strong>Therapy</strong> has seen many recent developments concerning applications to treat different tumors using a variety<br />
<strong>of</strong> novel vector systems that are now being exploited in human clinical trials. This session will deal with pre-clinical accomplishments<br />
concerning the use <strong>of</strong> chimeric antigen receptor (CAR) modiied T-cells directed against tumor associated antigens, the use <strong>of</strong> novel tumorselective<br />
replication-competent retroviruses expressing prodrugs, and the use <strong>of</strong> bifunctional shRNAi nanoplex technology for advanced<br />
methods <strong>of</strong> killing tumors using gene/cell therapies. Recent signiicant progress in clinical trials resulting from these approaches will also be<br />
presented.<br />
CHAIR: William F. Goins, PhD<br />
SPEAKERS<br />
John J. Nemunaitis, MD<br />
Clinical Update <strong>of</strong> bifunctional (bi)-shRNAi Nanoplex Technology in Cancer<br />
Several RNAi approaches (miRNA, siRNA, shRNA, bi-shRNA) are being explored to exquisitely disrupt post transcriptional gene<br />
silencing <strong>of</strong> cancer relevant signals. Twenty-six clinical studies involving RNAi technology have been published. bi-shRNAi (Issued<br />
Patent No. 8,252,526) utilizes a dual mechanism to knockdown sequence speciic targets by engaging both siRNA and miRNA-like<br />
RNA-inducing silence complex. The bi-shRNAi technology was initially introduced into the clinical areas to target a proprotease<br />
converting enzyme, furin, as one <strong>of</strong> 3 components involving a novel FANG vaccine (Senzer et. Al. Mol Ther 2012: 679-686) in which<br />
clinical demonstration <strong>of</strong> protein knockdown to the designed target, furin and downstream signals (TGFß1, ß2) was greater than<br />
90%, and immune response in correlation with survival advantage was demonstrated. In an effort to expand bi-shRNAi technology<br />
and to target other potentially therapeutic molecular signals, we have utilized a nanoplex approach by incorporating the bi-shRNAi<br />
plasmid into a bilamellar invaginated vesicle (BIV) (Issued Patent No. 7,037,520) which can be decorated with reversible masking<br />
lipids and targeting small molecule ligands (Issued U.S. Patient No. 8,333,988). Results <strong>of</strong> initial preclinical and clinical studies will<br />
be reviewed.<br />
Noriyuki Kasahara, MD, PhD<br />
Development <strong>of</strong> Novel Vectors and Strategies for <strong>Gene</strong> <strong>Therapy</strong> <strong>of</strong> Cancer<br />
Oncolytic virotherapy, engineered T cells and cancer vaccines, and stem cell-based tumor-targeting strategies are currently<br />
being evaluated in the clinic and show considerable promise. Newer approaches seek to further enhance the potency <strong>of</strong> RNA<br />
oligonucleotide-based therapeutics, replicating virus vectors, and engineered cell therapies, while maintaining tumor-selectivity<br />
and safety. An overview <strong>of</strong> recent advances showing signiicant promise for the development <strong>of</strong> clinically effective gene therapy<br />
against cancer will be presented.<br />
Gianpietro Dotti, MD<br />
Chimeric Antigen Receptor (CAR) Engineered T-cell Cancer Therapies<br />
Education Session 122<br />
1:15 pm - 2:45 pm<br />
ROOM: BALLROOM D<br />
Topical Review: <strong>Gene</strong> <strong>Therapy</strong> to the Marketplace<br />
CHAIR: H. Trent Spencer, PhD<br />
SPEAKERS<br />
H. Trent Spencer, PhD<br />
Replacing Treatments with Cures: Potential Therapeutics for Hemophilia A<br />
Hemophilia A is a monogenic bleeding disorder currently treated by protein replacement therapy. <strong>Gene</strong> therapy provides an<br />
alternative treatment that can eliminate the need for recombinant protein administration. Preclinical and clinical data show a long<br />
lasting/lifelong treatment is feasible, and various models have shown gene therapy for hemophilia A is also inancially attractive,<br />
which further justiies the development <strong>of</strong> these curative treatments. This presentation will summarize hemophilia treatment<br />
options with respect to their market potentials.<br />
R. Scott McIvor, PhD<br />
Discovery Genomics: Development <strong>of</strong> the Sleeping Beauty Transposon System for <strong>Gene</strong> <strong>Therapy</strong><br />
40<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013
<strong>Program</strong> Schedule, Wednesday, May 15, 2013<br />
Khursheed Anwer, PhD<br />
IL-12 <strong>Gene</strong> <strong>Therapy</strong>: From Bench to Clinic<br />
This presentation describes the clinical and preclinical development <strong>of</strong> EGEN-001, an interleukin-12 (IL-12) gene expression<br />
plasmid formulated with a biocompatible delivery lipopolymer, PEG-PEI-Cholesterol (PPC), for the treatment <strong>of</strong> peritoneally<br />
metastasized gynecological and gastrointestinal cancers. This cytokine expression <strong>of</strong> IL-12 is designed to increase the local<br />
concentration <strong>of</strong> IL-12 with minimal escape into the systemic circulation thus minimizing the systemic toxicities associated with<br />
the recombinant IL-12 therapy. Two Phase I trials <strong>of</strong> EGEN-001 administered as a single agent or in combination with standard<br />
chemotherapy agents in advanced ovarian cancer patients have been completed, a Phase II trial <strong>of</strong> EGEN-001 as a single agent in the<br />
platinum-resistant patient population is in progress, and a Phase I/II trial in combination with chemotherapy agents in metastatic<br />
colorectal cancer patients has been initiated. The manufacturing <strong>of</strong> this investigational product has been successfully advanced<br />
from bench scale to clinical scale and several cGMP lots have been successfully produced. A detailed description <strong>of</strong> EGEN-001<br />
pharmacology/toxicology and pharmaceutical properties will be presented.<br />
Wednesday, May 15, 2013<br />
Education Session 123<br />
1:15 pm - 2:45 pm<br />
ROOM: 150 DEFG<br />
Topical Review: Navigating a Dificult Funding Climate<br />
This session will present NIH training and career opportunities for investigators as well as what type <strong>of</strong> support is available from<br />
Foundations and Industry. In addition, two presentations will focus on how to write an application from the perspective <strong>of</strong> a senior and<br />
junior investigator.<br />
CO-CHAIRS: Scott Q. Harper, PhD and Sonia I. Skarlatos, PhD<br />
SPEAKERS<br />
Barry J. Byrne, MD, PhD<br />
Scott Q. Harper, PhD<br />
Startup Funds Don’t Last Forever: A Junior PI’s Path Toward Funding and Growing a New Lab<br />
Many <strong>of</strong> us spend a decade or more training to become independent scientists. When the opportunity inally arrives, walking into<br />
our own empty labs for the very irst time can be exhilarating and overwhelming. This moment also means the clock begins ticking<br />
on the next stage <strong>of</strong> our careers. Arguably the most critical part <strong>of</strong> this next stage is acquiring independent funding to sustain and<br />
grow our lab, which is no easy task in today’s economic climate. In this presentation, I will discuss my path for leveraging my lab<br />
startup package into independent funding.<br />
Drew E. Carlson, PhD<br />
Fellowships and Career Awards: Stepping Stones to Research Independence<br />
The NIH <strong>of</strong>fers a myriad <strong>of</strong> fellowships and K awards to facilitate the pursuit <strong>of</strong> a career in biomedical research. Understanding the<br />
objectives, eligibility requirements, review criteria, and process in selecting and applying for these funding opportunities is central<br />
to the submission <strong>of</strong> a competitive proposal. This presentation will focus on the general principles that apply to these mechanisms<br />
and how to navigate the intricacies <strong>of</strong> the NIH to identify the best options for your career stage and scientiic expertise.<br />
Eric A. Pierce, MD, PhD<br />
Funding from Foundations: Perspectives from the Foundation Fighting Blindness<br />
The Foundation Fighting Blindness (FFB) is the largest non-governmental supporter <strong>of</strong> research related to inherited retinal<br />
degenerations. The goal <strong>of</strong> this presentation is to describe the types <strong>of</strong> research funding available from FFB, which range from<br />
Career Development Awards to Individual Investigator Awards to Research Center grants. The grant application process and review<br />
processes will be described, with an emphasis on practical advice about preparing competitive proposals for foundations in general,<br />
using FFB as an example.<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013<br />
41
<strong>Program</strong> Schedule, Wednesday, May 15, 2013<br />
Wednesday, May 15, 2013<br />
Education Session 124<br />
1:15 pm - 2:45 pm<br />
ROOM: 150 ABC<br />
Topical Review: Synthetic <strong>Gene</strong> Delivery<br />
An overview <strong>of</strong> different philosophies <strong>of</strong> synthetic delivery systems for nucleic acids. The irst talk will address silk elastin-like proteins for<br />
gene delivery and how they can be combined with virus for an enhanced hybrid gene delivery vehicle. The second talk will focus on “smart”<br />
pH-sensitive polymeric carriers for therapeutic delivery <strong>of</strong> nucleic acids. The inal talk will examine cell-penetrating peptides for retinal gene<br />
delivery.<br />
CHAIR: James Yockman, PhD<br />
SPEAKERS<br />
Hamid Ghandehari, PhD<br />
Recombinant Polymers for <strong>Gene</strong> Delivery<br />
Polymeric systems have shown utility in gene delivery. They can form complexes with nucleic acids or be used as matrices for<br />
controlled release. Majority <strong>of</strong> polymers used for such purposes are synthesized by chemical strategies that result in limited control<br />
over polymer structure and therefore function. Recombinant polymers provide exquisite control over backbone structure. This talk<br />
will summarize the use <strong>of</strong> recombinant polymers for gene delivery applications.<br />
You Han Bae, PhD<br />
pH-Sensitive Polymeric Carrier for Nucleic Acid Delivery<br />
The journey <strong>of</strong> a gene carrier from an administration site to target individual cells encounters a series <strong>of</strong> formidable barriers in<br />
the blood compartment, when crossing vascular walls, and while penetrating extracellular matrix. <strong>Cell</strong> uptake and intracellular<br />
traficking steps then follow, which have been major foci in carrier development and in vitro tests. The carrier may require<br />
switching surface property depending on environmental conditions during the voyage for higher probability <strong>of</strong> reaching its target<br />
cell and action site. The potential roles <strong>of</strong> pH-responsive polymers, particularly weakly anionic polysulfonamide and cationic<br />
poly(L-histidine), in this challenging journey will be discussed.<br />
Rajendra Kumar-Singh, PhD<br />
Pegylated Peptide-DNA Nanoparticles Delay the Onset <strong>of</strong> Retinal Degeneration in Mouse Models <strong>of</strong> Retinitis<br />
Pigmentosa<br />
We have developed a novel peptide referred to as ‘Peptide for Ocular Delivery’ or POD that can penetrate cells and tissues in vivo<br />
within 5 minutes. When pegylated, POD can compact DNA and enable marker or therapeutic gene delivery to retina in vivo. These<br />
nanoparticles are approximately 150 nm in size and can enable therapeutic transgene expression in vivo for more than 70 days.<br />
POD-DNA nanoparticles can reduce apoptosis and delay retinal degeneration in a mouse model <strong>of</strong> retinitis pigmentosa. POD has<br />
potential therapeutic applications in ocular as well as non ocular organ systems.<br />
Education Session 125<br />
1:15 pm - 2:45 pm<br />
ROOM: 151 ABCG<br />
Topical Review: The Ins and Outs <strong>of</strong> Integration Deicient Lentiviral Vectors (IDLV’s)<br />
The Ins and Outs <strong>of</strong> Integration Deicient Lentiviral Vectors (IDLV’s)”, will focus on the development and utilization <strong>of</strong> IDLV’s in preclinical<br />
trials. The session comprises three presentations. The irst is an introductory presentation, which will concentrate on the molecular<br />
mechanisms involved in the production and regulation <strong>of</strong> gene expression from IDLV’s. The second presentation will focus on illegitimate<br />
integration <strong>of</strong> IDLV’s, and the third presentation will describe the development <strong>of</strong> a novel IDLV system for cancer immunotherapy.<br />
CHAIR: Tal Kafri, MD, PhD<br />
SPEAKERS<br />
Tal Kafri, MD, PhD<br />
The Basics <strong>of</strong> Integration Defective Lentiviral Vectors<br />
This introductory presentation will focus on the molecular mechanism involved in the generation <strong>of</strong> integration defective lentiviral<br />
vectors (IDLV’s) and the molecular assays employed to determine the eficacy and safety <strong>of</strong> the currently used IDLV gene delivery<br />
systems. In addition the presentation will outline advancement in IDLV design, which improve IDLV gene expression and reduce<br />
illegitimate integration.<br />
42<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013
<strong>Program</strong> Schedule, Wednesday, May 15, 2013<br />
Manfred Schmidt, PhD<br />
Clonality and Biosafety Aspects <strong>of</strong> Illegitimate Integration<br />
Integrating retroviral vectors have been used successfully in clinical applications. With the occurrence <strong>of</strong> integration associated<br />
severe side effects, enhanced efforts have been set for the clinical translation <strong>of</strong> integrase deicient viral vectors to guarantee<br />
improved biosafety. In this presentation, the focus is set on studies performed for assessing frequency, proile and mechanisms <strong>of</strong><br />
(illegitimate) integration.<br />
Scott H. Robbins, PhD<br />
Development <strong>of</strong> an ID-LV as a <strong>Gene</strong> Delivery Platform for Immunotherapy Applications<br />
One <strong>of</strong> the most fundamental challenges for the design <strong>of</strong> next generation vaccines is the generation <strong>of</strong> antigen-speciic CD8 T<br />
cell responses. The direct administration <strong>of</strong> lentivector-based vaccines genetically encoding antigen is proving to be an effective<br />
approach to elicit CD8 T cell responses in pre-clinical animal models. The ability to alter the tropism <strong>of</strong> lentiviral vectors via<br />
pseudotyping and other mechanisms makes them attractive as a platform technology on which to base product candidates for<br />
clinical applications such as antigen-directed immunotherapy and vaccines. Examples <strong>of</strong> the functionality <strong>of</strong> integration-competent<br />
and -deicient lentiviral vectors as antigen delivery vehicles in pre-clinical settings will be provided as well as an example <strong>of</strong> the<br />
design <strong>of</strong> a dendritic cell-directed integration-deicient lentiviral vector that is currently under development as an antigen-directed<br />
cancer immunotherapy (a “cancer vaccine”).<br />
Wednesday, May 15, 2013<br />
Oral Abstract Session 130<br />
3:15 pm - 5:15 pm<br />
ROOM: BALLROOM A<br />
AAV Vector Development & Application<br />
CO-CHAIRS: Mavis Agbandje-McKenna, PhD and Hiroyuki Nakai, MD, PhD<br />
(Abstracts 1 through 8; see page 87)<br />
Oral Abstract Session 131<br />
3:15 pm - 5:15 pm<br />
ROOM: BALLROOM B<br />
Cancer - Immunotherapy I<br />
CO-CHAIRS: Malcolm K. Brenner, MD, PhD and John J. Nemunaitis, MD<br />
(Abstracts 9 through 16; see pages 87-88)<br />
Oral Abstract Session 132<br />
3:15 pm - 5:15 pm<br />
ROOM: 150 DEFG<br />
Cancer - Oncolytic Viruses<br />
CO-CHAIRS: David Kirn, MD and Stephen J. Russell, MD, PhD<br />
(Abstracts 17 through 24; see page 88)<br />
Oral Abstract Session 133<br />
3:15 pm - 5:15 pm<br />
ROOM: BALLROOM C<br />
Advancing the Science <strong>of</strong> <strong>Gene</strong> <strong>Therapy</strong> for Hematologic Diseases<br />
CO-CHAIRS: Arnab Ghosh, MD, PhD and Donald B. Kohn, MD<br />
(Abstracts 25 through 32; see pages 88-89)<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013<br />
43
<strong>Program</strong> Schedule, Wednesday, May 15, 2013<br />
Wednesday, May 15, 2013<br />
Oral Abstract Session 134<br />
3:15 pm - 5:15 pm<br />
ROOM: BALLROOM D<br />
Neurological Disorders: Monogeneic Disorders<br />
CO-CHAIRS: Raymond T. Bartus, PhD and Nicholas Boulis, MD<br />
(Abstracts 33 through 40; see page 89)<br />
Oral Abstract Session 135<br />
3:15 pm - 4:15 pm<br />
ROOM: 151 ABCG<br />
Chemical and Molecular Conjugates<br />
CO-CHAIRS: Young Jik Kwon, PhD and Assem Ziady, PhD<br />
(Abstracts 41 through 44; see page 90)<br />
Oral Abstract Session 136<br />
3:15 pm - 4:15 pm<br />
ROOM: 150 ABC<br />
Infectious Diseases and Vaccines<br />
CO-CHAIRS: Renata Stripecke, PhD and Grant D. Trobridge, PhD<br />
(Abstracts 45 through 48; see page 90)<br />
Oral Abstract Session 137<br />
4:20 pm - 5:20 pm<br />
ROOM: 151 ABCG<br />
<strong>Cell</strong> Processing and Vector Production<br />
CO-CHAIRS: Adrian P. Gee, PhD and Isabelle Riviere, PhD<br />
(Abstracts 49 through 52; see page 90)<br />
Oral Abstract Session 138<br />
4:20 pm - 5:20 pm<br />
ROOM: 150 ABC<br />
Sensory (Ophthalmic & Auditory) <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong><br />
CO-CHAIRS: Alberto Auricchio, MD and Tonia S. Rex, PhD<br />
(Abstracts 53 through 56; see page 91)<br />
Exhibit Hall Welcome Reception<br />
5:15 pm - 7:15 pm<br />
ROOM: EXHIBIT HALL C/D<br />
44<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013
<strong>Program</strong> Schedule, Wednesday, May 15, 2013<br />
Workshop 140<br />
7:30 pm - 9:30 pm<br />
Room: 150 DEFG<br />
Commercialization Workshop<br />
CO-CHAIRS: Estuardo Aguilar-Cordova and Brendan Lee, MD, PhD<br />
SPEAKERS<br />
Peter Kolchinsky<br />
What makes Investors want to Invest<br />
Nick Leschly<br />
Who Utilizes Early Phase Resources<br />
Wednesday, May 15, 2013<br />
Fabrizia Bignami, PhD<br />
What is Big Pharma Looking for in <strong>Gene</strong> or <strong>Cell</strong> <strong>Therapy</strong><br />
Suzannah Sundby, JD<br />
What IP Issues are Relevant for Successfully Commercializing <strong>Gene</strong> and <strong>Cell</strong> Therapies<br />
<strong>Gene</strong> and cell therapies are different from classic small molecules in that intellectual property issues can evolve and become<br />
increasingly complex as scaling up from the laboratory setting and commercialization proceeds. What effect do modiications<br />
and optimizations to vector designs and manufacturing processes, including cell isolation and culturing techniques, have on one’s<br />
right to make, use, and sell the intellectual property Do you still own the rights to the intellectual property Will the intellectual<br />
property rights <strong>of</strong> another prevent you from commercializing your own product Ms. Sundby will discuss IP in the cell and gene<br />
therapy space with particular attention paid to what questions an investigator or institution should consider prior to investing<br />
further resources in pipeline development.<br />
New Member Welcome Reception<br />
7:30 pm - 8:30 pm<br />
ROOM: 255 B<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013<br />
45
<strong>Program</strong> Schedule, Thursday, May 16, 2013<br />
George Stamatoyannopoulos Lecture 200<br />
8:30 am - 9:35 am<br />
ROOM: BALLROOM E-J<br />
George Stamatoyannopoulos Lecture<br />
CHAIR<br />
SPEAKER<br />
Thursday, May 16, 2013<br />
David W. Russell, MD, PhD<br />
Mario R. Capecchi, PhD<br />
Modeling <strong>of</strong> Human Disease in the Mouse in the 21st Century: From Cancer to Neuropsychiatric Disorders<br />
<strong>Gene</strong> targeting allows the designed modiication <strong>of</strong> any gene in the mouse genome. Since genes impact all biological phenomena<br />
this methodology can be used to study any biological phenomena common to mammals in the mouse. We are using it to model<br />
human disease in the mouse. The models can be used to analyze the pathology <strong>of</strong> the disease at a level not feasible in humans and<br />
as a platform for the development <strong>of</strong> new therapeutic protocols. I will discuss modeling <strong>of</strong> a neuropsychiatric disorder, obsessive<br />
compulsive (OCD) spectrum disorder in the mouse. These studies provide the unexpected conclusion that microglia, the immune<br />
cells <strong>of</strong> the brain derived from bone marrow, are controlling behavior in the mouse.<br />
Exhibit Hall C<strong>of</strong>fee Social<br />
9:35 am - 10:30 am<br />
ROOM: EXHIBIT HALL C/D<br />
Scientific Symposium 210<br />
10:30 am - 12:30 pm<br />
ROOM: 151 ABCG<br />
50 Years <strong>of</strong> Research in Alpha-1 Antitrypsin<br />
CHAIR: Terence R. Flotte, MD<br />
SPEAKERS<br />
Robert A. Sandhaus, MD, PhD<br />
Historical Review <strong>of</strong> the Past 50 Years <strong>of</strong> Research on Alpha-1 Antitrypsin Disorder<br />
First described 50 years ago, Alpha-1 Antitrypsin Deiciency (AATD) was soon associated with the development <strong>of</strong> both lung and<br />
liver diseases. The rapidity with which the biochemical, pathophysiologic, epidemiologic, and therapeutic advances followed this<br />
initial discovery has been astounding, even by today’s standards. In spite <strong>of</strong> this, there is still much to be learned about the alpha-1<br />
antitrypsin protein and its roles in health and disease.<br />
Ronald G. Crystal, MD<br />
<strong>Gene</strong> <strong>Therapy</strong> for Alpha 1-antitrypsin Deiciency with AAV Intrapleural Administration<br />
a1-antitrypsin (a1AT) deiciency, caused by SERPINA1 gene mutations, results in low a1AT serum levels and development <strong>of</strong> earlyonset<br />
panacinar emphysema, reducing life span by 15 to 20 years. Current treatment is weekly intravenous administration <strong>of</strong> a1AT<br />
puriied from pooled human plasma. Adeno-associated (AAV)-mediated delivery <strong>of</strong> a1AT is an ideal strategy for gene therapy for<br />
the lung manifestations <strong>of</strong> a1AT deiciency because AAV vectors mediate sustained transgene expression. We have established a<br />
novel, highly eficient strategy for gene therapy for a1AT deiciency based on intrapleural administration <strong>of</strong> AAV serotype rh.10<br />
coding for human a1AT. The advantage this approach <strong>of</strong>fers is proximity <strong>of</strong> vector expression to the site <strong>of</strong> action. Pre-clinical<br />
studies comparing intrapleural administration <strong>of</strong> 25 AAV serotype vectors demonstrated that the highest a1AT levels were achieved<br />
with AAVrh.10, a serotype that is derived from a non-human primate, minimizing the challenge <strong>of</strong> pre-existing human anti-vector<br />
immunity limiting persistent gene expression. Safety studies in mice and non-human primates have shown this strategy is safe<br />
and will provide sustained expression a1AT. Our overall hypothesis is that a single intrapleural administration <strong>of</strong> an AAVrh.10<br />
gene transfer vector coding for the human a1AT cDNA will provide sustained therapeutic levels in the lung suficient to treat a1AT<br />
deiciency.<br />
46<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013
<strong>Program</strong> Schedule, Thursday, May 16, 2013<br />
Christian Mueller, PhD<br />
MicroRNA’s and Alpha-1 Antitrypsin Deiciency<br />
Terence R. Flotte, MD<br />
Advances in Clinical <strong>Gene</strong> <strong>Therapy</strong> Trials for Alpha-1 Anti-trypsin Deiciency<br />
Scientific Symposium 211<br />
10:30 am - 12:30 pm<br />
ROOM: BALLROOM C<br />
Clinical Updates <strong>of</strong> Stem <strong>Cell</strong> Therapies<br />
CO-CHAIRS: Jane S. Lebkowski, PhD and Eirini P. Papapetrou, MD, PhD<br />
SPEAKERS<br />
Michele De Luca, MD<br />
Corneal Regeneration By Cultured Limbal Stem <strong>Cell</strong>s<br />
Adult stem cells are cells with a high capacity for self-renewal that can produce terminally differentiated progeny. Stem cells<br />
generate an intermediate population <strong>of</strong> committed progenitors, <strong>of</strong>ten referred to as transit amplifying (TA) cells, that terminally<br />
differentiate after a limited number <strong>of</strong> cell divisions. Human keratinocyte stem cells are clonogenic and are known as holoclones.<br />
Human corneal stem cells are segregated in the limbus while limbal-derived TA cells form the corneal epithelium. Cultivated limbal<br />
stem cells generate sheets <strong>of</strong> corneal epithelium suitable for clinical application. We report long-term (up to 15 years) clinical<br />
results obtained in an homogeneous group <strong>of</strong> patients presenting with corneal opaciication and visual loss due to chemical burndependent<br />
limbal stem cell deiciency. The corneal epithelium and the visual acuity <strong>of</strong> these patients have been restored by grafts <strong>of</strong><br />
autologous cultured limbal keratinocytes. In post hoc analyses, success was associated with the percentage <strong>of</strong> p63-bright holocloneforming<br />
stem cells in culture, but not with total number <strong>of</strong> clonogenic cells, colony size or growth rate <strong>of</strong> the cultures. Graft failure<br />
was also associated with the type <strong>of</strong> initial ocular damage and postoperative complications.<br />
Joshua Hare, MD<br />
<strong>Cell</strong> <strong>Therapy</strong> for Chronic Ischemic Heart Disease from Concept to Clinic<br />
Thursday, May 16, 2013<br />
Brian W. Bigger, PhD<br />
Hematopoietic Stem <strong>Cell</strong> <strong>Gene</strong> <strong>Therapy</strong> for Neurological Diseases<br />
Haematopoietic stem cell transplantation (HSCT) has traditionally been used to treat a select group <strong>of</strong> single gene neurological<br />
diseases, with particular success in the neurodegenerative lysosomal disease Hurler syndrome. In many lysosomal diseases<br />
exogenous enzyme can be taken up by, and cross-correct affected cells, but the blood brain barrier excludes enzyme from the<br />
brain. Neurological correction after HSCT relies on monocyte/microglial donor cell traficking into the brain to deliver enzyme, and<br />
enzyme dose is thought to be one <strong>of</strong> the factors limiting HSCT to a relatively narrow range <strong>of</strong> neurological indications. Autologous<br />
haematopoietic stem cell gene therapy, using retroviral or lentiviral vectors to overexpress enzyme in ex vivo transduced and<br />
transplanted cells has been used to great effect in X-SCID and other immunodeiciencies and more recently has been adopted as a<br />
potential treatment for neurological lysosomal and related diseases. In this talk the initial clinical trial results in this ield will be<br />
discussed, together with the current challenges faced by this therapeutic approach, what is being done to address them and where<br />
the future for this exciting therapy lies.<br />
Gary K. Steinberg, MD, PhD<br />
Stem <strong>Cell</strong> <strong>Therapy</strong> for Stroke<br />
No treatment currently exists to restore lost brain function after stroke. Animal studies have demonstrated that post-ischemic<br />
intraparenchymal, intravenous and intra-arterial administration <strong>of</strong> various types <strong>of</strong> stem cells can improve neurologic outcome.<br />
Although some integration <strong>of</strong> neural stem cells into the peri-infarct brain has been shown, the main mechanisms underlying<br />
recovery <strong>of</strong> function are likely secretion <strong>of</strong> trophic factors and immunomodulation that enhance endogenous plasticity and<br />
recovery. These native repair processes include axonal sprouting, dendritic branching, neovascularization, neurogenesis,<br />
gliogenesis, synaptogenesis and decreased inlammation. To date 11 trials <strong>of</strong> cellular therapy for stroke have been completed<br />
and more than 15 additional trials are underway. This talk will briely review the experimental basis leading to the clinical trials,<br />
and focus on summarizing the clinical results <strong>of</strong> published and ongoing stroke trials using neural, bone marrow derived and<br />
other stem cells. Unresolved issues will be discussed, including the best cell source and type, optimal route <strong>of</strong> delivery, timing <strong>of</strong><br />
transplantation and patient selection.<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013<br />
47
<strong>Program</strong> Schedule, Thursday, May 16, 2013<br />
Scientific Symposium 212<br />
10:30 am - 12:30 pm<br />
ROOM: BALLROOM D<br />
Frontiers <strong>of</strong> Cardiovascular <strong>Gene</strong> and <strong>Cell</strong> <strong>Therapy</strong><br />
CO-CHAIRS: David A. Dichek, MD and Michael J. Passineau, PhD<br />
SPEAKERS<br />
Charles R. Bridges, MD, ScD<br />
Challenges <strong>of</strong> <strong>Gene</strong> and <strong>Cell</strong> <strong>Therapy</strong> to the Heart<br />
In this presentation I will describe the main challenges <strong>of</strong> current cardiac cell and gene therapy with an emphasis on challenges to<br />
eficient delivery. I will give a detailed comparison <strong>of</strong> available gene and cell delivery techniques. I will discuss documented and<br />
potential complications <strong>of</strong> cell injections and gene transfer such as the risk <strong>of</strong> arrhythmias, incidence <strong>of</strong> coronary restenosis and<br />
malignancy. <strong>Final</strong>ly, I will focus on the most recent results <strong>of</strong> cardiac cell and gene therapy trials and the major lessons drawn from<br />
these trials.<br />
Thursday, May 16, 2013<br />
Andrew H. Baker, PhD<br />
Vascular MiRNA and Pulmonary Hypertension<br />
MicroRNAs (miRNAs) are short non-coding RNAs which post-transcriptionally regulate gene expression. miRNAs are important<br />
in governing cell differentiation, development and disease. We have investigated the role or individual miRNAs in pathological<br />
vascular remodeling, as well as the potential therapeutic value <strong>of</strong> manipulating selected miRNA in vivo. I will discuss these<br />
indings with emphasis on vascular remodeling associated with both venous and arterial remodeling, i.e. vein graft failure and the<br />
development <strong>of</strong> pulmonary arterial hypertension.<br />
Marc Penn, MD, PhD<br />
<strong>Gene</strong> <strong>Therapy</strong> for Heart Failure<br />
The success <strong>of</strong> establishing antegrade low in patients with acute myocardial infarction has led to signiicant advancements in the<br />
survival <strong>of</strong> patients with acute ischemic events <strong>of</strong> the myocardium. Unfortunately this success had led to a signiicant increase in<br />
the prevalence <strong>of</strong> heart failure. To address this need several investigators have turned to gene therapy as a strategy to either repair,<br />
restore or optimize cardiac function. Excitingly these strategies have entered clinical trials with encouraging results. We will discuss<br />
the strategies currently under investigation as well as those that maybe entering the clinical realm in the future.<br />
Doris A. Taylor, PhD<br />
Personalizing <strong>Cell</strong> <strong>Therapy</strong>: Initial CCTN Experience<br />
Scientific Symposium 213<br />
10:30 am - 12:30 pm<br />
ROOM: BALLROOM B<br />
New Paradigms for Immune <strong>Cell</strong> Therapies<br />
CO-CHAIRS: Cynthia Dunbar, MD and Paris Margaritis, DPhil<br />
SPEAKERS<br />
David L. Porter, MD<br />
Chimeric Antigen Receptor Therapies for Lymphoid Malignancies<br />
This presentation will describe the biological activity and outcomes <strong>of</strong> patients treated with CART19 (CTL019) cells; these are<br />
autologous T cells collected by leukapheresis and transduced with a lentivirus encoding anti-CD19 scFv linked to 4-1BB and CD3-z<br />
signaling domains. <strong>Gene</strong>-modiied T cells can then target CD19+ cells. <strong>Cell</strong>s are expanded and activated ex-vivo by exposure to anti-<br />
CD3/CD28 beads prior to infusion. Patients are treated for advanced and/or refractory CLL or ALL. We previously reported marked<br />
in-vivo expansion, long-term persistence and effective anti-tumor activity <strong>of</strong> CART19 cells in 3 patients with CLL with relatively<br />
short follow up (Porter, et al NEJM 2011; Kalos et al Sci Trans Med 2011). Results from the clinical trials will be updated with longterm<br />
follow-up and include presentation <strong>of</strong> data from a larger cohort <strong>of</strong> patients. We show that these cells can undergo massive<br />
in-vivo expansion, persist for more than 2 years and remain biologically active, and can induce potent and sustained responses for<br />
patients with advanced, refractory and high risk CLL and relapsed, refractory ALL.<br />
Maria-Grazia Roncarolo, MD, PhD<br />
<strong>Cell</strong> <strong>Therapy</strong> with Regulatory T <strong>Cell</strong>s to Dampen Undesired Immune Responses<br />
48<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013
<strong>Program</strong> Schedule, Thursday, May 16, 2013<br />
Nicholas Restifo, MD<br />
Curative Cancer Immunotherapy Using Stem cell-like T cells<br />
T lymphocytes play a critical role in the immune response against infectious disease and cancer. The recently identiied T memory<br />
stem cells (TSCM) are endowed with the stem cell–like attributes <strong>of</strong> self-renewal capacity and multipotency. These qualities make<br />
TSCM an attractive subset to employ in adoptive immunotherapies for cancer because they might overcome current limitations,<br />
including ineficient T-cell engraftment, poor persistence, and inability to mediate a prolonged immune attack. TSCM display robust<br />
proliferative and survival capacities and were superior to other memory T-cell subsets in causing regression <strong>of</strong> large, established<br />
tumors in preclinical models. Protocols to facilitate translation <strong>of</strong> TSCM into the clinic will be discussed.<br />
Jeffrey S. Miller, MD<br />
Educating Natural Killer <strong>Cell</strong>s to Kill Cancer<br />
Natural killer (NK) cells express a number <strong>of</strong> activating and inhibitory receptors to determine their function through a process<br />
called NK cell education. We have shown that adoptive transfer <strong>of</strong> haploidentical NK cells can induce remissions in some<br />
patients with refractory acute myeloid leukemia (AML). Limitations <strong>of</strong> this therapy include host immune rejection, how to<br />
maximize the persistence and expansion <strong>of</strong> donor NK cells and overcoming regulatory T-cell suppression. Lastly, donor Killer-cell<br />
Immunoglobulin-like Receptor (KIR) genes may inluence NK cell activity to enhance anti-tumor eficacy. Understanding the role <strong>of</strong><br />
KIR and other receptors may allow us to exploit adoptive transfer <strong>of</strong> NK cells for therapeutic use in patients with advanced cancers.<br />
Scientific Symposium 214<br />
10:30 am - 12:30 pm<br />
ROOM: 150 DEFG<br />
Recent Advancements in the Non-Viral Delivery <strong>of</strong> Nucleic Acid Based Drugs<br />
CO-CHAIRS: Ian MacLachlan, PhD and Olivia Merkel, PhD<br />
SPEAKERS<br />
Daniel G. Anderson, PhD<br />
Combinatorial Development <strong>of</strong> Synthetic Nucleic Acid Delivery Systems<br />
Thursday, May 16, 2013<br />
Jianjun Cheng, PhD<br />
a-Helical Polypeptides for Non-Viral <strong>Gene</strong> Delivery<br />
We designed a class <strong>of</strong> helical, cationic charged polypeptides via the ring-opening polymerization <strong>of</strong> vinyl benzyl glutamic acid<br />
N-carboxyanhydrides. These materials have unusual helical stability and display excellent cell penetrating property outperforming<br />
TAT peptide. By controlling the chemical, morphological and self-assembly structures <strong>of</strong> these side chain charged, helical<br />
polypeptides, we identiied a few materials that showed excellent gene/siRNA delivery eficiency in vitro and in vivo.<br />
Thomas Knotts, PhD<br />
Protein Structure and How it Changes in Association with Surfaces<br />
Proteins/surface interactions are important in a variety <strong>of</strong> existing and emerging biomedical techniques and devices. It has been<br />
known for decades that surfaces can drastically change the structure <strong>of</strong> attached or adsorbed proteins, but a priori prediction <strong>of</strong> the<br />
changes is not currently possible. In an attempt to develop some predictive capabilities with respect to protein/surface interactions,<br />
we have used advanced molecular simulation to test several theories as to how protein folding mechanisms and stabilities are<br />
affected by surfaces. This talk will present many <strong>of</strong> these results, and more importantly, design heuristics that can be used to ensure<br />
proteins <strong>of</strong> interest are stable, and functional on surfaces. Taken as a whole, the results <strong>of</strong>fer compelling evidence that the rational<br />
design <strong>of</strong> technologies involving proteins interacting with surfaces is possible.<br />
Hamid Ghandehari, PhD<br />
Silk-elastinlike Polymers for Matrix-mediated <strong>Gene</strong> Delivery<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013<br />
49
<strong>Program</strong> Schedule, Thursday, May 16, 2013<br />
Scientific Symposium 215<br />
10:30 am - 12:30 pm<br />
ROOM: BALLROOM A<br />
The GTRP: Perspectives <strong>of</strong> an Investigator and the <strong>Program</strong> Cores<br />
CO-CHAIRS: Cheryl L. McDonald, MD and Sonia I. Skarlatos, PhD<br />
SPEAKERS<br />
Thursday, May 16, 2013<br />
Daniel Rader, MD<br />
How the GTRP Facilitates Translational Research: An Investigator’s Perspective<br />
Atherosclerosis is a complex and heritable disease intimately inluenced by lipoprotein metabolism. Unbiased genome-wide<br />
association studies (GWAS) have identiied dozens <strong>of</strong> novel loci robustly associated with both plasma lipid traits and atherosclerotic<br />
coronary artery disease (CAD). Several <strong>of</strong> the genes at these loci could represent potential novel therapeutic targets for the<br />
treatment <strong>of</strong> inherited dyslipidemias and the prevention or treatment <strong>of</strong> CAD. Adeno-associated viral (AAV) vectors are a valuable<br />
tool for effective and stable in vivo expression <strong>of</strong> cDNAs in relevant animal models <strong>of</strong> disease. Through support from the GTRP, we<br />
have used AAV8 vectors to express a number <strong>of</strong> potential therapeutic genes at lipid/CAD GWAS loci in the livers <strong>of</strong> ‘humanized’<br />
mice. This systematic approach has not only yielded important new biological information about the effects <strong>of</strong> novel GWAS genes on<br />
lipid metabolism and atherosclerosis, but also has placed a number <strong>of</strong> new ‘therapeutic’ genes on the map. This classic translational<br />
approach moving from unbiased human genetics to mechanisms and therapeutic effects in model systems is made possible by the<br />
support <strong>of</strong> the GTRP.<br />
Sonia I. Skarlatos, PhD<br />
GTRP: Facilitating Bench to Bedside<br />
The purpose <strong>of</strong> the GTRP is to provide researchers with resources that are critical to advancing investigational gene therapy<br />
products into clinical testing. Services along the product development spectrum – ranging from production <strong>of</strong> preclinical vectors to<br />
assistance with early-phase clinical trials – are <strong>of</strong>fered through the <strong>Program</strong> at no cost to the investigator. The <strong>Program</strong> primarily<br />
supports research in heart, lung, and blood diseases.<br />
James M. Wilson, MD, PhD<br />
A Toolkit <strong>of</strong> Vectors for <strong>Gene</strong> <strong>Therapy</strong><br />
The Preclinical Vector Core provides high quality viral vectors based on adeno-associated virus (AAV), adenovirus and lentivirus<br />
as well as non-viral vectors for basic research directed toward clinical application. Also <strong>of</strong>fered are a host <strong>of</strong> other services such as<br />
consultation on vector construction and experimental design, quality control services appropriate for vectors destined for use in<br />
small or large animal models, immunology testing and preclinical biodistribution analysis. Of particular importance to NHLBI GTRP<br />
investigators has been distribution through the program <strong>of</strong> novel serotype AAV vectors which have become the gold standards in<br />
the ield for mediating gene delivery to heart, lung and liver. In addition to <strong>of</strong>fering a variety <strong>of</strong> vector platforms, the Preclinical<br />
Core rapidly transitions new technologies from key laboratories for distribution as well as develops reagents and maintains a large<br />
inventory <strong>of</strong> vectors in order to provide GTRP participants with a veritable vector toolbox for advancing preclinical studies toward<br />
clinical translation.<br />
Janet Benson, PhD, DABT<br />
From Mice to Man: IND-enabling Preclinical Studies<br />
The mission <strong>of</strong> the Pharmacology/Toxicology (Pharm/Tox) Core is to aid investigators in the design and implementation <strong>of</strong> INDenabling<br />
preclinical safety and biodistribution studies. This is <strong>of</strong>ten one <strong>of</strong> the most challenging and costly steps in the translational<br />
process. The Pharm/Tox Core scientists work closely with the Investigator(s), the GTRP Core providing the vector (if applicable),<br />
and the GTRP Clinical Coordinating Center to facilitate development <strong>of</strong> the IND packages and the pre-clinical study protocol. The<br />
GTRP Pharm/Tox Core provides dosing, endpoint evaluations including but not limited to: clinical observations, clinical pathology,<br />
histopathology, safety pharmacology, vector biodistribution (qPCR), immunology endpoints, as well as assessments <strong>of</strong> transgene<br />
expression. Upon completion <strong>of</strong> the study, the Pharm/Tox Core staff prepares an audited inal report for inclusion in the IND<br />
submission to the FDA.<br />
50<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013
<strong>Program</strong> Schedule, Thursday, May 16, 2013<br />
J. Fraser Wright, PhD<br />
AAV Vectors for Clinical Trials: Challenges, and Strategies for Success<br />
Recombinant viral vectors based on adeno-associated viruses (AAV) have shown tremendous promise for clinical therapeutic<br />
gene delivery, and an acceleration <strong>of</strong> gene-based product development programs has been catalyzed by recent clinical successes.<br />
This presentation will describe strategies for advancing AAV-based investigational product chemistry, manufacturing and<br />
controls to effectively support the translational pathway from IND-supporting pre-clinical studies through clinical studies. An<br />
important requirement for successful translational research includes well deined manufacturing processes capable <strong>of</strong> reliable and<br />
reproducible yields vector <strong>of</strong> high purity, potency and safety. Another critical requirement is the set <strong>of</strong> analytical methods required<br />
to ensure comprehensive investigational product characterization and quality control testing. In this presentation the vector<br />
manufacturing processes and characterization methods used to support AAV vector-based translational research supported by<br />
GTRP will be presented.<br />
Kenneth Cornetta, MD<br />
Lentiviral Vectors: Can We Move Past Phase I/II<br />
Lentiviral vectors have advanced into early Phase clinical trials in the setting <strong>of</strong> genetic diseases, cancer, and other applications<br />
where integration and long-term expression is required. Compared to traditional integrating vectors, the methods used in<br />
manufacturing lentiviral vectors are well suited to rapid implementation into Phase I clinical trials. Unfortunately, this property can<br />
present challenges to generating a licensed product. This talk will discuss the current status <strong>of</strong> lentiviral vectors and highlight the<br />
challenges <strong>of</strong> moving these vectors from research tools to an FDA approved agent.<br />
Susan B. Sepelak, MS, MSM<br />
Regulatory Requirements: Navigation is Easier with Assistance<br />
The GTRP Clinical Coordinating Center provides assistance to investigators in navigating the regulatory processes in translational<br />
gene therapy research. For approved requests, assistance may be provided directly to the Investigator - assistance ranging from<br />
help with scheduling pre-preIND meetings with the FDA through organization, compilation and submission <strong>of</strong> IND applications.<br />
Regulatory support with clinical site-related issues may also be requested by Investigators to help site staff and Study Coordinators<br />
prepare for study implementation and manage ongoing regulatory requirements. Investigators who receive clinical trial funding<br />
assistance through the GTRP may, additionally, apply for support with site selection, study monitoring, and preparation <strong>of</strong> NHLBIrequired<br />
study reports.<br />
Thursday, May 16, 2013<br />
Lunch Break (On Own - Not Provided; Pre-ordered boxed lunches available for pick up at Registration Counter)<br />
12:30 pm - 2:00 pm<br />
Meet-the-Investigator Lunch Session 220<br />
12:30 pm - 2:00 pm<br />
ROOM: 251 E<br />
New Frontiers – Hemoglobinopathies<br />
SPEAKERS<br />
Donald B. Kohn, MD<br />
Punam Malik, MD, PhD<br />
Meet-the-Investigator Lunch Session 221<br />
12:30 pm - 2:00 pm<br />
MEET AT THE SOUTH ENTRANCE OF THE SALT PALACE CONVENTION CENTER<br />
Huntsman Cancer Institute Tour<br />
Boxed lunches will be available at the South Entrance shortly before departure to the Huntsman Cancer Institute.<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013<br />
51
<strong>Program</strong> Schedule, Thursday, May 16, 2013<br />
Oral Abstract Session 230<br />
2:00 pm - 4:00 pm<br />
ROOM: BALLROOM A<br />
Clinical <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong><br />
CO-CHAIRS: Carl H. June, MD and Harry L. Malech, MD<br />
(Abstracts 57 through 62; see page 92)<br />
Oral Abstract Session 231<br />
2:00 pm - 4:00 pm<br />
ROOM: BALLROOM B<br />
RNA Virus Vectors<br />
CO-CHAIRS: Brian D. Brown, PhD and Jennifer E. Adair, PhD<br />
(Abstracts 63 through 70; see pages 92-93)<br />
Thursday, May 16, 2013<br />
Oral Abstract Session 232<br />
2:00 pm - 4:00 pm<br />
ROOM: BALLROOM C<br />
AAV Vector Biology<br />
CO-CHAIRS: Casey A. Maguire, PhD and David V. Schaffer, PhD<br />
(Abstracts 71 through 78; see page 93)<br />
Oral Abstract Session 233<br />
2:00 pm - 4:00 pm<br />
ROOM: BALLROOM D<br />
Stem <strong>Cell</strong> Engineering and <strong>Therapy</strong><br />
CO-CHAIRS: Hans-Peter Kiem, MD, FACP and Eirini P. Papapetrou, MD, PhD<br />
(Abstracts 80 through 86; Abstract 79 Withdrawn and Abstract 470 moved from Poster Session II; see pages 93-94)<br />
Oral Abstract Session 234<br />
2:00 pm - 4:00 pm<br />
ROOM: 150 DEFG<br />
Musculo-skeletal <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> I<br />
CO-CHAIRS: Charles A. Gersbach, PhD and Louise R. Rodino-Klapac, PhD<br />
(Abstracts 87 through 94; see pages 94-95)<br />
Oral Abstract Session 235<br />
2:00 pm - 3:00 pm<br />
ROOM: 150 ABC<br />
Cardiovascular <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong><br />
CO-CHAIRS: Dongsheng Duan, PhD and Joseph E. Rabinowitz, PhD<br />
(Abstracts 95 through 98; see page 95)<br />
52<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013
<strong>Program</strong> Schedule, Thursday, May 16, 2013<br />
Oral Abstract Session 236<br />
2:00 pm - 3:15 pm<br />
ROOM: 151 ABCG<br />
Physical Methods <strong>of</strong> Delivery<br />
CO-CHAIRS: King F. Kwong, MD and Kevin G. Rice, PhD<br />
(Abstracts 99 through 103; see page 95)<br />
Oral Abstract Session 237<br />
3:05 pm - 4:05 pm<br />
ROOM: 150 ABC<br />
Evolution <strong>of</strong> Pulmonary Treatments<br />
CO-CHAIRS: Uta Griesenbach, PhD and Katherine J. Exc<strong>of</strong>fon, PhD<br />
(Abstracts 104 through 107; Abstract 105 Withdrawn and Abstract 432 moved from Poster Session II; see pages 95-96)<br />
Oral Abstract Session 238<br />
3:20 pm - 4:05 pm<br />
ROOM: 151 ABCG<br />
Immunologic & Host Responses in <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong><br />
CO-CHAIRS: Antonia Follenzi, MD, PhD and Federico Mingozzi, PhD<br />
(Abstracts 108 through 110; see page 96)<br />
Thursday, May 16, 2013<br />
Exhibit Hall Open: Networking Reception & Poster Session I<br />
4:00 pm - 6:00 pm<br />
ROOM: EXHIBIT HALL C/D<br />
AAV Vectors - I<br />
(Abstracts 111-125; see pages 96-97)<br />
Adenovirus and Other DNA Virus Vectors I<br />
(Abstracts 126-138; and Abstract 561 moved from Poster Session III; see pages 97-98)<br />
Physical Methods <strong>of</strong> Delivery<br />
(Abstracts 139-154; see pages 98-99)<br />
Chemical and Molecular Conjugates<br />
(Abstracts 155-165; Abstract 165 has been withdrawn from presentation; see page 99)<br />
Musculo-skeletal <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> I<br />
(Abstracts 166-182; see pages 99-100)<br />
Infectious Diseases and Vaccines<br />
(Abstracts 183-195; see pages 100-101)<br />
Cancer - Immunotherapy I<br />
(Abstracts 197-209; Abstract 196 withdrawn; see pages 101-102)<br />
Cancer-Targeted <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>: Viral Vector / siRNA-Mediated Therapies<br />
(Abstracts 210-221; see pages 102-103)<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013<br />
53
<strong>Program</strong> Schedule, Thursday, May 16, 2013<br />
Sensory (Ophthalmic and Auditory) <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong><br />
(Abstracts 222-232; see page 103)<br />
<strong>Gene</strong> Regulation<br />
(Abstracts 233-246; see page 104)<br />
Fetal and Adult Stem <strong>Cell</strong>s<br />
(Abstracts 247-258; Abstract 252 has been withdrawn from presentation; see pages 104-105)<br />
<strong>Cell</strong> Processing and Vector Production<br />
(Abstracts 259-265; see page 105)<br />
Late Breaking Abstracts I<br />
(Abstracts 677-700; Abstracts 687 and 698 Withdrawn from Presentation; see pages 105-107)<br />
Thursday, May 16, 2013<br />
Canadian Attendee Reception<br />
6:00 pm - 7:00 pm<br />
ROOM: 255 B<br />
Foundation Symposium 240<br />
6:00 pm - 8:00 pm<br />
ROOM: BALLROOM B<br />
<strong>Gene</strong> and <strong>Cell</strong> <strong>Therapy</strong> for Cancer<br />
Supported by the Alliance for Cancer <strong>Gene</strong> <strong>Therapy</strong>.<br />
CO-CHAIRS: Xandra O. Breakeield, PhD and Savio L.C. Woo, PhD<br />
SPEAKERS<br />
Joseph C. Glorioso, III, PhD<br />
Targeted Cancer Therapies<br />
Cancer cell genetic analyses have identiied driver mutations that dysregulate cell proliferation- and differentiation-related<br />
pathways, providing focal points for targeting <strong>of</strong> cancer therapy. At least six targeting strategies have been employed: (i) the use <strong>of</strong><br />
drugs that block different steps in cancer causing signaling cascades leading to the arrest <strong>of</strong> tumor cell growth and the induction <strong>of</strong><br />
senescence, apoptosis and autophagy, (ii) application <strong>of</strong> drugs or immune mediators that attack the tumor microenvironment (iii)<br />
the use <strong>of</strong> antibodies that inhibit tumor cell growth by blocking growth factor receptors, (iv) coupling <strong>of</strong> therapeutics to antibodies,<br />
nanoparticles or other ligands that recognize tumor-associate antigens (v) immune based strategies to induce anti-tumor immunity,<br />
reduce immune down regulation or exploit engineered immune acting cells to destroy tumors, and (vi) the use <strong>of</strong> designer biologics<br />
such as viruses, bacteria or stem cells to selectively destroy tumors. Some notable successes will be highlighted for each <strong>of</strong> these<br />
approaches, however tumor heterogeneity makes it unlikely that any one strategy will succeed. Cancer virotherapy remains a<br />
very versatile strategy that takes advantage <strong>of</strong> advances in enhanced vector design to provide the most potent, highly selective<br />
tumor killers. Current methods to control vector transduction and transcription, evasion <strong>of</strong> innate immunity and enhanced vector<br />
replication and spread will be presented.<br />
Stephen J. Russell, MD, PhD<br />
Oncolytic Virotherapy<br />
Oncolytic viruses (OV) selectively destroy neoplastic tissue and it is theoretically possible to engineer or adapt any naturally<br />
occurring virus for this purpose; key engineering strategies will be reviewed. A spreading intratumoral OV infection can damage<br />
both infected and uninfected tumor cells; the mechanisms underlying both <strong>of</strong> these activities will be discussed. Oncolytic viruses<br />
can be combined with drugs or armed with transgenes to accelerate their spread, modulate their immunogenicity/potency or<br />
facilitate the noninvasive monitoring <strong>of</strong> their in vivo fate; illustrative examples will be discussed. <strong>Final</strong>ly, several oncolytic viruses<br />
are currently being tested in clinical trials and the current status <strong>of</strong> this work will be summarized.<br />
54<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013
<strong>Program</strong> Schedule, Thursday, May 16, 2013<br />
Carl H. June, MD<br />
T <strong>Cell</strong> Mediated <strong>Gene</strong> Transfer for Cancer <strong>Therapy</strong><br />
The adoptive transfer <strong>of</strong> anti-tumor T cells can mediate regression <strong>of</strong> established metastatic cancers in patients. We have begun a<br />
series <strong>of</strong> clinical trials using the transduction <strong>of</strong> genes encoding chimeric antigen receptors (CARs) or anti-tumor T cell receptors<br />
(TCR) into normal peripheral lymphocytes for use in adoptive cell transfer. Anti-tumor TCRs have been identiied that recognize<br />
cancer testis antigens. Autologous cells transduced with genes encoding the anti-NY-ESO-1 TCR cancer-testes antigen mediated<br />
objective clinical responses in patients with myeloma. An afinity enhanced MAGE A3 TCR caused severe adverse events with<br />
fatal <strong>of</strong>f-target cardiac toxicity, pointing to serious concerns with the ability <strong>of</strong> pre-clinical testing to identify <strong>of</strong>f target effects <strong>of</strong><br />
TCRs. Patients with acute pre-B cell leukemia or chronic lymphocytic leukemia exhibited a high objective regression rate using<br />
T cells transduced with a CD19 CAR. The cellular and molecular mechanisms <strong>of</strong> T cell destruction <strong>of</strong> cancers are under active<br />
investigation. Ongoing studies are evaluating chimeric antibody receptors targeting mesothelin, and EGFRvIII. Together these<br />
studies demonstrate the power and potential <strong>of</strong> adoptive T cell immunotherapy to mediate the regression <strong>of</strong> established advanced<br />
cancer in humans.<br />
Lieping Chen, MD, PhD<br />
New Perspectives in the Development <strong>of</strong> Cancer Vaccines<br />
There is ample evidence that human malignancies express tumor-associated or tumor-speciic antigens. Immunization with cancer<br />
antigen-based vaccine in animal models or patients with advanced cancer induce tumor-reactive T cells in lymphoid organs and<br />
these T cells can be detected in blood. However, increased circulating tumor-reactive T cells <strong>of</strong>ten do not correlate with regression<br />
<strong>of</strong> advanced cancer. Recent studies reveal that speciic resistant mechanisms are developed in the tumor microenvironment to<br />
suppress activity <strong>of</strong> tumor-iniltrating T cells. It is thus important to understand and identify molecules underlying these resistance<br />
mechanisms. I will focus my discussion on analysis <strong>of</strong> mechanisms in the tumor microenvironment in the context <strong>of</strong> improvement<br />
<strong>of</strong> the eficacy <strong>of</strong> cancer vaccines.<br />
Thursday, May 16, 2013<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013<br />
55
<strong>Program</strong> Schedule, Thursday, May 16, 2013<br />
Foundation Symposium 241<br />
6:00 pm - 8:00 pm<br />
ROOM: BALLROOM A<br />
Association Francaise Contre les Myopathies/Muscular Dystrophy Association Symposium<br />
Jointly planned by the Association Francaise contre les Myopathies and the Muscular Dystrophy Association.<br />
CO-CHAIRS: Charles A. Gersbach, PhD and Kathryn R. Wagner, MD, PhD<br />
SPEAKERS<br />
Thursday, May 16, 2013<br />
Jeffrey Chamberlain, PhD<br />
Lentiviral Modiication <strong>of</strong> Myogenic Stem <strong>Cell</strong>s<br />
Over the past decade adeno-associated viral (AAV) vectors have merged as a potential therapeutic for muscle disorders. While<br />
AAV vectors can eficiently transduce post-mitotic myoibers, they display low tropism for myogenic stem cells and are rapidly lost<br />
during cell proliferation due to minimal genomic integration. In contrast, lentiviral vectors can stably transduce myogenic stem cells<br />
both in vitro and in vivo, enabling expansion and transplantation <strong>of</strong> genetically modiied cells for therapeutic interventions. This<br />
lecture will review approaches for modifying myogenic stem cells for in vivo and ex vivo gene therapy <strong>of</strong> myogenic disorders such as<br />
the muscular dystrophies.<br />
Xiao Xiao, PhD<br />
Regional Limb Vein Perfusion in Muscular Dystrophy Dogs and Human Patients<br />
Fulvio Mavilio, PhD<br />
<strong>Gene</strong>-based Therapies for Neuromuscular Disorders<br />
Most inherited neuromuscular diseases have no therapy, and collectively represent a signiicant unmet medical need. <strong>Gene</strong>thon and<br />
its collaborating clinical centers have launched an ambitious program aimed at developing gene therapy approaches for at least<br />
three <strong>of</strong> these diseases (Duchenne muscular dystrophy, myotubular myopathy and spinal muscular atrophy) based on systemic<br />
or loco-regional delivery <strong>of</strong> AAV vectors. We will provide an overview <strong>of</strong> the program and <strong>of</strong> the scientiic, regulatory and clinical<br />
challenges associated with its development.<br />
Anna Buj Bello, MD, PhD<br />
Myotubularin Deicie ncy<br />
Myotubular myopathy (XLMTM) is a very severe congenital muscle disorder due to mutations in the MTM1 gene. An overview<br />
about the disease, animal models and current experimental therapies, in particular our results on intravenous AAV-mediated gene<br />
replacement therapy, will be presented. Outstanding Achievement Award Lecture 300<br />
56<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013
<strong>Program</strong> Schedule, Thursday, May 16, 2013<br />
Plenary Session 300<br />
8:00 am - 9:00 am<br />
ROOM: BALLROOM E-J<br />
Outstanding Achievement Award Lecture and Presentation <strong>of</strong> the Distinguished Service Award<br />
and Special Recognition Award<br />
CHAIR<br />
Katherine P. Ponder, MD<br />
DISTINGUISHED SERVICE AWARD RECIPIENTS<br />
SPECIAL RECOGNITION AWARD RECIPIENT<br />
Sonia I. Skarlatos, PhD Catherine McKeon, PhD John J. Rossi, PhD<br />
SPEAKER<br />
Thursday, May 16, 2013<br />
Mark A. Kay, MD, PhD<br />
Deciphering Vector and Non-coding RNA Biology: A New Frontier in Nucleic Acid based Therapies<br />
In the last decade, we have witnessed great improvements in vector biology and utilized these for novel biomedical discoveries and<br />
therapeutic applications. In addition, it is now established that over 90% <strong>of</strong> the genome is transcribed, yet the role for most <strong>of</strong> the<br />
non-coding RNAs remains unclear. I will discuss our studies aimed at both uncovering the generation and function <strong>of</strong> non-coding<br />
RNAs, as well as the mechanisms important for the development <strong>of</strong> new vector-based gene transfer approaches to manipulate gene<br />
expression. Together, these approaches have provided continued and powerful insight into gene therapy mechanisms used to treat<br />
an array <strong>of</strong> diseases.<br />
Exhibit Hall C<strong>of</strong>fee Social<br />
9:00 am - 9:45 am<br />
ROOM: EXHIBIT HALL C/D<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013<br />
57
<strong>Program</strong> Schedule, Friday, May 17, 2013<br />
Scientific Symposium 310<br />
9:45 am - 11:45 am<br />
ROOM: BALLROOM D<br />
Cancer <strong>Gene</strong> and <strong>Cell</strong> <strong>Therapy</strong>: Results from Clinical Trials<br />
CO-CHAIRS: Evanthia Galanis, MD, DSc and Juan F. Vera, MD<br />
SPEAKERS<br />
Svend O. Freytag, PhD<br />
Randomized, Controlled, Phase 2 Trial <strong>of</strong> Oncolytic Adenovirus-mediated Suicide <strong>Gene</strong> <strong>Therapy</strong> Plus IMRT<br />
Versus IMRT Alone in neWly-diagnosed, Intermediate-risk Prostate Cancer<br />
We have developed a multi-modal, gene therapy-based approach for the treatment <strong>of</strong> cancer involving an oncolytic adenovirus,<br />
suicide gene therapy and radiation therapy. We have evaluated the safety and eficacy <strong>of</strong> our approach in six clinical trials, including<br />
a randomized, controlled phase 2 trial in prostate cancer. The results <strong>of</strong> these clinical studies will be presented and the lessons<br />
learned discussed.<br />
Evanthia Galanis, MD, DSc<br />
Oncolytic Measles Virus Strains in Cancer Treatment Trials: An Update<br />
Measles virus is a negative strand RNA virus (paramyxovirus) with oncolytic properties. The signiicant preclinical activity <strong>of</strong> MV-<br />
Edmonston vaccine strains against multiple different animal tumor models, their potent bystander effect, their selectivity against<br />
tumor cells, and their ability to retain their oncolytic properties when engineered and retargeted, makes them a promising oncolytic<br />
platform. Two trackable engineered measles virus strain derivatives are currently in clinical testing; a derivative expressing the<br />
human carcinoembryonic antigen (MV-CEA) and a strain expressing the human sodium iodine symporter (MV-NIS). Phase I/II trials<br />
are ongoing or have been completed in ovarian cancer, glioblastoma multiforme, multiple myeloma, mesothelioma and head and<br />
neck cancer and a phase I/II trial <strong>of</strong> mesenchymal stem cell delivery will be activated later this year. Clinical data to date, challenges<br />
in clinical applications <strong>of</strong> oncolytic measles virus strains and preclinical data supporting future translational directions will be<br />
discussed.<br />
Friday, May 17, 2013<br />
Renier J. Brentjens, MD, PhD<br />
CAR Modiied T-cells, From the Bench to the Bedside<br />
T cells may be genetically modiied to express artiicial T cell receptors, termed chimeric antigen receptors or CARs targeted to<br />
tumor associated antigens. Previous work in preclinical models has demonstrated the potential <strong>of</strong> this approach in targeting T<br />
cells to systemic tumor and in turn eradicating tumor. More recently, we and others have successfully moved this technology to the<br />
clinical setting demonstrating early very promising responses with CD19 targeted CAR modiied T cells in patients with relapsed<br />
B cell malignancies including acute lymphoblastic and chronic lymphocytic leukemias as well as indolent B cell non-Hodgkins<br />
lymphomas. However, limitations to this adoptive T cell approach need to be addressed before this approach may be successfully<br />
optimized to B cell malignancies and further expanded to other cancers targeting other tumor associated antigens. One such an<br />
approach is the design <strong>of</strong> “armored CAR” T cells additionally modiied to express cytokines or co-stimulatory ligands to enhance in<br />
vivo cytotoxicity as well as persistence <strong>of</strong> CAR modiied T cells following infusion.<br />
Stephen Gottschalk, MD<br />
<strong>Cell</strong> Therapies for Solid Tumors: Successes and Challenges<br />
The outcome for patients with advanced solid tumors has not signiicantly improved over the last 2 decades. Thus, new targeted<br />
therapies are needed to improve current outcomes, and T-cell immunotherapy has the potential to fulill this need. In my talk I will<br />
review i) the use <strong>of</strong> Epstein-Barr virus (EBV)-speciic T cells to treat patients with advanced stage EBV+ nasopharyngeal carcinoma,<br />
ii) our recent studies to target solid tumors and their supporting stroma with chimeric antigen receptor (CAR) T cells, and iii) novel<br />
approaches to render T cells speciic for solid tumor antigens.<br />
58<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013
<strong>Program</strong> Schedule, Friday, May 17, 2013<br />
Scientific Symposium 311<br />
9:45 am - 11:45 am<br />
ROOM: BALLROOM B<br />
<strong>Cell</strong> and Tissue Engineering for Musculoskeletal Disease<br />
CO-CHAIRS: Alan J. Nixon, DVM, MS and Bing Wang, MD, PhD<br />
SPEAKERS<br />
Rita Perlingeiro, PhD<br />
From iPS cells to Skeletal Muscle Precursors: Potential for <strong>Gene</strong> Correction and Therapeutic Application<br />
A major obstacle in the application <strong>of</strong> cell-based therapies for the treatment <strong>of</strong> neuromuscular disorders is obtaining the<br />
appropriate number <strong>of</strong> stem/progenitor cells to produce effective engraftment. The use <strong>of</strong> pluripotent embryonic stem (ES) or<br />
induced pluripotent stem (iPS) cells could overcome this hurdle. We have developed a method to eficiently generate skeletal<br />
myogenic progenitors from pluripotent stem cells through Pax3 or Pax7 induction. We have applied this method to mouse ES<br />
and iPS cells, and more recently to human ES and iPS cells. In each case, Pax7-induced cells engraft extensively and promote<br />
improved contractility in transplanted dystrophic muscles (using a mouse model for Duchenne Muscular Dystrophy). In addition<br />
to generating new myoibers, the transplanted cells also seeded the muscle satellite cell compartment. Accordingly, in long-term<br />
cohorts, the frequency <strong>of</strong> human-speciic dystrophin+ ibers declined only modestly at one year post-transplant, compared to one<br />
month. Based on these indings, we are now focusing on the genetic correction <strong>of</strong> iPS cells encoding mutations in the dystrophin<br />
gene. Such studies are crucial for the future therapeutic application <strong>of</strong> iPS-derived myogenic progenitors in the autologous setting<br />
for DMD. These more recent studies will also be discussed.<br />
Rocky S. Tuan, PhD<br />
Regenerative Applications <strong>of</strong> Adult Stem <strong>Cell</strong>s<br />
Farshid Guilak, PhD<br />
Engineering Functional Tissue Replacements to Treat Osteoarthritis<br />
Osteoarthritis is painful and debilitating joint disease that is characterized by pain and degenerative changes in the articular<br />
cartilage and other joint tissues. While a number <strong>of</strong> techniques have been developed over the years for the treatment <strong>of</strong> small<br />
cartilage defects, there have been few attempts at tissue-engineered therapies for end-stage osteoarthritis. Some <strong>of</strong> the major<br />
considerations for this approach include the identiication and characterization <strong>of</strong> an abundant and accessible cell source as well as<br />
the design <strong>of</strong> biologically and mechanically functional scaffolds that can withstand joint loading. Here we describe the engineering<br />
<strong>of</strong> large, anatomically-shaped cartilage constructs using different stem cell sources (adipose stem cells, mesenchymal stem cells,<br />
or induced pluripotent stem cells), combined with mechanically functional cell-instructive scaffolds based on three-dimensional<br />
weaving <strong>of</strong> biocompatible ibers. As each iber is selected individually, this textile processing technique allows site-speciic<br />
functionalization <strong>of</strong> proteins or virus to facilitate the formation <strong>of</strong> complex inhomogenous tissues from a single cell source.<br />
Brian Johnstone, PhD<br />
Stem <strong>Cell</strong>s for Cartilage Repair<br />
Scientific Symposium 312<br />
9:45 am - 11:45 am<br />
ROOM: 150 DEFG<br />
<strong>Gene</strong> <strong>Therapy</strong> and Regenerative Medicine for Ocular and Metabolic Diseases<br />
CO-CHAIRS: Alberto Auricchio, MD and Thierry C. VandenDriessche, PhD<br />
SPEAKERS<br />
Friday, May 17, 2013<br />
Ian Constable, AO<br />
Development <strong>of</strong> AAV-sFLT-1 for the Treatment <strong>of</strong> Wet Age-related Macular Degeneration<br />
The eye is an attractive organ in which to develop gene therapy. There is relative ease <strong>of</strong> access, a benign immunological response<br />
and the availability <strong>of</strong> high resolution non-invasive structural and functional tests. The safety and possible eficacy <strong>of</strong> Adenoassociated<br />
Viral (AAV) gene therapy is now well established in young humans with RPE-65 defective retinal blindness. We have<br />
adopted similar techniques to treat wet macular degeneration by subretinal injection <strong>of</strong> AAV-sFLT-1 to up-regulate production <strong>of</strong><br />
sFLT-1, a competitive analogue <strong>of</strong> the soluble Vascular Endothelial Growth Factor receptor.<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013<br />
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<strong>Program</strong> Schedule, Friday, May 17, 2013<br />
Ludovic Vallier, PhD<br />
<strong>Gene</strong> Correction <strong>of</strong> AAT Deiciency Using iPS-derived Hepatic <strong>Cell</strong>s<br />
The possibility <strong>of</strong> generating human Induced Pluripotent Stem <strong>Cell</strong>s from somatic cells using epigenetic reprogramming represents<br />
a unique opportunity for personalised regenerative medicine. Indeed, these pluripotent stem cells could enable the production<br />
<strong>of</strong> patient speciic cell types which are fully immuno-compatible with the original donor thereby avoiding the need for immune<br />
suppressive treatment during cell based therapy. Here, we will present the challenges and the most recent advances toward the use<br />
<strong>of</strong> hIPSC lines for cell based therapy <strong>of</strong> inherited metabolic disorders.<br />
Daniel Gaudet, MD, PhD<br />
<strong>Gene</strong> Replacement <strong>Therapy</strong>: Lessons from Lipoprotein Lipase Deiciency<br />
Lipoprotein Lipase Deiciency (LPLD) is a rare autosomal recessive disease characterized by severe hypertriglyceridemia,<br />
chylomicronemia and risk <strong>of</strong> recurrent pancreatitis or other complications. AAV1- LPL S447X gene therapy (Glybera ®) has been<br />
developed to decrease the clinical morbidity <strong>of</strong> LPLD. The treatment is based on the rationale that by adding episomal copies<br />
<strong>of</strong> functional LPL genes into muscle cells lacking active LPL, metabolic function could be improved or restored. The clinical<br />
development program for Glybera included two observational studies, three open-label interventional studies and one case<br />
note review analysis. Based on the results <strong>of</strong> these studies, Glybera gene therapy has recently been approved by the European<br />
Commission, demonstrating that it is possible for a gene-based medication to be approved despite all the barriers, which include<br />
the rarity <strong>of</strong> the disease, the complexity <strong>of</strong> the treatment as well as a huge amount <strong>of</strong> technical, clinical, social, economical and<br />
ethical issues. Several key questions regarding the eficacy, safety, cost-effectiveness, accessibility and impact <strong>of</strong> Glybera on the<br />
treatment <strong>of</strong> LPLD will require time and long-term follow-up to be answered. These questions will be discussed with an emphasis<br />
on the clinician’s perspective and the clinician-patient relationship. Given the time and cost required to develop gene-based<br />
treatments, the approval <strong>of</strong> Glybera could have an encouraging effect on the development <strong>of</strong> gene therapy for other diseases.<br />
Alessandro Aiuti, MD, PhD<br />
Progress in <strong>Gene</strong> <strong>Therapy</strong> for Wiskott-Aldrich Syndrome<br />
Wiskott-Aldrich Syndrome (WAS) is a primary immunodeiciency associated with eczema, thrombocytopenia, and high risk <strong>of</strong><br />
developing autoimmunity and cancer. <strong>Gene</strong> therapy with ex vivo transduced hematopoietic stem cells (HSC) could represent a valid<br />
therapeutic option for patients lacking an HLA-identical donor. We designed a phase I/II clinical trial based on reduced intensity<br />
conditioning and infusion <strong>of</strong> autologous HSC transduced with a lentiviral vector expressing WAS protein under its endogenous<br />
promoter. High-throughput vector integration analyses demonstrated highly polyclonal hematopoiesis in vivo and improved safety<br />
proile over previously used retroviral technology platforms. <strong>Gene</strong> therapy resulted in unprecedented levels <strong>of</strong> progenitor cell<br />
engraftment, restored WAS expression and correction <strong>of</strong> the disease phenotype in affected children.<br />
Friday, May 17, 2013<br />
Scientific Symposium 313<br />
9:45 am - 11:45 am<br />
ROOM: BALLROOM A<br />
microRNA on <strong>Cell</strong>ular Immune Response<br />
CO-CHAIRS: Shulin Li, PhD and Denise Sabatino, PhD<br />
SPEAKERS<br />
Ryan M. O’Connell, PhD<br />
Micro-managing Inlammation<br />
MicroRNAs represent a powerful mode <strong>of</strong> posttranscriptional gene regulation, and have recently been linked to inlammatory<br />
disease in mammals. We have identiied speciic miRNAs that are expressed in immune cells during inlammatory responses, and<br />
have determined that they are central regulators <strong>of</strong> distinct types <strong>of</strong> inlammation. Our work has also unveiled direct, functionally<br />
relevant mRNA targets <strong>of</strong> these miRNAs, providing new insights into the molecular networks that govern inlammatory phenotypes.<br />
Based upon their emerging importance, miRNAs are now being targeted therapeutically in an effort to mitigate human disease.<br />
Brian D. Brown, PhD<br />
microRNA Regulation <strong>of</strong> Dendritic <strong>Cell</strong>s<br />
Plasmacytoid dendritic cells (pDC) are major interferon producing cells which are specialized at sensing the initial stages <strong>of</strong> viral<br />
infection. Because <strong>of</strong> this, pDCs are particularly relevant in the host response to vaccines and gene therapy vectors. I will discuss<br />
our work which has uncovered evidence <strong>of</strong> a microRNA that is crucial for proper pDC development and response to pathogen<br />
associated nucleic acids.<br />
Ian MacLachlan, PhD<br />
Immunological Considerations <strong>of</strong> Therapeutic siRNA<br />
60<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013
<strong>Program</strong> Schedule, Friday, May 17, 2013<br />
Judy Lieberman, MD, PhD<br />
Aptamer-siRNA Chimeras Targeting CD4 <strong>Cell</strong>s Inhibit HIV Transmission<br />
One attractive strategy for interrupting HIV transmission is a topical microbicide, but the need for application around the time <strong>of</strong><br />
sexual intercourse leads to poor patient compliance. Intravaginal application <strong>of</strong> CD4 aptamer-siRNA chimeras (CD4-AsiCs) targeting<br />
the HIV coreceptor CCR5, gag and vif protected humanized mice from sexual transmission. In nondividing cells and tissue, RNAimediated<br />
gene knockdown lasts for several weeks, providing an opportunity for infrequent dosing not temporally linked to sexual<br />
intercourse, when compliance is challenging. In follow-up studies, we investigated the durability <strong>of</strong> gene knockdown and protection,<br />
protection afforded by CCR5 or HIV gene knockdown on their own, and effectiveness <strong>of</strong> CD4-AsiCs formulated in a gel in polarized<br />
human cervicovaginal explants and in humanized mice. These data, showing persistent gene knockdown and sustained protection<br />
that was improved in a gel formulation, suggest that CD4-AsiCs are a promising approach for developing an HIV microbicide.<br />
Scientific Symposium 314<br />
9:45 am - 11:45 am<br />
ROOM: 151 ABCG<br />
Realizing the Potential: Getting to Funding, Getting to Approval and Getting to Market<br />
CO-CHAIRS: Anthony T. Cheung, PhD and Karen Kozarsky, PhD<br />
SPEAKERS<br />
Sander van Deventer, MD, PhD<br />
Development <strong>of</strong> a Commercial <strong>Gene</strong> <strong>Therapy</strong> Product<br />
The market authorization <strong>of</strong> Glybera by the EMA has given development <strong>of</strong> gene therapies a signiicant boost. In this presentation<br />
I will discuss regulatory and inancial challenges associated with the development <strong>of</strong> (future) gene therapy products, which is<br />
essential for successful transition <strong>of</strong> academic projects into commercial products.<br />
David Kirn, MD<br />
Oncolytic and Immunotherapeutic Vaccinia Viruses for Cancer: Clinical Pro<strong>of</strong>-<strong>of</strong>-concept with JX-594<br />
(Pexa-Vec)<br />
This talk will be an overview <strong>of</strong> oncolytic and immunotherapeutic vaccinia viruses for cancer, with a focus on clinical pro<strong>of</strong>-<strong>of</strong>concept<br />
data with JX-594 (Pexa-Vec). Dr. Kirn will review this emerging novel therapeutic class, including the scientiic rationale<br />
for cancer-selectivity, mechanisms-<strong>of</strong>-action, and human clinical trial data from Phase 1 and 2 studies with Pexa-Vec. Clinical trial<br />
data will include pharmacokinetics, pharmacodynamics, tumor biopsy analyses, safety, anti-cancer eficacy and survival. Data from<br />
a randomized dose-inding Phase 2 trial will be presented, demonstrating a dose-related overall survival beneit <strong>of</strong> high dose Pexa-<br />
Vec treatment in patients with advanced liver cancer (Nature Medicine 2013).<br />
Samuel C. Wadsworth, PhD<br />
Realizing the Potential: Getting to Approval and Getting to Market; Focus on Orphan Indications<br />
The successful path to approval and market for new drugs is a series <strong>of</strong> effective decisions. Basic science, clinical research and<br />
manufacturing expertise represent some <strong>of</strong> the major stepping stones on this path, although there are many others. Academic<br />
scientists, regulators, corporate scientists, regulators, patients and patient advocacy groups all play their roles on the journey. For<br />
new technologies like cell and gene therapy, the beacon that should guide us throughout this journey should be the demonstration<br />
<strong>of</strong> truly transformative beneit to patients.<br />
Friday, May 17, 2013<br />
Artur Isaev, MD, MBA<br />
Development, Clinical Trials and Registration <strong>of</strong> Neovasculgen® <strong>Gene</strong> Therapeutic Drug<br />
Presentation will cover the key steps in “Neovasculgen” VEGF165 plasmid gene therapy drug development and commercialization<br />
starting from choosing a target disease to its marketing, including assessment <strong>of</strong> key characteristics <strong>of</strong> the future drug,<br />
determination <strong>of</strong> the therapeutic approach to be used and design and performance <strong>of</strong> preclinical studies. The main focus <strong>of</strong><br />
the talk would be on “Neovasculgen” clinical trials design for chronic lower limb ischemia and their main results, which would<br />
highlight the necessity <strong>of</strong> precise deinition <strong>of</strong> the end points to obtain objective data about safety and eficacy <strong>of</strong> the drug. <strong>Final</strong>ly,<br />
the presentation would summarize speciicity <strong>of</strong> drug development in Russia, current stage <strong>of</strong> “Neovasculgen” development, and<br />
perspectives <strong>of</strong> plasmid gene therapy in general.<br />
PANEL DISCUSSION<br />
MODERATOR<br />
Estuardo Aguilar-Cordova, PhD<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013<br />
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<strong>Program</strong> Schedule, Friday, May 17, 2013<br />
PANEL<br />
Thomas W. Chalberg, Jr., PhD<br />
Artur Isaev, MD, MBA<br />
David Kirn, MD<br />
Sander van Deventer, MD, PhD<br />
Samuel C. Wadsworth, PhD<br />
Scientific Symposium 315<br />
9:45 am - 11:45 am<br />
ROOM: BALLROOM C<br />
Target Non Coding RNAs with Oligonucleotides<br />
CO-CHAIRS: Kevin V. Morris, PhD and Marco S. Weinberg, PhD<br />
SPEAKERS<br />
Claes Wahlestedt, MD, PhD<br />
RNA-Based Strategies To Upregulate <strong>Gene</strong> Expression<br />
Recently, key roles for regulatory long noncoding RNAs (lncRNAs) in the regulation <strong>of</strong> gene expression have emerged. I will review<br />
current understanding <strong>of</strong> the mechanisms <strong>of</strong> action <strong>of</strong> lncRNAs and their roles in disease, focusing on recent work in the design<br />
<strong>of</strong> inhibitors <strong>of</strong> the natural antisense transcript (NAT) class <strong>of</strong> lncRNAs, known as AntagoNAT oligonucleotides, and the issues<br />
associated with their therapeutic application.<br />
Christine Esau, PhD<br />
Targeting microRNAs in Human Disease: miR-33 in the Treatment <strong>of</strong> Atherosclerosis<br />
microRNAs are regulators <strong>of</strong> the genome that can modulate the activity <strong>of</strong> speciic gene networks. Single stranded oligonucleotides<br />
can be designed to inhibit the activity <strong>of</strong> individual microRNAs that are overexpressed in human disease. For example, miR-<br />
33 is a microRNA that is known to be overexpressed in macrophages that may be involved in the formation <strong>of</strong> atherosclerotic<br />
plaques. Inhibition <strong>of</strong> miR-33 has been shown de-repress known target mRNAs such as ABCA1, increase cholesterol eflux<br />
from macrophages and regress atherosclerotic lesions in rodent models. Thus targeting microRNAs holds great promise for the<br />
treatment <strong>of</strong> human diseases.<br />
Friday, May 17, 2013<br />
Keith Gagnon, PhD<br />
Long Noncoding RNAs Link Transcriptional Regulation <strong>of</strong> Inlammatory Pathway <strong>Gene</strong>s<br />
Coordinated expression <strong>of</strong> the eicosanoid signaling pathway genes COX-2 and PLA2G4A are regulated by a network <strong>of</strong> non-coding<br />
RNAs and gene looping. The long non-coding RNAs overlapping the COX-2 gene promoter can be targeted by complementary<br />
endogenous and synthetic small RNAs to elicit activation <strong>of</strong> both COX-2 and PLA2G4A transcription. This activation requires RNAi<br />
factors like Argonaute 2 and GW182, recruitment <strong>of</strong> RNA Polymerase II, and chromatin modifying factors. These indings suggest<br />
that non-coding RNA has the potential to assemble related genes into functional operons for regulation and small RNAs can control<br />
this mechanism to synchronously activate transcription.<br />
Kevin V. Morris, PhD<br />
Long non-coding RNAs, Epigenetics and <strong>Gene</strong> Expression<br />
In human cells some non-coding RNAs have been found to transcriptionally modulate gene expression by targeting epigenetic<br />
complexes to particular loci. The targeted disruption <strong>of</strong> these suppressor non-coding RNAs can result in targeted activation <strong>of</strong><br />
gene expression. Examples <strong>of</strong> this form <strong>of</strong> gene activation in disease models ranging from cystic ibrosis to HIV to cancer will be<br />
discussed.<br />
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<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013
<strong>Program</strong> Schedule, Friday, May 17, 2013<br />
Scientific Symposium 316<br />
9:45 am - 11:45 am<br />
ROOM: 150 ABC<br />
Partially supported by the Juvenile Diabetes Research Foundation.<br />
<strong>Gene</strong> and <strong>Cell</strong> <strong>Therapy</strong> for Diabetes<br />
CO-CHAIRS: Nicola Brunetti-Pierri, MD and Lawrence C. Chan, MD<br />
SPEAKERS<br />
H. Teresa Ku, PhD<br />
Pancreatic Stem and Progenitor <strong>Cell</strong>s<br />
Adult pancreatic stem and progenitor cells are potential unlimited sources <strong>of</strong> beta cells, however their existence is highly<br />
controversial. To address this issue, we report here the establishment <strong>of</strong> two in vitro pancreatic colony assays that enable<br />
quantitative measurement <strong>of</strong> self-renewal and differentiation at a single cell level, which is required to deinitively demonstrate<br />
progenitor cell activities. We ind that single pancreatic colony-forming cells are capable <strong>of</strong> self-renewal in the presence <strong>of</strong> Matrigel<br />
and a Wnt signaling agonist, and can differentiate into ductal-like cells (in the presence <strong>of</strong> Matrigel) as well as endocrine and acinar<br />
lineage cells (in the presence <strong>of</strong> an unique laminin hydrogel) in culture. These results demonstrate robust pancreatic progenitor cell<br />
activities in vitro. Our results call for further investigation <strong>of</strong> progenitor cells in the adult pancreas in vivo, a research activity that<br />
has largely ceased after a series <strong>of</strong> recent publications reporting negative indings.<br />
Lawrence C. Chan, MD<br />
<strong>Gene</strong> <strong>Therapy</strong> to Reverse Autoimmune Type 1 Diabetes<br />
Type 1 diabetes (T1D) happens when most <strong>of</strong> the body’s pancreatic beta cells are destroyed by autoimmunity. Therefore, to reverse<br />
diabetes in T1D patients, we need to [1] re-establish suficient beta cell mass, and [2] neutralize the effects <strong>of</strong> autoimmunity. Our<br />
laboratory has developed a strategy that couples gene therapy-mediated induction <strong>of</strong> new beta cell formation in the liver with an<br />
immunomodulatory regimen that protects the neo-beta cells from autoimmune destruction. This strategy successfully reverses<br />
diabetes in an autoimmune model in rodents; the concept <strong>of</strong> therapeutic hepatic islet induction may also work in nonhuman<br />
primates.<br />
Andrea Ballabio, MD<br />
Rescue <strong>of</strong> Fat Diet-induced Obesity and Diabetes by TFEB-mediated Regulation <strong>of</strong> Autophagy and Lipid<br />
Catabolism<br />
The lysosomal-autophagic pathway is activated by starvation and plays an important role in both cellular clearance and lipid<br />
catabolism. However, the transcriptional regulation <strong>of</strong> this pathway in response to metabolic cues is currently uncharacterized.<br />
Here we show that the transcription factor EB (TFEB), a master regulator <strong>of</strong> lysosomal biogenesis and autophagy, is induced by<br />
starvation through an autoregulatory feedback loop and exerts a global transcriptional control on lipid catabolism via PGC1α and<br />
PPARα. Thus, during starvation a transcriptional mechanism links the autophagic pathway to cellular energy metabolism. Viral<br />
delivery <strong>of</strong> TFEB to the liver rescued obesity, diabetes and the metabolic syndrome in both diet-induced and genetic mouse models<br />
<strong>of</strong> obesity, suggesting a novel therapeutic strategy for disorders <strong>of</strong> lipid metabolism.<br />
Friday, May 17, 2013<br />
Markus Grompe, MD<br />
Reprogramming Gall Bladder to make Insulin<br />
Lunch Break (On Own - Not Provided; Pre-ordered boxed lunches available for pick up at Registration Counter)<br />
11:45 am - 1:00 pm<br />
Oral Abstract Session 320<br />
1:00 pm - 3:00 pm<br />
ROOM: BALLROOM A<br />
DNA Vectorology & <strong>Gene</strong> Targeting<br />
CO-CHAIRS: Matthew Hirsch, PhD and Carol H. Miao, PhD<br />
(Abstracts 266 through 273; see page 108)<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013<br />
63
<strong>Program</strong> Schedule, Friday, May 17, 2013<br />
Oral Abstract Session 321<br />
1:00 pm - 3:00 pm<br />
ROOM: BALLROOM B<br />
<strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> <strong>of</strong> Diabetes, Metabolic and <strong>Gene</strong>tic Diseases I<br />
CO-CHAIRS: Sihong Song, PhD and Stephanie Cherqui, PhD<br />
(Abstracts 274 through 281; see pages 108-109)<br />
Oral Abstract Session 322<br />
1:00 pm - 3:00 pm<br />
ROOM: 150 ABC<br />
Neurological <strong>Therapy</strong>: Neurodegeneration and Imaging<br />
CO-CHAIRS: Kryst<strong>of</strong> Bankiewicz, MD, PhD and Brian K. Kaspar, PhD<br />
(Abstracts 282 through 289; see page 109)<br />
Oral Abstract Session 323<br />
1:00 pm - 3:00 pm<br />
ROOM: 150 DEFG<br />
Musculo-skeletal <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> II<br />
CO-CHAIRS: Jacques P. Tremblay, PhD and Annemieke Aartsma-Rus, PhD<br />
(Abstracts 290 through 297; see pages 109-110)<br />
Friday, May 17, 2013<br />
Oral Abstract Session 324<br />
1:00 pm - 3:00 pm<br />
ROOM: BALLROOM C<br />
Cancer - Targeted <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong><br />
CO-CHAIRS: Karen S. Aboody, MD and Janaiah Kota, PhD<br />
(Abstracts 298 through 305; see page 110)<br />
Oral Abstract Session 325<br />
1:00 pm - 3:00 pm<br />
ROOM: 151 ABCG<br />
<strong>Gene</strong> <strong>Therapy</strong> for Hematologic Diseases: From Models to Clinical Results<br />
CO-CHAIRS: Fabio Candotti, MD and Arthur W. Nienhuis, MD<br />
(Abstracts 306 through 313; see page 111)<br />
Oral Abstract Session 326<br />
1:00 pm - 3:00 pm<br />
ROOM: BALLROOM D<br />
<strong>Gene</strong> Regulation and Animal Models<br />
CO-CHAIRS: David W. Emery, PhD and David M. Spencer, PhD<br />
(Abstracts 314 through 321; see pages 111-112)<br />
64<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013
<strong>Program</strong> Schedule, Friday, May 17, 2013<br />
Presidential Symposium 330<br />
3:15 pm - 5:30 pm<br />
ROOM: BALLROOM E-J<br />
Presidential Symposium<br />
CHAIR<br />
SPEAKER<br />
Xandra O. Breakeield, PhD<br />
David Baltimore, PhD<br />
<strong>Gene</strong>-based Anti-Viral Protection<br />
Standard vaccination development methods have not provided protections against certain infectious agents, notably HIV. However,<br />
very effective monoclonal antibodies to such agents have been found. We have undertaken to use gene therapy-like methods<br />
to provide protection against HIV and other agents, using Adenovirus-Associated Virus in a methodology we termed Viral<br />
ImmunoProphylaxis (VIP). This has proved very effective in humanized mice and is being developed for human trials.<br />
PRESENTATION OF TOP ABSTRACTS:<br />
Sylvie Rival-Gervier, PhD<br />
Abstract 322: Kinetics and Epigenetics <strong>of</strong> Retroviral Silencing in Mouse Embryonic Stem <strong>Cell</strong>s Deined by<br />
Deletion <strong>of</strong> a D4Z4 Barrier Element<br />
Mahzad Akbarpour, DVM<br />
Abstract 323: Liver-Directed, Insulin-Based <strong>Gene</strong> <strong>Therapy</strong> Protects NOD Mice from Diabetes<br />
Thomas J. Cradick, PhD<br />
Abstract 324: On-Target Cleavage and Off-Target Activity <strong>of</strong> TALEN ‘Nickases’<br />
Rimas J. Orentas, PhD<br />
Abstract 325: Targeting B <strong>Cell</strong> Precursor Acute Lymphoblastic Leukemia (ALL) with Chimeric Antigen<br />
Receptors (CARs) Speciic for CD19 or CD22<br />
Exhibit Hall Open: Networking Reception & Poster Session II<br />
5:30 pm - 7:30 pm<br />
ROOM: EXHIBIT HALL C/D<br />
AAV Vectors - II<br />
(Abstracts 326-340; Abstract 329 has been withdrawn from presentation; see pages 112-113)<br />
Friday, May 17, 2013<br />
DNA Vectorology & <strong>Gene</strong> Targeting I<br />
(Abstracts 341-352; see pages 113-114)<br />
<strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> <strong>of</strong> Diabetes, Metabolic and <strong>Gene</strong>tic Diseases I<br />
(Abstracts 353-365; see pages 114-115)<br />
Cardiovascular <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong><br />
(Abstracts 366-375; see page 115)<br />
Neurologic <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> I<br />
(Abstracts 376-391; and Abstract 590 moved from Poster Session III; see pages 115-116)<br />
Cancer - Immunotherapy II<br />
(Abstracts 392-404; see page 117)<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013<br />
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<strong>Program</strong> Schedule, Friday, May 17, 2013<br />
Cancer-Oncolytic Viruses I<br />
(Abstracts 405-417; see pages 117-118)<br />
Hematologic and Immunologic <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> I<br />
(Abstracts 418-431; see pages 118-119)<br />
<strong>Gene</strong> and <strong>Cell</strong> <strong>Therapy</strong> for Pulmonary Diseases<br />
(Abstracts 432-440; see page 119)<br />
Immunologic & Host Responses in <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong><br />
(Abstracts 441-449; see page 120)<br />
Oligonucleotide & RNAi Therapeutics<br />
(Abstracts 450-464; see pages 120-121)<br />
Pluripotent Stem <strong>Cell</strong>s<br />
(Abstracts 465-476; Abstract 475 Withdrawn from Presentation; see pages 121-122)<br />
Late Breaking Abstracts II<br />
(Abstracts 702-724; Abstract 701 Withdrawn from Presentation; see pages 122-123)<br />
New Investigator Town Hall Gathering<br />
7:30 pm - 8:30 pm<br />
ROOM: BALLROOM B<br />
CO-CHAIRS: Jennifer E. Adair, PhD and Luk H. Vandenberghe, PhD<br />
SPEAKERS<br />
Friday, May 17, 2013<br />
David V. Schaffer, PhD<br />
The Structural Organization <strong>of</strong> ASGCT<br />
The ASGCT is a broad organization that serves many needs <strong>of</strong> its members, and there are numerous opportunities for young<br />
investigators to become actively involved in our society. This talk will discuss the overall organization <strong>of</strong> ASGCT, including our<br />
committee structures, annual meeting, and awards.<br />
Andrew H. Baker, PhD<br />
British <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> and <strong>Cell</strong> <strong>Therapy</strong><br />
Discussion around the role <strong>of</strong> BSGCT and the opportunities for interactions for early career researchers.<br />
Luigi M. Naldini, MD, PhD<br />
Opportunity for Young Scientists to Contribute to the European <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> and <strong>Cell</strong> <strong>Therapy</strong><br />
Kenzaburo Tani, MD, PhD<br />
Current Situation <strong>of</strong> New Investigator Support Opportunities in JSGT<br />
JSGT have been supporting new investigators by supplying several educational lectures, awards and research grants. We also have<br />
open several postdoctoral positions for foreign new investigators.<br />
Zoe Fonseca-Kelly, PhD<br />
Strategies for Career Success Beyond Academia<br />
The role and work <strong>of</strong> the National Postdoctoral Association will be presented along with data pertaining to postdoctoral issues<br />
and developments. In addition basic strategies in planning a successful career beyond ones postdoc both within and outside <strong>of</strong><br />
academia will be presented.<br />
66<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013
<strong>Program</strong> Schedule, Friday, May 17, 2013<br />
Robert M. Frederickson, PhD<br />
Molecular <strong>Therapy</strong> and the ASGCT Publishing <strong>Program</strong><br />
This presentation will provide a brief overview <strong>of</strong> the <strong>Society</strong>’s lagship journal, Molecular <strong>Therapy</strong>, as well as the new online-only,<br />
open-access sibling journals. It will also present a roadmap <strong>of</strong> the editorial processes at the journals along with suggestions on how<br />
to navigate these processes successfully. <strong>Final</strong>ly, we will outline opportunities for new investigators to get involved and contribute<br />
to the ASGCT publishing program.<br />
New Investigator Mentoring Event<br />
8:30 pm - 9:30 pm<br />
ROOM: BALLROOM D<br />
Foundation Symposium 340<br />
7:30 pm - 9:30 pm<br />
ROOM: BALLROOM A<br />
<strong>Cell</strong> and <strong>Gene</strong> <strong>Therapy</strong> in the Eye, Navigating the Road from the Lab to the Clinic<br />
Supported by the Foundation Fighting Blindness.<br />
CHAIR: Stephen M. Rose, PhD<br />
SPEAKERS<br />
Stephen M. Rose, PhD<br />
The Role <strong>of</strong> the Foundation Fighting Blindness in Bringing Treatments to the Clinic - Funding the “The Valley <strong>of</strong><br />
Death”<br />
Sue Washer, MBA<br />
Small Company Experience; Maximizing Sources <strong>of</strong> Funding and Shots on Goal<br />
Samuel C. Wadsworth, PhD<br />
Large Company Experience, Navigating Through “New Technology” Challenge in a Large Organization<br />
New technological advancements drive development <strong>of</strong> new therapeutic modalities. Newness implies risk <strong>of</strong> failure which is<br />
the norm for scientiic endeavors because out <strong>of</strong> failure <strong>of</strong>ten comes insight that propels ields to new heights <strong>of</strong> understanding.<br />
Failure is an accepted ingredient <strong>of</strong> product development as well – obviously well-managed and early failure is ideal in a corporate<br />
environment. Layering on <strong>of</strong> new technologies such as gene and cell therapies complicates the process. Navigation through this<br />
arena will be discussed.<br />
Michael Young, PhD<br />
Retinal Stem <strong>Cell</strong>s: Working with Biotech<br />
I will present my perspective on collaboration with biotech in the development <strong>of</strong> a stem cell therapy for retinal degeneration. My<br />
group has worked with Reneuron, Inc. for almost 8 years now, and we hope to translate our work into a clinical product . I will<br />
discuss the challenges one encounters when working closely with industry.<br />
Friday, May 17, 2013<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013<br />
67
<strong>Program</strong> Schedule, Friday, May 17, 2013<br />
Workshop 350<br />
7:30 pm - 9:30 pm<br />
ROOM: BALLROOM C<br />
International Consortium for <strong>Gene</strong> and <strong>Cell</strong> <strong>Therapy</strong> Workshop<br />
CHAIR: Jacques P. Tremblay, PhD<br />
SPEAKERS<br />
Jacques P. Tremblay, PhD<br />
Introduction to the Creation <strong>of</strong> an International Consortium <strong>of</strong> <strong>Gene</strong><br />
Dr. Tremblay has recently published in collaboration with 50 experts in gene therapy a letter in Molecular <strong>Therapy</strong> proposing<br />
the creation <strong>of</strong> an International Consortium <strong>of</strong> <strong>Gene</strong> <strong>Therapy</strong> to accelerate the development <strong>of</strong> clinical applications. Indeed the<br />
recent progress in genomics has permitted to identify the genes responsible for 10,000 hereditary monogenic diseases. Although<br />
each one <strong>of</strong> these diseases is rare (they are thus designated as orphan diseases), this whole group <strong>of</strong> diseases affects 8% <strong>of</strong> the<br />
human population and is thus a major cost for the public health systems. On the other hand, gene therapy has recently permitted<br />
successful treatment <strong>of</strong> some <strong>of</strong> these hereditary diseases. In particular, the new AAV serotypes permit to deliver a corrective gene<br />
more eficiently because human are not pre-immunized to some <strong>of</strong> the new serotypes. There are also several new tools to correct<br />
a mutated gene: meganucleases, ZFNs, TALENs and the CRISPR system, or intervene at the level <strong>of</strong> the mRNA: oligonucleotides for<br />
exon skipping, siRNA and shRNA. Promising results have been recently obtained in several animal models <strong>of</strong> hereditary diseases.<br />
The limiting step to be able to push these discoveries to a clinical application is the high cost <strong>of</strong> producing the clinical grade vectors.<br />
The workshop during the annual meeting <strong>of</strong> the ASGCT is to set the basis <strong>of</strong> that consortium, deine its goals and elect a direction<br />
committee. Among the proposed objectives will be to lobby governments for increase funding <strong>of</strong> this type <strong>of</strong> research, establishing<br />
international patient registry, developing more GMP facilities to produce the therapeutic agents, making the regulations more<br />
uniform in various countries. A large part <strong>of</strong> the workshop will be devoted to an open discussion so that ALL the participants can<br />
make their suggestions about the objectives and the methods to reach them.<br />
Yu-Quan Wei, MD, PhD<br />
<strong>Gene</strong> <strong>Therapy</strong> for <strong>Gene</strong>tic Diseases in China<br />
Xiao Xiao, PhD<br />
Current Status <strong>of</strong> Clinical Grade AAV Vector Production<br />
Friday, May 17, 2013<br />
Annemieke Aartsma-Rus, PhD<br />
The Treat-nmd Paradigm<br />
The TREAT-NMD Alliance is a network for the neuromuscular ield that provides an infrastructure to facilitate and accelerate<br />
the clinical development <strong>of</strong> new therapies. During its EU funded period (2007-2011) the network has collaborated with the<br />
USA to develop tools such as disease registries, standard <strong>of</strong> care guidelines, a clinical trial site registry, standardized operating<br />
procedures for preclinical animal studies, has set up the TREAT-NMD advisory committee on therapeutics (TACT), has maintained<br />
a biobank with samples from neuromuscular disorder patients and has gathered a wealth <strong>of</strong> information for the neuromuscular<br />
ield (see www.treat-nmd.eu). Following the end <strong>of</strong> the EU funding, the Alliance and its tools have been maintained and the<br />
network is running with other sources <strong>of</strong> funding. Current focus is on furthering the biobanks, setting up patient registries for<br />
additional neuromuscular disorders and in more countries, optimizing the clinical trial site registry, optimizing outcome measures<br />
for ambulant and non-ambulant patients and generating standards <strong>of</strong> care for additional diseases and updating existing care<br />
guidelines. The TREAT-NMD Alliance has also given rise to new EU funded projects such as RD-Connect, Neuromics, Optimistic and<br />
SKIP-NMD. While for the TREAT-NMD infrastructure the disease area is the connecting factor, it can serve as a paradigm for the gene<br />
therapy consortium where the therapeutic tool is the connecting factor.<br />
Philip J. Brooks, PhD<br />
Activities <strong>of</strong> the Ofice <strong>of</strong> Rare Diseases Research, NCATS, NIH, in Facilitating Translational Research in Rare<br />
Diseases Around the World<br />
68<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013
<strong>Program</strong> Schedule, Saturday, May 18, 2013<br />
ASGCT Business Meeting<br />
7:30 am - 8:00 am<br />
ROOM: 150 ABC<br />
Plenary Session 400<br />
8:00 am - 10:00 am<br />
ROOM: BALLROOM E-J<br />
Outstanding New Investigator Symposium<br />
CHAIR<br />
SPEAKERS<br />
Katherine P. Ponder, MD<br />
Aravind Asokan, PhD<br />
A Sweet Side to AAV Biology<br />
Adeno-associated viruses (AAV) exploit a spectrum <strong>of</strong> glycans for cell surface attachment and transduction. Understanding the<br />
structural basis and functional implications <strong>of</strong> such virus-host interactions can guide development <strong>of</strong> recombinant AAV vectors<br />
for clinical gene therapy applications. This talk will highlight our contributions towards discovery <strong>of</strong> AAV receptors, structural<br />
determinants <strong>of</strong> AAV tissue tropism and new strategies to re-engineer AAV-glycan interactions.<br />
Paloma H. Giangrande, PhD<br />
Targeted-Image Guided RNA (TIGR) Therapies<br />
The successful application <strong>of</strong> RNAi therapeutics to humans awaits the development <strong>of</strong> robust delivery technologies. <strong>Cell</strong> conjugation<br />
<strong>of</strong> siRNAs to cell-speciic ligands provides a viable solution to this problem. Synthetic RNA ligands (aptamers) represent an<br />
emerging class <strong>of</strong> pharmaceuticals with great potential for targeted therapeutic/diagnostic applications. Here, we describe<br />
advancements in aptamer technology for enabling the development <strong>of</strong> effective image-guided RNA therapies.<br />
Saturday, May 18, 2013<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013<br />
69
<strong>Program</strong> Schedule, Saturday, May 18, 2013<br />
Michael A. Lalamme, MD, PhD<br />
Cardiac Repair with Human Pluripotent Stem <strong>Cell</strong>s<br />
Human pluripotent stem cells have a number <strong>of</strong> potential advantages for use in myocardial infarct repair, including tremendous<br />
capacity for expansion in the undifferentiated state and unambiguous cardiac potential. Our group irst reported what are now<br />
widely applied methods to eficiently guide the differentiation <strong>of</strong> human embryonic stem cells (hESCs) and induced pluripotent<br />
stem cells (hiPSCs) into cardiomyocytes, as well as their further differentiation to speciic cardiac subtypes (e.g. pacemaker or<br />
ventricular myocytes). In early pro<strong>of</strong>-<strong>of</strong>-concept studies using a rat infarct model, we showed that the transplantation <strong>of</strong> hESCderived<br />
cardiomyocytes results in partial remuscularization <strong>of</strong> the infarct scar and improved contractile function. More recently,<br />
we have used a guinea pig infarct model to show that hESC-derived cardiomyocyte grafts can integrate electrically and contract<br />
synchronously with host muscle; and that these grafts exert an intriguing arrhythmia-suppressive effect. Our ongoing efforts are<br />
focused on promoting the structural and functional maturation <strong>of</strong> hESC-derived cardiomyocytes, as well as their electromechanical<br />
integration in vivo.<br />
Ann M. Leen, PhD<br />
T cell <strong>Therapy</strong> for Viruses and Cancer<br />
Severe and fatal viral infections remain common after hematopoietic stem cell transplantation. Adoptive transfer <strong>of</strong> in vitro<br />
expanded cytotoxic T lymphocytes speciic for EBV, CMV and Adv antigens can treat infections that are impervious to conventional<br />
therapies. However, the time taken to prepare patient-speciic products, the limited breadth <strong>of</strong> viral speciicities that can be<br />
accommodated in a single line, and the lack <strong>of</strong> virus-speciic T-cells in cord blood and seronegative donors restricts application. In<br />
this presentation I will summarize our preclinical efforts to address these limitations and will present our clinical trial results using<br />
these products.<br />
Oral Abstract Session 410<br />
10:15 am - 12:15 pm<br />
ROOM: 150 DEFG<br />
Late Breaking Abstracts<br />
CO-CHAIRS: TO BE DETERMINED<br />
(Abstracts 725 through 732; see page 124)<br />
Saturday, May 18, 2013<br />
Oral Abstract Session 411<br />
10:15 am - 12:15 pm<br />
ROOM: 150 ABC<br />
Adenovirus and Other DNA Virus Vectors<br />
CO-CHAIRS: Prem Seth, PhD and Miguel Sena-Esteves, PhD<br />
(Abstracts 477 through 484; see pages 124-125)<br />
70<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013
<strong>Program</strong> Schedule, Saturday, May 18, 2013<br />
Oral Abstract Session 412<br />
10:15 am - 12:15 pm<br />
ROOM: BALLROOM A<br />
Nuclease Mediated Genome Editing<br />
CO-CHAIRS: Shondra M. Miller, PhD and Andy Wilber, PhD<br />
(Abstracts 485 through 492; see page 125)<br />
Oral Abstract Session 413<br />
10:15 am - 12:15 pm<br />
ROOM: BALLROOM B<br />
<strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> <strong>of</strong> Diabetes, Metabolic and <strong>Gene</strong>tic Diseases II<br />
CO-CHAIRS: Gerald S. Lipshutz, PhD and Nicola Brunetti-Pierri, MD<br />
(Abstracts 494 through 500; see pages 125-126)<br />
Oral Abstract Session 414<br />
10:15 am - 12:15 pm<br />
ROOM: 151 ABCG<br />
Cancer - Immunotherapy II<br />
CO-CHAIRS: Laurence JN Cooper, MD, PhD and Richard G. Vile, PhD<br />
(Abstracts 501 through 508; see page 126)<br />
Oral Abstract Session 415<br />
10:15 am - 12:15 pm<br />
ROOM: BALLROOM C<br />
Oligonucleotide & RNAi Therapeutics<br />
CO-CHAIRS: Scott Q. Harper, PhD and M. Corinna Palanca-Wessels, MD, PhD<br />
(Abstracts 509 through 516; see page 127)<br />
Lunch Break (On Own - Not Provided; Pre-ordered boxed lunches available for pick up at Registration Counter)<br />
12:15 pm - 1:45 pm<br />
Meet-the-Investigator Lunch Session 420<br />
12:15 pm - 1:45 pm<br />
ROOM: 251 E<br />
Targeting TTR in the Liver<br />
SPEAKER<br />
Daniel G. Anderson, PhD<br />
Meet-the-Investigator Lunch Session 421<br />
12:15 pm - 1:45 pm<br />
MEET AT THE SOUTH ENTRANCE OF THE SALT PALACE CONVENTION CENTER<br />
Huntsman Cancer Institute Tour<br />
Boxed lunches will be available at the South Entrance shortly before departure to the Huntsman Cancer Institute.<br />
Saturday, May 18, 2013<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013<br />
71
<strong>Program</strong> Schedule, Saturday, May 18, 2013<br />
Scientific Symposium 430<br />
1:45 pm - 3:45 pm<br />
ROOM: BALLROOM B<br />
Clinical Trials<br />
CO-CHAIRS: Donald B. Kohn, MD and Miguel Sena-Esteves, PhD<br />
SPEAKERS<br />
Helen E. Heslop, MD<br />
Immunotherapy with Viral Speciic T <strong>Cell</strong>s<br />
Immunotherapy with antigen-speciic T cells has the potential to reconstitute antiviral activity post transplant without inducing<br />
GVHD and our group has performed a series <strong>of</strong> clinical studies showing that virus-speciic cytotoxic T lymphocytes (CTLs) can<br />
reconstitute immunity and treat viral reactivation and disease. To extend this approach to EBV-associated lymphoma occurring in<br />
the immunocompetent host expressing Type 2 latency, we have generated CTL lines targeting the subimmunodominant antigens<br />
LMP1 and LMP2 using antigen presenting cells genetically modiied to overexpress either LMP2 alone or LMP1 and LMP2 by<br />
transduction with an Ad5f35 vectors. 27 <strong>of</strong> 29 high-risk and/or multiply relapsed patients who received LMP-CTL as adjuvant<br />
treatment remain in remission for a median <strong>of</strong> 2.5 years after CTL while 13 <strong>of</strong> 21 patients with detectable disease had clinical<br />
responses. In conclusion, immunotherapy with CTL targeting LMP antigens is well tolerated in patients with EBV+ lymphoma and<br />
infused LMP-CTL can accumulate at tumor sites and induce complete and sustained clinical responses.<br />
Roger J. Hajjar, MD<br />
<strong>Gene</strong> <strong>Therapy</strong> for Heart Failure<br />
Congestive heart failure remains a progressive disease with a desperate need for innovative therapies to reverse the course <strong>of</strong><br />
ventricular dysfunction. One <strong>of</strong> the key abnormalities in both human and experimental HF is a defect in sarcoplasmic reticulum<br />
(SR) function, which is responsible for abnormal intracellular Ca2+ handling. Deicient SR Ca2+ uptake during relaxation has been<br />
identiied in failing hearts from both humans and animal models and has been associated with a decrease in the activity <strong>of</strong> the SR<br />
Ca2+-ATPase (SERCA2a). Over the last ten years we have undertaken a program <strong>of</strong> targeting important calcium cycling proteins<br />
in experimental models <strong>of</strong> heart by somatic gene transfer. This has led to the completion <strong>of</strong> a irst-in-man phase 1 clinical trial<br />
<strong>of</strong> gene therapy for heart failure using adeno-associated vector (AAV) type 1 carrying SERCA2a. The safety proile <strong>of</strong> AAV gene<br />
therapy along with the positive biological signals obtained from this phase 1 trial has led to the initiation and recent completion <strong>of</strong><br />
a phase 2 trial <strong>of</strong> AAV1.SERCA2a in NYHA class III/IV patients. In the phase 2 trial, gene transfer <strong>of</strong> SERCA2a was found to be safe<br />
and associated with beneit in clinical outcomes, symptoms, functional status, NT-proBNP and cardiac structure. Furthermore, the<br />
recent successful and safe completion <strong>of</strong> the CUPID trial along with the start <strong>of</strong> more recent phase 1 trials usher a new era for gene<br />
therapy for the treatment <strong>of</strong> heart failure.<br />
Andrew M. David<strong>of</strong>f, MD<br />
AAV-mediated FIX <strong>Gene</strong> Transfer for Hemophilia B<br />
We are conducting a phase I/II clinical trial <strong>of</strong> factor IX gene transfer for severe hemophilia B. In the trial we are using a serotype-8<br />
pseudotyped self-complementary adeno-associated virus (scAAV) vector expressing a codon-optimized coagulation factor IX (FIX)<br />
transgene (scAAV2/8-LP1-hFIXco). AAV-mediated FIX activity at 1-6% <strong>of</strong> normal has been established in all participants with<br />
follow-up now <strong>of</strong> between 3 months and 3 years following gene transfer.<br />
Saturday, May 18, 2013<br />
C. Frank Bennett, PhD<br />
Antisense Oligonucleotide Therapies for the Treatment <strong>of</strong> Neurodegenerative Diseases<br />
Antisense oligonucleotides are chemically modiied oligonucleotides that are designed to speciically bind to a targeted RNA<br />
through Watson-Crick base pairing, modulating the function <strong>of</strong> the RNA. Because antisense oligonucleotides target RNA, rather<br />
than protein, they can be used to target a broader range <strong>of</strong> therapeutic targets than can be approached with conventional small<br />
molecule or protein based drugs. Neurodegenerative diseases are an attractive area for antisense technology in that there a number<br />
<strong>of</strong> diseases in which the genetic basis is known and not easily approachable with small molecule drugs.<br />
Scientific Symposium 431<br />
1:45 pm - 3:45 pm<br />
ROOM: 150 ABC<br />
Do Effective <strong>Gene</strong> Therapies and Regenerative Medicine Affect Dilemmas <strong>of</strong> Resource<br />
Allocation<br />
CHAIR: TBD<br />
72<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013
<strong>Program</strong> Schedule, Saturday, May 18, 2013<br />
SPEAKERS<br />
Jeffrey Chamberlain, PhD<br />
Augmentation <strong>of</strong> Muscle Function Via <strong>Gene</strong> Transfer-The Good, The Bad and The Ugly<br />
Emerging gene and stem cell therapies for inherited muscle disorders aim to stop muscle degeneration while increasing muscle<br />
mass and strength. Interventions that regulate muscle mass are also potentially powerful approaches to slow the loss <strong>of</strong> strength<br />
that accompany aging (sarcopenia) and cancer (cachexia). However, any safe method developed to increase muscle mass or<br />
strength could also be attractive for use by individuals desiring greater strength or prowess, either due to slight build or athletic<br />
ambition. This lecture will review current approaches for enhancing muscle mass and their potential for use and abuse.<br />
Tho mas H. Murray, PhD<br />
Ethics, <strong>Gene</strong> and <strong>Cell</strong>ular Enhancements in Sport and Beyond<br />
Athletes will be quick to grasp the possible applications <strong>of</strong> gene and cell therapies for sarcopenia and other diseases for enhanced<br />
performance in sport. How should we think about the ethics <strong>of</strong> enhancement in sport and in other spheres <strong>of</strong> human life Examples<br />
from sport and from the practice <strong>of</strong> medicine demonstrate that understanding the ethics <strong>of</strong> any particular enhancement begins with<br />
appreciating the values we seek within that sphere and the meaning <strong>of</strong> that activity in people’s lives.<br />
Leonard M. Fleck, PhD<br />
Successful and Safe <strong>Gene</strong> <strong>Therapy</strong> Raises New Quandaries <strong>of</strong> Health Care Justice<br />
Our fundamental question is this: Given that any society has only limited resources to meet virtually unlimited health care needs,<br />
how high a priority, ethically speaking, ought gene therapy have in meeting those needs This leads to two subsidiary questions. (1)<br />
Who can be expected to beneit the most from emerging gene therapies, mostly younger individuals or mostly older individuals (2)<br />
What can we reasonably expect in the way <strong>of</strong> outcomes from various emerging gene therapies, cures or only halfway technologies<br />
so characteristic <strong>of</strong> medicine today If we had a form <strong>of</strong> gene therapy that could substantially address the loss <strong>of</strong> muscle function in<br />
Duchenne patients and could also address loss <strong>of</strong> muscle function in the elderly, would we argue from the perspective <strong>of</strong> health care<br />
justice in a world <strong>of</strong> limited resources with unlimited needs that both sorts <strong>of</strong> patients had an equal more right to this therapy Or<br />
would we argue instead that the younger Duchenne patients had the stronger just claim to this therapeutic intervention<br />
Scientific Symposium 432<br />
1:45 pm - 3:45 pm<br />
ROOM: 150 DEFG<br />
Exploiting Unique Attributes <strong>of</strong> Vectors for Design <strong>of</strong> Vaccines and Immunotherapies<br />
CHAIR: Eric Poeschla, MD<br />
SPEAKERS<br />
Shan Lu, MD, PhD<br />
DNA Immunization – Pro<strong>of</strong> <strong>of</strong> the Potential <strong>of</strong> <strong>Gene</strong>-based Delivery <strong>of</strong> Vaccines<br />
While gene therapy remains a challenge to treat any genetic and acquired illness, the emergence <strong>of</strong> DNA immunization has provided<br />
a unique model system to study the in vivo expression <strong>of</strong> proteins by a gene-based vector and the subsequent biological effects.<br />
Since its discovery in the early 1990s, the understanding <strong>of</strong> DNA immunization has grown substantially. New results suggest<br />
that DNA immunization is extremely effective in priming antigen-speciic B cells, establishing the stage for protective antibody<br />
responses, which are the critical components for an effective vaccine. More signiicantly, such indings are supported by clinical<br />
studies in humans, indicating a new era <strong>of</strong> gene-based vaccines.<br />
Lukas Flatz, MD<br />
Single <strong>Cell</strong> <strong>Gene</strong> Expression Proiling Reveals Qualitatively Distinct CD8 T cells Elicited by Different <strong>Gene</strong>based<br />
Vaccines<br />
Immunological techniques have dramatically advanced since the discovery <strong>of</strong> the immune system at the beginning <strong>of</strong> last<br />
century. However, to further improve immunotherapies and vaccination we have to better understand the events shaping an<br />
antigen encountering T cell on single cell base. We therefore used microluidics based single cell real-time PCR to analyze vaccine<br />
elicited antigen-speciic CD8 T cells <strong>of</strong> similar magnitude, phenotype and functionality as measured by conventional techniques.<br />
Remarkably, analysis <strong>of</strong> single cell gene expression proiles enabled the discrimination <strong>of</strong> previously unrecognized subsets <strong>of</strong><br />
central and effector memory CD8 T cells elicited by the three different gene-based vaccines. Single cell real-time PCR is a promising<br />
new technology increasing the resolution <strong>of</strong> T cell analysis.<br />
Saturday, May 18, 2013<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013<br />
73
<strong>Program</strong> Schedule, Saturday, May 18, 2013<br />
Aliasger K. Salem, PhD<br />
Adjuvants for Vector-Mediated Cancer Immunotherapy<br />
This presentation will cover the use <strong>of</strong> adjuvants such as biodegradable particles and oligonucleotides to enhance the magnitude <strong>of</strong><br />
antigen-speciic anti-tumor CD8+ T-cell responses generated from treatment with adenovirus based vaccines. The presentation will<br />
also discuss the use <strong>of</strong> strategies to suppress negative regulatory elements within the tumor such as myeloid derived suppressor<br />
cells (MDSCs) and regulatory T-cells (Treg) to augment the anti-tumor activity generated by treatment with adenovirus based<br />
vaccines.<br />
Andrew Geall, PhD<br />
Non-viral Delivery <strong>of</strong> Self-amplifying RNA Vaccines<br />
Novartis has developed the SAM(tm) vaccine platform. The Platform takes advantage <strong>of</strong> cell-free RNA production from a<br />
transcription reaction and delivery with a synthetic delivery system. The broad utility <strong>of</strong> this novel vaccine technology has been<br />
demonstrated with genes encoding antigens from several pathogens and found to elicit broad and potent protective immune<br />
responses. Responses are comparable to a viral delivery technology, but without the inherent limitations <strong>of</strong> viral vectors.<br />
Scientific Symposium 433<br />
1:45 pm - 3:45 pm<br />
ROOM: BALLROOM D<br />
Host <strong>Cell</strong> Responses to Viral Vectors<br />
CO-CHAIRS: John T. Gray, PhD and Dmitry M. Shayakhmetov, PhD<br />
SPEAKERS<br />
Vojo Deretic, PhD<br />
Autophagy - A Biological Paradigm for Everyone<br />
Autophagy is a homeostatic process that continuously cleanses the cytosol by removing defunct organelles, protein aggregates,<br />
microbial invaders, and endogenous sources <strong>of</strong> inlammation. It also supplies the cell with alternative sources <strong>of</strong> energy and<br />
nutrients by auto-digestion <strong>of</strong> the cytoplasm during starvation. Autophagy is <strong>of</strong> signiicance in cancer, aging, neurodegeneration,<br />
metabolic disorders, inlammatory disease such as Crohn’s disease, and infectious diseases. The presentation will cover the<br />
pathway, its connections to disease, and focus on anti-inlammatory aspects <strong>of</strong> autophagy.<br />
Roland W. Herzog, PhD<br />
Innate Immunity to AAV Vectors<br />
Rapid sensing <strong>of</strong> pathogen-associated molecular patterns by the innate immune system is a mechanism to quickly shut down<br />
a viral infection via inlammatory responses or IFN I signaling. While a major concern for other viral gene transfer vectors,<br />
innate responses to AAV are very short-lived and weak. Nonetheless, TLR9 sensing <strong>of</strong> the AAV genome has emerged as a critical<br />
component <strong>of</strong> the response, and evidence for IFN I induction, induction <strong>of</strong> NF-kB and expression <strong>of</strong> inlammatory cytokines has<br />
been obtained. Importantly, immune responses to vector and in some cases also the transgene product are impacted by innate<br />
immunity.<br />
Saturday, May 18, 2013<br />
John C. Bell, PhD<br />
Innate Immunity and Oncolytic Viruses<br />
Oncolytic Viruses have been engineered or selected to speciically replicate in and kill tumour cells. This selectivity is <strong>of</strong>ten based<br />
upon aberrant signalling pathways that are characteristic <strong>of</strong> malignant cells. However, normal cells found within the tumour<br />
microenvironment are exposed to constitutive and elevated levels <strong>of</strong> cancer cell derived growth factors leading to a disruption <strong>of</strong><br />
normal which in turn can lead to overstimulation <strong>of</strong> normal cellular regulatory pathways. We have found that both neo-vascular<br />
endothelial cells and cancer associated ibroblasts are sensitized to oncolytic virus infection and killing following exposure to<br />
tumour cell derived growth factors. The molecular and biological consequences <strong>of</strong> this sensitization will be discussed.<br />
Dmitry M. Shayakhmetov, PhD<br />
Sensing <strong>of</strong> Endosomal Rupture by the Host<br />
To achieve therapeutic beneits using gene delivery vectors, both viral and non-viral vectors must be able to eficiently<br />
penetrate through the plasma membrane <strong>of</strong> target cells and deliver the therapeutic cargo into cytosolic and/or nuclear cellular<br />
compartments. However, dedicated molecular sensors may exist that detect perturbations <strong>of</strong> the integrity <strong>of</strong> the plasma membrane<br />
and endosomal compartments within the cell. When triggered, these sensors activate innate immune defense mechanisms which<br />
can limit the eficacy <strong>of</strong> the therapy. We will review current understanding <strong>of</strong> the molecular mechanisms that are triggered within<br />
the cell upon virus entry, and present new data on molecular sensors <strong>of</strong> adenovirus-mediated endosome rupture.<br />
74<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013
<strong>Program</strong> Schedule, Saturday, May 18, 2013<br />
Scientific Symposium 434<br />
1:45 pm - 3:45 pm<br />
ROOM: 151 ABCG<br />
Lessons Learned from the Manufacture and Clinical Translation <strong>of</strong> <strong>Gene</strong> and <strong>Cell</strong> <strong>Therapy</strong><br />
Products<br />
CO-CHAIRS: <strong>Gene</strong> Liau, PhD and Gabor Veres, PhD<br />
SPEAKERS<br />
Barrie J. Carter, PhD<br />
Overview on Product and Clinical Translation<br />
After two decades <strong>of</strong> development, the irst gene therapy in the western world that was recently approved for marketing in Europe<br />
was an rAAV, glybera. The progress over these two decades in developing and testing gene therapy products will be reviewed using<br />
rAAV as the illustrative example. Substantial progress has been made in scaled up production <strong>of</strong> rAAV vectors, at least ive different<br />
manufacturing systems have been employed to advance products into clinical testing. The puriication and quality testing <strong>of</strong> rAAV<br />
vectors has advanced signiicantly and as more products approach licensing analytics for rAAV will be further reined. Pathways<br />
for preclincal testing <strong>of</strong> rAAV for safety, toxicity and biodistribution are established. Importantly, clinical studies with rAAV have<br />
generated clear evidence that clinical beneits can be gained and have also illuminated some important considerations such as host<br />
immune responses.<br />
Harold Petry, PhD<br />
Lessons from the Glybera ® European Approval Process to Build a <strong>Gene</strong> <strong>Therapy</strong> Product Pipeline<br />
The presentation will cover the development <strong>of</strong> Glybera ® , a gene therapy product based on the use <strong>of</strong> recombinant Adenoassociated<br />
virus for gene delivery, designed for patients with Lipoprotein Lipase Deiciency. The European Medicines Agency’s<br />
Committee for Medicinal Products for Human Use (CHMP) has approved authorisation <strong>of</strong> Glybera (alipogene tiparvovec) for<br />
marketing in the European Union. It is intended to treat lipoprotein lipase (LPL) deiciency in patients with severe or multiple<br />
pancreatitis attacks, despite dietary fat restrictions. Hurdles which were recognized during the development program towards<br />
market authorization will be discussed.<br />
Bruce L. Levine, PhD<br />
<strong>Gene</strong>ration <strong>of</strong> T <strong>Cell</strong>s that Engraft, Expand and Persist<br />
Dr. Levine directs the Clinical <strong>Cell</strong> and Vaccine Production Facility and is an Associate Pr<strong>of</strong>essor in the Department <strong>of</strong> Pathology<br />
and Laboratory Medicine at the University <strong>of</strong> Pennsylvania. The CVPF develops, manufactures and tests novel cell and gene<br />
therapies in clinical trials at Penn and collaborating institutions. Critical components for generation <strong>of</strong> cells with non-homologous<br />
function include 1) source and isolation <strong>of</strong> cells, 2) method <strong>of</strong> activation, 3) method <strong>of</strong> expansion, 4) characterization and testing. T<br />
lymphocytes can be activated, cultured, and endowed with novel functions ex vivo. Ex vivo modiied cells can engraft, signiicantly<br />
expand in vivo by >1000 fold, mediate potent clinical responses, and can survive for years.<br />
Raymond T. Bartus, PhD<br />
CERE-120 (AAV-neurturin) for the Treatment <strong>of</strong> Parkinson’s Disease: Experience from 4 Clinical Trials and<br />
Human Autopsy Data<br />
CERE-120 is an AAV serotype 2 vector expressing the gene for human neurturin, a neurotrophic factor with the natural ability<br />
to protect dying dopaminergic neurons in Parkinson’s patients from cell death and stimulate their ability to produce tyrosine<br />
hydroxylase (TH), an enzyme necessary dopamine synthesis. Since CERE-120 can inhibit cell death and stimulate dopamine, it has<br />
the potential ability to provide both symptomatic improvement as well as slow the neurodegenerative process, hence be disease<br />
modifying. Ceregene has completed over 24 preclinical studies on CERE-120 in multiple animal models <strong>of</strong> Parkinson’s disease as<br />
well as multiple preclinical toxicology studies. Ceregene has completed 4 CERE-120 clinical trials including 2 Phase 1 studies and 2<br />
Phase 2/2b studies in Patients with moderately advanced Parkinson’s disease. Data from these studies as well as from the analysis<br />
<strong>of</strong> 4 brains obtained at autopsy from subjects treat with CERE-120 will be discussed.<br />
Saturday, May 18, 2013<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013<br />
75
<strong>Program</strong> Schedule, Saturday, May 18, 2013<br />
Scientific Symposium 435<br />
1:45 pm - 3:45 pm<br />
ROOM: BALLROOM C<br />
Technological Advances in Non-Viral <strong>Gene</strong> Transfer & Targeting<br />
CO-CHAIRS: Richard Heller, PhD and Yoichi Negishi, PhD<br />
SPEAKERS<br />
Daniel Scherman, PhD<br />
Improved Eficiency Biosafe Plasmid for Sustained Expression in Various Tissues in Non-Viral <strong>Gene</strong> <strong>Therapy</strong><br />
Nonviral gene therapy requires plasmid eukaryotic expression vectors that meet deined criteria such as biosafety and low<br />
production cost. We have designed a novel antibiotic-free selection system, the pFAR plasmids, which is based on the suppression <strong>of</strong><br />
a chromosomal amber mutation in the essential thyA gene by a plasmid-borne function. The electrotransfer <strong>of</strong> LUC-encoding pFAR<br />
into muscle led to high luciferase activities, and in skin and transplanted tumors, transgene expression levels were at least one log<br />
superior and more sustained with pFAR plasmid electrotransfer, as compared with a plasmid carrying a kanamycin resistance gene.<br />
Most importantly, pFAR plasmids hydrodynamic delivery to liver lead to more than 9 months high and stable transgene expression,<br />
thus avoiding the classically observed liver gene extinction, by a process whose mechanism will be analyzed.<br />
Harvinder Singh Gill, PhD<br />
Microneedles for <strong>Gene</strong> and Oligonucleotide Delivery<br />
Tens to hundreds <strong>of</strong> minimally invasive microneedles conigured in an array-format with controlled spacing can be used to<br />
uniformly deliver genes and oligonucleotides to tissues for therapeutic and diagnostic applications. This presentation will describe<br />
our results from the use <strong>of</strong> microneedle arrays to deliver a gene-based hepatitis C vaccine to the skin in a mouse model, and the<br />
ability <strong>of</strong> microneedles to deliver oligonucleotides into cells situated in 3-D tissues.<br />
Andrew M. Scharenberg, MD<br />
High Eficiency Genome Editing Using Endonuclease/Exonuclease Co-expression<br />
An important factor in applying gene editing technologies in translational applications is the eficiency at which a target gene can<br />
be modiied in the desired cell population. We have explored the use <strong>of</strong> exonuclease/endonuclease co-expression as a means to<br />
increase the eficiency <strong>of</strong> gene editing by several endonuclease platforms, and in both tumor and primary cell types. Results from<br />
evaluation <strong>of</strong> several different exonuclease/endonuclease platform combinations, along with mechanistic analysis <strong>of</strong> how speciic<br />
exonucleases are interacting with free DNA ends will be presented.<br />
Evan C. Unger, MD<br />
<strong>Gene</strong> Delivery with Ultrasound and Microbubbles<br />
Safe, highly eficient vectors for gene delivery should expand clinical applications <strong>of</strong> gene therapy. Ultrasound is widely used for<br />
diagnostic imaging and has therapeutic applications for heating tissues, increasing tissue and capillary permeability and other<br />
applications in drug and gene delivery. Microbubbles lower the thresh-hold <strong>of</strong> energy for cavitation by ultrasound. FDA approved<br />
ultrasound contrast agents are currently being tested as gene delivery agents, but existing agents probably do not bind the genetic<br />
materials. New microbubbles that speciically bind genetic materials are also being tested. In my presentation I will review current<br />
developments in this ield and touch upon opportunities for development <strong>of</strong> new agents.<br />
Saturday, May 18, 2013<br />
76<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013
<strong>Program</strong> Schedule, Saturday, May 18, 2013<br />
Poster Session III & Reception<br />
3:45 pm - 5:45 pm<br />
ROOM: EXHIBIT HALL C/D<br />
RNA Virus Vectors<br />
(Abstracts 517-535; see pages 127-128)<br />
AAV Vectors - III<br />
(Abstracts 536-551; see pages 128-129)<br />
Adenovirus and Other DNA Virus Vectors II<br />
(Abstracts 552-564; Abstract 561 has been moved to Poster Session I after Abstract 138; see pages 129-130)<br />
DNA Vectorology & <strong>Gene</strong> Targeting II<br />
(Abstracts 565-577; see pages 130-131)<br />
<strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> <strong>of</strong> Diabetes, Metabolic and <strong>Gene</strong>tic Diseases II<br />
(Abstracts 578-587; Abstract 588 has been withdrawn; see page 131)<br />
Neurologic <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> II<br />
(Abstracts 589-604; Abstract 590 has been moved to Poster Session II after Abstract 391; see pages 131-132)<br />
Musculo-skeletal <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> II<br />
(Abstracts 605-620; see page 133)<br />
Cancer - Immunotherapy III<br />
(Abstracts 621-633; see pages 133-134)<br />
Cancer-Oncolytic Viruses II<br />
(Abstracts 634-647; Abstract 646 Withdrawn from Presentation; see pages 134-135)<br />
Cancer-Targeted <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>: <strong>Cell</strong> <strong>Therapy</strong> / Immunotherapy / <strong>Cell</strong>ular Targets<br />
(Abstracts 648-660; see pages 135-136)<br />
Hematologic and Immunologic <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> II<br />
(Abstracts 661-674; Abstract 675 has been withdrawn from presentation; see pages 136-137)<br />
Late Breaking Abstracts III<br />
(Abstracts 733-756; Abstracts 734, 745, and 749 Withdrawn from Presentation; see pages 137-138)<br />
Saturday, May 18, 2013<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013<br />
77
ASGCT 16th Annual Meeting<br />
May 15-18, 2013<br />
Salt Palace Convention Center (Hall C/D)<br />
Salt Lake City, Utah<br />
Exhibit Hall Floor Plan<br />
78<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013
Exhibitor Descriptions<br />
Aldevron<br />
3233 15th Street South<br />
Fargo, ND 58104<br />
Phone: (701) 306-2675<br />
Fax: (701) 280-1642<br />
E-mail: vchambers@aldevron.com<br />
Website: www.aldevron.com<br />
Booth Number: 203<br />
Aldevron’s custom plasmid puriication service is used<br />
to support many cell and gene therapy applications. Visit<br />
with our team to learn about new options for bench scale,<br />
research grade, and clinical grade DNA production. Aldevron<br />
also provides ready-to-use plasmids including cytokine,<br />
reporter, and viral helper vectors. We can also produce<br />
recombinant proteins, mRNA, and genetic antibodies.<br />
Aldevron’s mission is to advance science and it would be a<br />
privilege to work with your group.<br />
AltheaDx<br />
3550 Dunhill Street<br />
San Diego, CA 92121<br />
Phone: (858) 224-7200<br />
Fax: (858) 224-7220<br />
E-mail: abud<strong>of</strong>f@altheadx.com<br />
Website: www.altheadx.com<br />
Booth Number: 109<br />
AltheaDx <strong>of</strong>fers comprehensive Biodistribution Studies<br />
under GLP, designed to determine the distribution and<br />
persistence <strong>of</strong> cellular and gene therapy and viral vector<br />
products at key tissue sites. Our custom designed real-time<br />
qPCR analysis provides a powerful tool for assessing the<br />
safety and eficacy <strong>of</strong> nucleic acid and cellular therapeutics.<br />
<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong><br />
555 East Wells Street<br />
Milwaukee, WI 53202<br />
Phone: (414) 278-1341<br />
Fax: (414) 276-3349<br />
E-mail: info@asgct.org<br />
Website: www.asgct.org<br />
Booth Number: 415<br />
Stop by the ASGCT membership booth to ind out where the<br />
2014 Annual Meeting will be held and to ind out more about<br />
ASGCT activities.<br />
Biomiga, Inc.<br />
10637 Roselle Street<br />
Suite C<br />
San Diego, CA 92121<br />
Phone: (858) 597-0602<br />
Fax: (858) 538-1698<br />
E-mail: info@biomiga.com<br />
Website: www.biomiga.com<br />
Booth Number: 413<br />
Featured products:<br />
- Virus Puriication Kits (Adeno virus, Adeno associate virus,<br />
Retrovirus and Lentivirus)<br />
- DNA/RNA Puriication Kits (Membrane and Magnetic<br />
beads).<br />
Contract services:<br />
- From gene to virus: gene synthesis, cloning, mutagenesis,<br />
plasmid puriication, virus production and validation.<br />
- shRNA design, gene knockdown validation, stable cell line<br />
generation and single cell cloning.<br />
BTX/Harvard Apparatus<br />
84 October Hill Road<br />
Holliston, MA 01746<br />
Phone: (800) 272-2775<br />
Fax: (508) 429-5732<br />
E-mail: support.btx@harvardapparatus.com<br />
Website: www. btxonline.com<br />
Booth Number: 404<br />
BTX Harvard Apparatus is a global manufacturer and<br />
distributor <strong>of</strong> electroporation and electr<strong>of</strong>usion systems<br />
and accessories. Our product <strong>of</strong>fering accommodates a<br />
wide range <strong>of</strong> standard applications along with specialty<br />
applications such as in vivo electroporation as well as large<br />
volume transfection and cell fusion.<br />
<strong>Cell</strong>Genix GmbH<br />
One New Hampshire Ave., Suite 125<br />
Portsmouth, NH 03801<br />
Phone: (603) 373-0408<br />
Fax: (603) 373-8104<br />
E-mail: mcdermott@cellgenix.com<br />
Website: www.cellgenix.com<br />
Booth Number: 303<br />
<strong>Cell</strong>Genix manufactures high quality cytokines and media<br />
for ex-vivo cell culture <strong>of</strong> Dendritic <strong>Cell</strong>s, T-<strong>Cell</strong>s, NK-<strong>Cell</strong>s,<br />
Hematopoietic stem cells, Chondrocytes and Mesenchymal<br />
stromal cells. Our products are manufactured in accordance<br />
with GMP guidelines and USP and are all component<br />
free providing reliability, safety and reproducible results<br />
for pre-clincal and clinical applications. <strong>Cell</strong>Genix has built<br />
strong partnerships with clinical centers world-wide in<br />
cancer and regenerative medicine.<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013<br />
79
Exhibitor Descriptions<br />
Charles River<br />
251 Ballardvale Street<br />
Wilmington, MA 01887<br />
Phone: 1-877-criver1<br />
Fax: (978) 988-9236<br />
E-mail: askcharlesriver@crl.com<br />
Website: www.criver.com<br />
Booth Number: 112<br />
Charles River supports biomedical researchers with<br />
services spanning the drug development continuum. Our<br />
comprehensive preclinical <strong>of</strong>ferings include the design,<br />
execution and documentation <strong>of</strong> GLP safety tests before<br />
and after clinical trials begin. These safety programs<br />
are designed to mitigate complications <strong>of</strong> cross-species<br />
reactivity and risk factors inherent to developing novel<br />
cellular therapeutics.<br />
Children’s Hospital <strong>of</strong> Philadelphia<br />
Room 5059<br />
Colket Translational Research Building<br />
3501 Civic Center Blvd.<br />
Philadelphia, PA 19147<br />
Phone: (267) 426-5256<br />
Fax: (215) 590-3660<br />
E-mail: sunj@email.chop.edu<br />
Website: www.research.chop.edu/programs/ccmt/<br />
Booth Number: 414<br />
CCMT is dedicated to the understanding, development, and<br />
application <strong>of</strong> gene and cell therapies and the promotion <strong>of</strong><br />
pr<strong>of</strong>essional and public education in the ield. The Center<br />
fosters a multidisciplinary approach among researchers<br />
and scientists working to discover new therapies in search<br />
<strong>of</strong> cures for debilitating and life threatening childhood<br />
disorders.<br />
Cincinnati Children’s Hospital Medical Center<br />
3333 Burnet Ave. ML 7013<br />
Cincinnati, OH 45229<br />
Phone: (513) 803-1066<br />
Fax: (513) 636-1446<br />
E-mail: han.vanderloo@cchmc.org<br />
Website: https://research.cchmc.org/translationalcores/<br />
Booth Number: 103<br />
The Cincinnati Children’s Hospital Translational<br />
Laboratories were established in 2003 to eficiently move<br />
innovative ideas from the bench to the bedside. We <strong>of</strong>fer a<br />
one-stop shop approach to support research, scale-up, GMP<br />
manufacturing, testing, quality assurance, and clinical trial<br />
support <strong>of</strong> your novel cell and gene therapies.<br />
First Biotech Inc.<br />
340 West State Street<br />
Athens, OH 45701<br />
Phone: (614) 600-3341<br />
Fax: (740) 597-1548<br />
E-mail: lhallak@irstbiotech.com<br />
Website: www.FirstBiotech.com<br />
Booth Number: 211<br />
First Biotech Inc. is a DNA research reagents company. It<br />
manufactures a high idelity First Mutation site directed<br />
DNA mutagenesis kit for large plasmids up to 50 kilobase<br />
pairs. The kit is based on a new patented technology that<br />
does not require traditional sub-cloning or cloning vectors.<br />
Florida Biologix<br />
13702 Innovation Drive<br />
Alachua, FL 32615<br />
Phone: (386) 418-8199<br />
Fax: (386) 462-7838<br />
E-mail: jiannarelli@cerhb.ul.edu<br />
Website: www.loridabiologix.ul.com<br />
Booth Number: 113<br />
Florida Biologix ® is a biologics contract development,<br />
manufacturing, and testing organization staffed with 65<br />
industry-experienced personnel in a 35,000 ft 2<br />
facility<br />
<strong>of</strong>fering a variety <strong>of</strong> services, including: cell line engineering,<br />
cell banking, process development, assay development,<br />
cGMP vector/protein production in mammalian and insect<br />
cells, cell therapies, aseptic illing, product testing, and<br />
regulatory support.<br />
GE Healthcare<br />
800 Centennial Avenue<br />
Piscataway, NJ 08854<br />
Phone: (800) 526-3593<br />
Fax: (877) 295-8102<br />
E-mail: liz.pita@ge.com<br />
Website: www.gelifesciences.com<br />
Booth Number: 214<br />
GenScript USA Inc.<br />
860 Centennial Ave<br />
Piscataway, NJ 08854<br />
Phone: (732) 885-9188<br />
Fax: (732) 210-0262<br />
E-mail: info@genscript.com<br />
Website: www.GenScript.com<br />
Booth Number: 403<br />
Founded in 2002, GenScript is now the world’s leading gene<br />
synthesis company, and is particularly skillful with long and/<br />
or complex sequences. Besides, GenScript <strong>of</strong>fers a variety<br />
<strong>of</strong> services and products for preclinical drug discovery.<br />
Headquartered in USA, GenScript has currently 1,300+<br />
employees worldwide with subsidiaries in Europe, Japan and<br />
China.<br />
80<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013
Exhibitor Descriptions<br />
HypOxygen<br />
5111 Pegasus Ct., Ste. H<br />
Frederick, MD 21704<br />
Phone: (877) 497-6994<br />
Fax: (301) 662-8096<br />
E-mail: info@hppoxygen.us<br />
Website: www.hypoxygen.us<br />
Booth Number: 416<br />
HypOxygen is featuring the HypOxystation hypoxia<br />
incubator, designed for physiological cell culture research.<br />
Scientists have precise control <strong>of</strong> oxygen, carbon dioxide,<br />
temperature, humidity and can manipulate cells in-situ. We<br />
will also be showcasing our new INCOmed CO2 incubators,<br />
which boast Peltier technology, accurate infrared (NDIR)<br />
CO2 sensors and custom options.<br />
Lovelace Respiratory Research Institute<br />
2425 Ridgecrest Dr. SE<br />
Albuquerque, NM 87108<br />
Phone: (505) 348-9534<br />
Fax: (505) 348-4983<br />
E-mail: aholley@lrri.org<br />
Website: www.lrri.org<br />
Booth Number: 406<br />
The Lovelace Respiratory Research Institute is a private notfor-proit<br />
biomedical research organization. Lovelace <strong>of</strong>fers<br />
animal models <strong>of</strong> disease, evaluation <strong>of</strong> eficacy <strong>of</strong> potential<br />
new therapeutics, and pre-clinical assessments including<br />
evaluation <strong>of</strong> pharmacokinetics/biodistribution in large and<br />
small animal models. Studies are routinely performed under<br />
GLP guidelines.<br />
Mary Ann Liebert, Inc.<br />
140 Huguenot Street<br />
New Rochelle, NY 10801<br />
Phone: (914) 740-2100<br />
Fax: (914) 740-2101<br />
E-mail: info@liebertpub.com<br />
Website: www.liebertpub.com<br />
Booth Number: 107<br />
Mary Ann Liebert Inc. publishes the premier journal<br />
program in gene therapy, consisting <strong>of</strong> Human <strong>Gene</strong> <strong>Therapy</strong>,<br />
HGT Methods, & new in 2013, HGT Clinical Development.<br />
This expanded coverage provides unprecedented access<br />
to research and applications critical to the evolution <strong>of</strong> the<br />
ield! Visit us @ booth# 107.<br />
Miltenyi Biotec<br />
2303 Lindbergh Street<br />
Auburn, CA 95602<br />
Phone: (530) 887-5385<br />
Fax: (530) 888-8925<br />
E-mail: tausha@miltenyibiotec.com<br />
Website: www.miltenyibiotec.com<br />
Booth Number: 202<br />
Miltenyi Biotec provides MACS® Technology, the gold<br />
standard in cell separation for research and clinical scale,<br />
as well as sample preparation, cell analysis and molecular<br />
biology products and services to the biomedical research<br />
community. The company also manufactures cGMP<br />
custom reagents, antibodies, proteins, peptides, and other<br />
molecules.<br />
MPI Research<br />
54943 North Main Street<br />
Mattawan, MI 49071<br />
Phone: (269) 668-3336<br />
Fax: (269) 668-4151<br />
E-mail: info@mpiresearch.com<br />
Website: www.mpiresearch.com<br />
Booth Number: 201<br />
MPI Research is a preclinical and early clinical CRO that<br />
provides discovery, surgery, safety evaluation, bioanalytical,<br />
and analytical services. We exceed expectations<br />
through consistency and quality, with a commitment<br />
to communication and innovation, delivering beneits<br />
throughout all phases <strong>of</strong> development. Learn how we can go<br />
beyond for you at www.mpiresearch.com.<br />
NanoSight<br />
6660 N. High Street<br />
Suite 2A<br />
Worthington, OH 43085<br />
Phone: (614) 888-0023<br />
Fax: (614) 987-0045<br />
E-mail: admin@nanosight.com<br />
Website: www.nanosight.com<br />
Booth Number: 204<br />
NanoSight manufactures instruments for Counting, Sizing<br />
and Visualizing Nanoparticles from 10 nm to 2.0 µm. Size<br />
& Concentration measurements along with the ability to<br />
visualize the nanoparticles in real time and a captured video<br />
<strong>of</strong> the sample being analyzed are created for each analysis.<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013<br />
81
Exhibitor Descriptions<br />
National <strong>Gene</strong> Vector Biorepository (NGVB)<br />
980 W. Walnut Street R3 C602<br />
Indianapolis, IN 46202<br />
Phone: (317) 274-4519<br />
Fax: (317) 278-3327<br />
E-mail: lrubin@iupui.edu<br />
Website: https://www.ngvbcc.org<br />
Booth Number: 412<br />
The National <strong>Gene</strong> Vector Biorepository provides support<br />
for gene therapy research by <strong>of</strong>fering several services. Check<br />
out the reagent repository comprising cell lines, plasmids,<br />
vectors and other tools used in gene therapy research. There<br />
are archiving services to meet FDA requirements. The NGVB<br />
will store clinical post-trial samples, inal product reserves,<br />
and master cell banks as well as pharmacology/toxicology<br />
samples. A pharm/tox database containing gene therapy<br />
tox studies is a valuable resource. We <strong>of</strong>fer assistance<br />
with insertional mutagenesis analysis through the SeqMap<br />
Bioinformatics Tool. Check out the <strong>Gene</strong> <strong>Therapy</strong> Data Map a<br />
tool for locating gene therapy research. Go to https://www.<br />
ngvbcc.org or come and see us in Booth 412.<br />
Nature Publishing Group<br />
75 Varick Street, 9th Floor<br />
New York, NY 10013-1917<br />
Phone: (212) 726-9200<br />
Fax: (212) 689-9711<br />
E-mail: institutions@nature.com<br />
Website: www.nature.com<br />
Booth Number: 402<br />
Nature Publishing Group brings you leading scientiic and<br />
medical research. The NPG portfolio combines the continued<br />
excellence <strong>of</strong> Nature, its associated research and review<br />
journals, and 50 leading academic and society journals in the<br />
life, physical and clinical sciences. Visit Booth 402 for free<br />
sample copies.<br />
Nature Technology Corporation<br />
4701 Innovation Drive<br />
Lincoln, NE 68521<br />
Phone: (402) 323-6289<br />
Fax: (402) 323-6292<br />
E-mail: hodgson@natx.com<br />
Website: www.natx.com<br />
Booth Number: 312<br />
Nature Technology Corporation is a biotechnology<br />
company that provides industry partners with world class<br />
technology platforms including safe and eficacious nonviral<br />
vectors (plasmids) and processes for gene therapy<br />
and DNA vaccination vector production. NTC also provides<br />
cost effective outsourcing <strong>of</strong> product development services<br />
including synthetic gene design, vector construction, plasmid<br />
DNA manufacturing and recombinant proteins.<br />
Nexcelom Bioscience<br />
360 Merrimack Street, Building 9<br />
Lawrence, MA 01843<br />
Phone: (978) 327-5340<br />
Fax: (978) 327-5341<br />
E-mail: lvavra@nexcelom.com<br />
Website: www.nexcelom.com<br />
Booth Number: 114<br />
Nexcelom’s <strong>Cell</strong>ometer line <strong>of</strong> simple-to-use cell counters<br />
automate manual cell counting procedures by obtaining<br />
accurate counts, viability, and cell sizes in less than 30<br />
seconds & only 20uL <strong>of</strong> sample. Fluorescence detection<br />
capabilities enable fast & simple determination <strong>of</strong> GFP<br />
transfection rates, PI-viability, & direct counting <strong>of</strong> WBCs<br />
without lysing.<br />
NHLBI <strong>Gene</strong> <strong>Therapy</strong> Resource <strong>Program</strong><br />
2 Rockledge Center, 6701 Rockledge Drive<br />
Bethesda, MD 20892-7940<br />
Phone: (301) 435-0477<br />
Fax: (301) 628-1929<br />
E-mail: gtrpccc@s-3.com<br />
Website: www.gtrp.org<br />
Booth Number: 408<br />
The NHLBI <strong>Gene</strong> <strong>Therapy</strong> Resource <strong>Program</strong> (GTRP)<br />
facilitates the translation <strong>of</strong> gene therapy research<br />
into clinical interventions primarily in heart, lung, and<br />
blood diseases. Services provided by the <strong>Program</strong><br />
include preclinical and clinical-grade vector production,<br />
pharmacology/toxicology testing, immunology testing,<br />
assistance with regulatory activities, and partial inancial<br />
support <strong>of</strong> clinical trials.<br />
NIH Office <strong>of</strong> Biotechnology Activities<br />
6705 Rockledge Dr.<br />
Suite 750<br />
Bethesda, MD 20892-7985<br />
Phone: (301) 496-9838<br />
Fax: (301) 496-9839<br />
E-mail: oba@od.nih.gov<br />
Website: http://oba.od.nih.gov<br />
Booth Number: 105<br />
NIH Ofice <strong>of</strong> Biotechnology Activities staff is available to<br />
answer questions about human gene transfer research<br />
under the NIH Guidelines and provide information about<br />
GeMCRIS, a web-based, information resource and analytical<br />
tool that provides information about HGT trials and enables<br />
investigators to ile adverse event and annual reports<br />
electronically.<br />
82<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013
Exhibitor Descriptions<br />
Panasonic Healthcare Company <strong>of</strong> North America<br />
1300 Mitchell Drive, Suite A<br />
Wood Dale, IL 60191<br />
Phone: (800) 858-8442<br />
Fax: (630) 238-0074<br />
E-mail: phc_info@us.panasonic.com<br />
Website: www.panasonic.com/biomedical<br />
Booth Number: 411<br />
Panasonic Healthcare Company is a global, leading provider<br />
<strong>of</strong> energy, environment, and lifestyle applications with<br />
laboratory equipment for pharmaceutical, life science and<br />
biotechnology applications. Product lines include spaceeficient<br />
VIP ® ultra-low temperature freezers, cryogenic<br />
freezers, pharmaceutical and medical refrigerators, cell<br />
culture incubators, plant growth chambers, and portable<br />
autoclaves.<br />
Penn Vector Core<br />
125 South 31st Street, Suite 2000<br />
University <strong>of</strong> Pennsylvania<br />
<strong>Gene</strong> <strong>Therapy</strong> <strong>Program</strong><br />
Perelman School <strong>of</strong> Medicine<br />
Philadelphia, PA 19104<br />
Phone: (215) 573-0633<br />
Fax: (215) 261-2442<br />
E-mail: aksandhu@mail.med.upenn.edu<br />
Website: http://www.med.upenn.edu/gtp/vector_core.shtml<br />
Booth Number: 410<br />
Penn Vector Core is a state-<strong>of</strong>-the-art viral vector production<br />
facility for investigators both within and external to the<br />
University <strong>of</strong> Pennsylvania. The main objective <strong>of</strong> the Core is<br />
to make various types <strong>of</strong> high quality viral vectors for gene<br />
transfer/Vaccine studies at the preclinical and basic research<br />
level.<br />
PerkinElmer<br />
68 Elm Street<br />
Hopkinton, MA 01748<br />
Phone: (781) 663-6900<br />
E-mail: CustomerCareUS@perkinelmer.com<br />
Website: www.perkinelmer.com<br />
Booth Number: 101<br />
Biological complexity raises questions requiring<br />
translational research from the well, to the cell, to the animal<br />
and back again. PerkinElmer enables you to approach<br />
your target from multiple perspectives: locate, detect and<br />
quantitate your biology <strong>of</strong> interest; analyze and understand<br />
it in wider physiological contexts.<br />
PolyPlus-Transfection<br />
Bioparc BP 90018<br />
Illkirch SA 67401<br />
France<br />
Phone: 33-(0)3-90-40-61-80<br />
Fax: 33-(0)3-90-40-61-81<br />
E-mail: info@polyplus-transfection.com<br />
Website: www.polyplus-transfection.com<br />
Booth Number: 104<br />
Polyplus transfection provides state-<strong>of</strong>-the-art quality<br />
transfection reagents for in vitro and in vivo DNA and<br />
siRNA delivery. Polyplus also <strong>of</strong>fers qualiied reagents for<br />
bioproduction including virus production for the clinic and<br />
supplies GMP grade reagents for human gene therapy and<br />
cell therapy clinical trials.<br />
REGENX Biosciences, LLC<br />
750 17th Street NW<br />
Suite 1100<br />
Washington, DC 20006<br />
Phone: (202) 778-2365<br />
Fax: (202) 785-7439<br />
E-mail: jbrown@regenxbio.com<br />
Website: www.regenxbio.com<br />
Booth Number: 401<br />
REGENX BioSciences is leading the effort to translate<br />
promising gene delivery applications into a pipeline <strong>of</strong><br />
next generation personalized therapies for a range <strong>of</strong><br />
severe diseases with serious unmet needs. We believe that<br />
the NAV technology, to which we have exclusive rights,<br />
represents the potential promise <strong>of</strong> curing the root cause <strong>of</strong><br />
disease rather than the symptoms, and we are committed to<br />
establishing best in class standards for our NAV vectors.<br />
SAFC<br />
BioReliance by SAFC<br />
Glasgow G20 0XA<br />
UNITED KINGDOM<br />
Phone: +44(0)141 576 2462<br />
Fax: +44(0)141 948 0000<br />
E-mail: susan.livingston@bioreliance.com<br />
Website: www.safcglobal.com<br />
Booth Number: 115<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013<br />
83
Exhibitor Descriptions<br />
St. Jude Children’s Research Hospital<br />
262 Danny Thomas Place<br />
Memphis, TN 38015<br />
Phone: (901) 595-2750<br />
Fax: (901) 595-5376<br />
E-mail: postdoc@stjude.org<br />
Website: www.stjude.org/research<br />
Booth Number: 215<br />
At St Jude Children ’s Research Hospital, a non-proit<br />
biomedical research institution in Memphis, TN, 190 basic<br />
science and clinical researchers investigate the molecular<br />
basis <strong>of</strong> normal cellular and diseased processes. Visit our<br />
booth to discuss and apply for Postdoctoral Fellowship<br />
positions.<br />
Contact Information:<br />
St. Jude Children’s Research Hospital<br />
Ofice <strong>of</strong> Academic <strong>Program</strong>s<br />
262 Danny Thomas Place, MS 276<br />
Memphis, TN 38105<br />
Email: postdoc@stjude.org<br />
http://www.stjude.org/research<br />
The Baker Company<br />
P. O. Drawer E<br />
Sanford, ME 04073<br />
Phone: (800) 992-2537<br />
Fax: (207) 324-3869<br />
E-mail: bakerco@bakerco.com<br />
Website: www.bakerco.com<br />
Booth Number: 302 & 304<br />
For 60 years, The Baker Company has delivered high quality<br />
contamination control products to meet the unique and<br />
evolving needs <strong>of</strong> laboratory pr<strong>of</strong>essionals. Learn about<br />
our new generation <strong>of</strong> high-performance, precision-crafted<br />
biomedical refrigeration, controlled atmosphere and<br />
Ruskinn hypoxia workstations on our website at www.<br />
bakerco.com.<br />
TriLink BioTechnologies, Inc.<br />
9955 Mesa Rim Road<br />
San Diego, CA 92121<br />
Phone: (858) 546-0004<br />
Fax: (858) 546-0020<br />
E-mail: info@trilinkbiotech.com<br />
Website: www.trilinkbiotech.com<br />
Booth Number: 301<br />
TriLink manufactures custom oligonucleotides, modiied<br />
nucleoside triphosphates and CleanAmp PCR products<br />
for the diagnostic and OEM markets. In addition, custom<br />
chemistry, long RNA transcript synthesis and contract<br />
research services and ISO/QSR compliant cGMP production<br />
facilities are <strong>of</strong>fered. TriLink’s solutions help advance drug<br />
discovery and biomedical research.<br />
UAB Vector Production Facility<br />
550 11th Street South<br />
Birmingham, AL 35294-4558<br />
Phone: (205) 975-9871<br />
Fax: (205) 934-9511<br />
E-mail: tvstrong@uab.edu<br />
Website: www.uab.edu/vector/<br />
Booth Number: 316<br />
The goal <strong>of</strong> the UAB Vector Production Facility (VPF) is to<br />
accelerate the translation <strong>of</strong> novel biological therapies into<br />
Phase I human clinical trials. The UAB VPF produces largescale,<br />
high quality viral vectors, cell banks and recombinant<br />
proteins for research and clinical applications, in accordance<br />
with current Good Manufacturing Practices.<br />
UNC <strong>Gene</strong> <strong>Therapy</strong> Vector Core<br />
7119 Thurston Bowles<br />
Chapel Hill, NC 27599<br />
Phone: (919) 962-3285<br />
Fax: (919) 843-9410<br />
E-mail: vectorcore@unc.edu<br />
Website: http://genetherapy.unc.edu/jvl.htm<br />
Booth Number: 102<br />
University <strong>of</strong> Iowa/<strong>Gene</strong> Transfer Vector Core<br />
<strong>Gene</strong> Transfer Vector Core<br />
500 Newton Road<br />
221 EMRB<br />
Iowa City, IA 52242<br />
Phone: (319) 335-6726<br />
Fax: (319) 335-6895<br />
E-mail: maria-scheel@uiowa.edu<br />
Website: www.uiowa.edu/~gene/<br />
Booth Number: 313<br />
The GTVC’s objective is to support investigators in the<br />
use <strong>of</strong> gene transfer technologies. The Core utilizes<br />
molecular biology techniques to engineer and develop viral<br />
vectors based on multiple vector systems. Core staff and<br />
investigators work together allowing for cross fertilization<br />
<strong>of</strong> ideas, technical advancements, and innovations in vector<br />
design.<br />
84<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013
Exhibitor Descriptions<br />
University <strong>of</strong> Utah Blood & Marrow Transplant<br />
<strong>Program</strong> - <strong>Cell</strong> <strong>Therapy</strong> & Regenerative Medicine<br />
Facility<br />
675 Arapeen Dr., Suite 300<br />
Salt Lake City, UT 84108<br />
Phone: (801) 585-6262<br />
Fax: (801) 585-3222<br />
E-mail: joanna.reems@hsc.utah.edu<br />
Website: http://medicine.utah.edu/cell/<br />
Booth Number: 314<br />
The <strong>Cell</strong> <strong>Therapy</strong> and Regenerative Medicine <strong>Program</strong> at<br />
the University <strong>of</strong> Utah supports Hematopoietic stem cell<br />
transplants and delivers innovative cellular and tissue<br />
engineered products to patients by providing comprehensive<br />
“bench to bedside” services that coordinate the efforts <strong>of</strong><br />
clinicians, researchers, and bioengineers.<br />
VGXI, Inc.<br />
2700 Research Forest Drive<br />
Suite 180<br />
The Woodlands, TX 77381<br />
Phone: (281) 296-7300<br />
Fax: (281) 296-7333<br />
E-mail: info@vgxii.com<br />
Website: www.vgxii.com<br />
Booth Number: 116<br />
VGXI Inc. is a leading provider <strong>of</strong> DNA plasmid<br />
manufacturing and development services for DNA vaccine<br />
and gene therapy clinical research. The Company has an<br />
outstanding track record <strong>of</strong> success manufacturing plasmid<br />
products under GMP for clients in the US, Europe, and<br />
Australia.<br />
Waisman Biomanufacturing<br />
1500 Highland Avenue<br />
Madison, WI 53705<br />
Phone: (608) 263-8940<br />
E-mail: info@gmpbio.org<br />
Website: www.gmpbio.org<br />
Booth Number: 213<br />
Waisman Biomanufacturing has in-depth experience<br />
manufacturing a wide range <strong>of</strong> biotherapeutics for human<br />
Phase I/II clinical trials including gene therapeutics, cell<br />
therapeutics, vaccines, and recombinant proteins. Services<br />
include process development through cGMP production<br />
and ill/inish capabilities— all with full quality system<br />
support—and comprehensive support for your IND iling.<br />
Wilson Wolf Corporation<br />
33 5th Avenue, NW<br />
New Brighton, MN 55112<br />
Phone: (651) 628-9259<br />
Fax: (651) 628-9259<br />
E-mail: info@wilsonwolf.com<br />
Website: www.wilsonwolf.com<br />
Booth Number: 111<br />
Wilson Wolf created “G-Rex” technology to simplify and<br />
reduce the cost cell production for Adoptive <strong>Cell</strong> <strong>Therapy</strong>.<br />
<strong>Cell</strong> production in G-Rex devices saves material and labor<br />
while simultaneously shortening production time, allowing<br />
the most practical and cost effective process for research and<br />
clinical work. Stop by our booth for details.<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013<br />
85
Exhibitor Information<br />
<strong>Gene</strong>ral Information<br />
The Exhibit Hall is located in Hall C/D <strong>of</strong> the Salt Palace<br />
Convention Center. All participants are urged to allow<br />
adequate time to visit the exhibits. The companies,<br />
organizations and institutions exhibiting at the ASGCT<br />
16th Annual Meeting will be providing you with the latest<br />
information on products and services directly related to<br />
your pr<strong>of</strong>essional needs, as well as recruiting for open<br />
positions.<br />
Exhibit Dates<br />
Exhibits will be open Wednesday, May 15 through Friday,<br />
May 17, 2013.<br />
Exhibitor Registration<br />
The Exhibitor Registration Desk is located at the East<br />
Registration Area – by the East Entrance on the 2nd loor <strong>of</strong><br />
the Salt Palace Convention Center<br />
Exhibitor Registration Desk Hours<br />
Tuesday, May 14 5:00 pm – 7:00 pm<br />
Wednesday, May 15 7:00 am – 7:30 pm<br />
Thursday, May 16 7:00 am – 6:30 pm<br />
Friday, May 17<br />
7:30 am – 7:30 pm<br />
Exhibit Move In<br />
Wednesday, May 15 9:00 am – 5:00 pm<br />
Exhibit Hall Hours<br />
Wednesday, May 15 5:15 pm – 7:15 pm<br />
Thursday, May 16 9:35 am – 10:30 am<br />
Thursday, May 16 4:00 pm – 6:00 pm<br />
Friday, May 17<br />
9:00 am – 9:45 am<br />
Friday, May 17<br />
5:30 pm – 7:30 pm<br />
Exhibit Move Out<br />
Friday, May 17<br />
7:30 pm – 9:30 pm<br />
Note: All exhibit booths must be staffed during hall hours.<br />
Packing <strong>of</strong> equipment/literature and the dismantling <strong>of</strong><br />
exhibits will not be permitted until after 7:30 pm on Friday,<br />
May 17, 2013.<br />
Exhibitor Badge Policy<br />
Admission to the Exhibit Hall will be by name badge only.<br />
Security guards will monitor exhibit hall entrances for<br />
proper I.D. Exhibit booth personnel must show an <strong>of</strong>icial<br />
ASGCT exhibitor name badge in order to gain access to the<br />
exhibit hall during installation, show, or dismantlement<br />
hours. Independent contractor personnel, hired by<br />
an exhibitor to install and dismantle their display, do<br />
not need to show exhibitor badges, but should possess<br />
identiication that they are employed by the independent<br />
contractor. If independent contractors do not possess proper<br />
identiication, they should obtain a name badge at the onsite<br />
exhibitor registration desk.<br />
Endorsement Disclaimer<br />
Products and services displayed in the Exhibit Hall or<br />
advertised in this program occur by contractual business<br />
arrangements between the ASGCT and participating<br />
companies and organizations. These arrangements do not<br />
constitute or imply an endorsement by the ASGCT <strong>of</strong> these<br />
products and services.<br />
86<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013
Abstract Directory, Wednesday, May 15, 2013<br />
Oral Abstract Session 130<br />
AAV Vector Development & Application<br />
3:15 PM - 5:15 PM<br />
Room: Ballroom A<br />
1 3:15 PM<br />
Identiication <strong>of</strong> Novel Vectors Capable <strong>of</strong> CNS<br />
Transduction in Adult Mice after Single Round<br />
Selection Using DNA Shufled AAV Capsid Library<br />
Sourav Choudhury, Stacy Maitland, Jennifer Ferreira, Miguel<br />
Sena-Esteves.<br />
2 3:30 PM<br />
Re-Engineering Capsid-Glycan Interactions Expands the<br />
CNS Transduction Proile <strong>of</strong> AAV Serotype 4<br />
Giridhar Murlidharan, Laura Sommerville, Troy Ghashghaei,<br />
Aravind Asokan.<br />
3 3:45 PM<br />
Directed Evolution <strong>of</strong> Adeno-Associated Virus for<br />
Enhanced <strong>Gene</strong> Delivery to Neural Stem <strong>Cell</strong>s In Vitro<br />
and In Vivo<br />
David V. Schaffer, Melissa A. Bartel, Jae-Hyung Jang, Tandis Vazin.<br />
4 4:00 PM<br />
A Rationally Engineered Novel Capsid Variant <strong>of</strong> AAV9<br />
for Pripheral Tissue-Detargeted and CNS-Directed<br />
Systemic <strong>Gene</strong> Delivery<br />
Li Zhong, Shaoyong Li, Kim Van Vliet, Mengxin Li, Jun Xie, Jia<br />
Li, Qin Su, Ran He, Yu Zhang, Huapeng Li, Dan Wang, Jason<br />
Goetzmann, Terence R. Flotte, Mavis Agbandje-McKenna,<br />
Guangping Gao.<br />
5 4:15 PM<br />
In Vivo Directed Evolution <strong>of</strong> a Novel Adeno-Associated<br />
Virus for Therapeutic Outer Retinal <strong>Gene</strong> Delivery from<br />
the Vitreous<br />
Leah C. Byrne, Deniz Dalkara, Ryan R. Klimczak, Meike Visel, Lu<br />
Yin, William H. Merigan, John G. Flannery, David V. Schaffer.<br />
6 4:30 PM<br />
Adeno-Associated Virus Capsid Motif That Inluences<br />
Tissue Speciic Vector Transduction In Vivo<br />
Jayme K. Warischalk, Richard J. Samulski.<br />
7 4:45 PM<br />
A Species Restricted rAAV Vector Obtained from Viral<br />
Capsid Shufling<br />
Leszek Lisowski, Kirk Chu, Mark A. Kay.<br />
8 5:00 PM<br />
Solving the T-<strong>Cell</strong> Problem: <strong>Gene</strong>ration <strong>of</strong> Immune<br />
Stealth AAV<br />
Thierry VandenDriessche, Frederico Mingozzi, Nisha Nair, Jessica<br />
Willems, Hanneke Evens, Omid Ghandeharian, Kathy High,<br />
Marinee K. Chuah.<br />
Oral Abstract Session 131<br />
Cancer Immunotherapy I<br />
3:15 PM - 5:15 PM<br />
Room: Ballroom B<br />
9 3:15 PM<br />
Enhanced DNA Vaccination Against a Tyrosinase (Tyr)<br />
Mediates Protection and Targets Myeloid-Derived<br />
Suppressor <strong>Cell</strong>s in a Syngeneic Murine Model<br />
Taron C. Gorham, Lavanya Mahadevan, Colleen Lucke, Randolph<br />
Lyde, Kenneth E. Ugen, Niranjan Y. Sardesai, Joseph J. Kim,<br />
Karuppiah Muthumani, David B. Weiner.<br />
10 3:30 PM<br />
First-in-Human Application <strong>of</strong> Sleeping Beauty System<br />
for <strong>Gene</strong> <strong>Therapy</strong><br />
Partow Kebriaei, Helen Huls, Harjeet Singh, Simon Olivares,<br />
Matthew J. Figliola, Margaret Dawson, Bipulendu Jena, Rineka<br />
Jackson, Doyle Bosque, Ian McNiece, Gabriela Rondon, Perry B.<br />
Hackett, Elizabeth J. Shpall, Richard E. Champlin, Laurence J. N.<br />
Cooper.<br />
11 3:45 PM<br />
AAV-Mediated CNS <strong>Gene</strong> Transfer <strong>of</strong> Bevacizumab<br />
Reduces Human Glioblastoma Growth and Increases<br />
Survival in Mice<br />
Martin J. Hicks, Kosuke Funato, Lan Wang, Eric Aronowitz,<br />
Jonathan P. Dyke, Douglas J. Ballon, Vivianne S. Tabar, David F.<br />
Havlicek, Esther Z. Frenk, Bishnu P. De, Maria J. Chiuchiolo, Dolan<br />
Sondhi, Neil R. Hackett, Stephen M. Kaminsky, Ronald G. Crystal.<br />
12 4:00 PM<br />
NKT <strong>Cell</strong>s as a Novel Platform for Cancer<br />
Immunotherapy with Chimeric Antigen Receptors<br />
Andras Heczey, Da<strong>of</strong>eng Liu, Amy Courtney, Ekaterina Marinova,<br />
Gengwen Tian, Eric Yvon, Jie Wei, Gianpietro Dotti, Leonid<br />
Metelitsa.<br />
13 4:15 PM<br />
Removal <strong>of</strong> Physical Barriers inside the Tumor<br />
Improves T-<strong>Cell</strong> <strong>Therapy</strong><br />
Hua Cao, Ines Beyer, Vy Phan-Lai, Nora Disis, Andre Lieber.<br />
14 4:30 PM<br />
Administration <strong>of</strong> GD2 Chimeric Antigen<br />
Receptor Modiied, Tri-Virus Speciic Cytotoxic T<br />
Lymphocytes after HLA Mismatched Allogeneic<br />
Stem <strong>Cell</strong> Transplantation for Relapsed, Refractory<br />
Neuroblastoma<br />
Doug Myers, Jiali Sun, Cliona M. Rooney, Natalia Lapteva, Adrian<br />
P. Gee, Gianpietro Dotti, Bambi Grilley, Malcolm K. Brenner, Robin<br />
Ryan.<br />
15 4:45 PM<br />
Nonclinical and Phase I Clinical Studies with a<br />
Regulated Adenoviral <strong>Gene</strong> Delivery <strong>of</strong> IL-12 Shows<br />
Promising Clinical Activity in Unresectable Stage III/IV<br />
Melanoma<br />
A. Vergara-Silva, J. Barrett, A. Marsh, R. Khosravi-Far, L. A. Martell,<br />
O. Hamid, T. Chan, T. D. Reed, H. Youssouian, J. Nemunaitis.<br />
Wednesday, May 15, 2013<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013<br />
87
Wednesday, May 15, 2013<br />
Abstract Directory, Wednesday, May 15, 2013<br />
Oral Abstract Session 131, continued<br />
Cancer Immunotherapy I<br />
3:15 PM - 5:15 PM<br />
Room: Ballroom B<br />
16 5:00 PM<br />
Effective and Safe <strong>Gene</strong>-Modiied T <strong>Cell</strong>s Expressing<br />
High Afinity NYESO-1 Speciic TCR Variants with<br />
Silencing Endogenous TCRs<br />
Sachiko Okamoto, Yasunori Amaishi, Hiroaki Ikeda, Hiroshi Shiku,<br />
Junichi Mineno.<br />
Oral Abstract Session 132<br />
Cancer-Oncolytic Viruses<br />
3:15 PM - 5:15 PM<br />
Room: 150 DEFG<br />
17 3:15 PM<br />
Intraperitoneal (IP) Administration <strong>of</strong> an Oncolytic<br />
Measles Virus (MV) Strain Expressing the Sodium<br />
Iodine Symporter <strong>Gene</strong> in Patients (pts) with Advanced<br />
Ovarian Cancer (OvCa)<br />
Evanthia Galanis, Pamela Atherton, Sean Dowdy, William Cliby,<br />
Paul Haluska, Jr, Harry Long, Ileana Aderca, Keith Knutson,<br />
Matthew Block, Mark Federspiel, Stephen Russell, Kah Whye Peng,<br />
Lynn Hartmann.<br />
18 3:30 PM<br />
In Vivo Expansion <strong>of</strong> a Recipient Population <strong>of</strong> <strong>Cell</strong><br />
Carriers Allows for Highly Effective Systemic Delivery <strong>of</strong><br />
Oncolytic Reovirus Even in the Presence <strong>of</strong> Neutralizing<br />
Antibody<br />
Timothy Kottke, Oliver Donnelly, Elizabeth Ilett, Jill Thompson,<br />
Rosa Diaz, Matt C<strong>of</strong>fey, Peter Selby, Hardev Pandha, Kevin<br />
Harrington, Alan Melcher, Richard Vile.<br />
19 3:45 PM<br />
Baseline Neutralizing Antibody Status Does Not Affect<br />
Intravenous Delivery <strong>of</strong> Oncolytic Vaccinia Pexa-Vec (JX-<br />
594) in Liver Cancer Patients<br />
Caroline J. Breitbach, Jeong Heo, Mong Cho, Anne Moon, Chang<br />
Won Kim, James Burke, Theresa Hickman, Kara Dubois, Yeon<br />
Sook Lee, Manijeh Daneshmand, John C. Bell, Richard Patt, Tae-Ho<br />
Hwang, David H. Kirn.<br />
20 4:00 PM<br />
Induction <strong>of</strong> Antiviral <strong>Gene</strong>s by the Tumor<br />
Microenvironment Confers Resistance to Virotherapy<br />
Yu-Ping Liu, Lukkana Suksanpaisan, Stephen J. Russell, Kah-Whye<br />
Peng.<br />
21 4:15 PM<br />
Superior Antitumoral Activity Induced by a Novel<br />
Oncolytic Adenovirus Armed with an EGFR-Targeted<br />
ImmunoRNase<br />
Ines Fernandez-Ulibarri, Michaela A. E. Arndt, Dominik E. Dorer,<br />
Sarah Engelhardt, Jürgen Krauss, Dirk M. Nettelbeck.<br />
22 4:30 PM<br />
The Oncolytic and Immunotherapeutic Vaccinia<br />
Virus Pexa-Vec (JX-594) Induces Antibody-Mediated<br />
Complement-Dependent Cancer <strong>Cell</strong> Cytolysis<br />
Mi Kyung Kim, Caroline H. Breitbach, Anne Moon, Jeong Heo, Yu<br />
Kyung Lee, Mong Cho, Jun Woo Lee, Seung Geun Kim, Dae Hwang<br />
Kang, Byung Ho Park, John C. Bell, David H. Kirn, Tae-Ho Hwang.<br />
23 4:45 PM<br />
Modulation <strong>of</strong> Innate Immunity with the Anti-CTLA4<br />
Antibody Ipilimumab (Ipi) in Measles Virotherapy for<br />
Glioblastoma<br />
Jayson J. Hardcastle, Hirosha Geekiyanage, Evidio Domingo<br />
Musibay, Cory Allen, Jin Fang, Aaron Johnson, Evanthia Galanis.<br />
24 5:00 PM<br />
Human Bone Marrow-Derived Mesenchymal<br />
Stromal <strong>Cell</strong>s Permit Delivery, and Enhance Eficacy<br />
<strong>of</strong> Systemically Administered Oncolytic Measles<br />
Virotherapy to Disseminated Human Pre-B ALL Murine<br />
Xenograft Models in the Presence <strong>of</strong> Humoral Immunity<br />
Anna Castleton, Aditi Dey, Bella Patel, Brendan Beaton, Anne<br />
Aucher, Daniel Davis, Adele Fielding.<br />
Oral Abstract Session 133<br />
Advancing the Science <strong>of</strong> <strong>Gene</strong> <strong>Therapy</strong> for<br />
Hematologic Diseases<br />
3:15 PM - 5:15 PM<br />
Room: Ballroom C<br />
25 3:15 PM<br />
Improved Chemotherapy Eficacy after MGMT(P140K)<br />
Hematopoietic Stem <strong>Cell</strong> <strong>Gene</strong> <strong>Therapy</strong> in Poor-<br />
Prognosis Glioblastoma by Patient-Speciic<br />
Mathematical Modeling<br />
Jennifer E. Adair, Brian C. Beard, Sandra K. Johnston, Maciej M.<br />
Mrugala, Russell C. Rockne, Kristin R. Swanson, Hans-Peter Kiem.<br />
26 3:30 PM<br />
Therapeutic Levels <strong>of</strong> Anti-Sickling Hemoglobin<br />
Production in Erythroid Progeny <strong>of</strong> Sickle <strong>Cell</strong> Disease<br />
CD34+ <strong>Cell</strong>s Following Lentivirus Vector-Mediated <strong>Gene</strong><br />
Delivery<br />
Fabrizia Urbinati, Phillip W. Hargrove, Sabine Geiger, Zulema<br />
Romero, Jennifer Wherley, Michael L. Kaufman, Roger P. Hollis,<br />
Christopher B. Chambers, Derek A. Persons, Donald B. Kohn,<br />
Andrew Wilber.<br />
27 3:45 PM<br />
Superior Stem <strong>Cell</strong> Mobilization by a Combination <strong>of</strong><br />
Plerixafor+G-CSF in Patients with Thalassemia<br />
Evangelia Yannaki, Achilles Anagnostopoulos, Garyfalia<br />
Karponi, Fani Zervou, Varnavas Constantinou, Nikoleta Psatha,<br />
Asimina Bouinta, Erica Jonlin, Thalia Papayannopoulou, George<br />
Stamatoyannopoulos.<br />
88<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013
Abstract Directory, Wednesday, May 15, 2013<br />
Oral Abstract Session 133, continued<br />
Advancing the Science <strong>of</strong> <strong>Gene</strong> <strong>Therapy</strong> for<br />
Hematologic Diseases<br />
3:15 PM - 5:15 PM<br />
Room: Ballroom C<br />
28 4:00 PM<br />
Tracking Fate and Plasticity <strong>of</strong> T <strong>Cell</strong>s in Humans by<br />
Retroviral Tagging<br />
Luca Biasco, Serena Scala, Cristina Baricordi, Nicoletta Cieri,<br />
Andrea Calabria, Eugenio Montini, Chiara Bonini, Alessandro Aiuti.<br />
29 4:15 PM<br />
Quantitative Tracking <strong>of</strong> the Output <strong>of</strong> Individual<br />
Hematopoietic Stem/Progenitor <strong>Cell</strong>s in a Non-Human<br />
Primate Model<br />
Chuanfeng Wu, Brian Li, Rong Lu, Alexander Jares, Robert<br />
Donahue, Irving Weissman, Cynthia Dunbar.<br />
30 4:30 PM<br />
Evaluation <strong>of</strong> Transduced CD34+ <strong>Cell</strong>s To Predict <strong>Gene</strong><br />
Marking Levels In Vivo in Rhesus <strong>Gene</strong> <strong>Therapy</strong> Model<br />
Naoya Uchida, Molly Evans, Aylin Bonifacino, Sandra Price, Allen<br />
Krouse, Mark Metzge, Matthew Hsieh, Robert Donahue, John<br />
Tisdale.<br />
31 4:45 PM<br />
Unimolecular Microchimerism Achieved through<br />
Hematopoietic Stem <strong>Cell</strong> Transplantation <strong>Gene</strong> <strong>Therapy</strong><br />
Facilitates Adjunctive <strong>Therapy</strong> with <strong>Gene</strong>tically-<br />
Modiied Mesenchymal Stem <strong>Cell</strong>s in Hemophilia A<br />
Allison M. Abuin, H. Trent Spencer, Christopher B. Doering.<br />
32 5:00 PM<br />
Rapamycin Treatment <strong>of</strong> Hematopoietic Stem <strong>Cell</strong>s<br />
Enhances Lentiviral Transduction through Autophagy<br />
Xueqian Wang**, Blythe D. Sather**, Iram Khan, Swati Singha,<br />
Gabrielle Curinga, David J. Rawlings, Bruce Torbett.<br />
Oral Abstract Session 134<br />
Neurological <strong>Therapy</strong>: Monogeneic Disorders<br />
3:15 PM - 5:15 PM<br />
Room: Ballroom D<br />
33 3:15 PM<br />
Intravenous AAV8-MTM1 Prolongs Life and<br />
Ameliorates Severe Muscle Pathology in Mouse and Dog<br />
Models <strong>of</strong> X-Linked Myotubular Myopathy<br />
Martin K. Childers, Romain Joubert, Melanie N. Holder, Robert<br />
W. Grange, Jon Doering, Michael W. Lawlor, Christelle Moal,<br />
Thibaud Jamet, Nathalie Daniele, Samia Martin, Christel Riviere,<br />
Kimberly Poppante, Thomas Soker, Caroline Hammer, Laetitia Van<br />
Wittenberghe, Melissa Goddard, Erin Mitchell, Jane Barber, Mark<br />
E. Furth, Alban Vignaud, Carole Masurier, Philippe Moullier, Alan<br />
H. Beggs, Alan H. Beggs, Anna Buj-Bello.<br />
34 3:30 PM<br />
Augmenting CNS Glucocerebrosidase Activity as a<br />
Therapeutic Strategy for Parkinsonism and Other<br />
Gaucher-Related Synucleinopathies<br />
Pablo S. Sardi, Lamya S. Shihabuddin, Richard L. Sidman, Seng H.<br />
Cheng.<br />
35 3:45 PM<br />
Eficacious and Sustained Canavan <strong>Gene</strong> <strong>Therapy</strong> by<br />
Single rAAV Injections Via IV at Postnatal Day 21 or ICV<br />
at a 100 Fold Lower Dose<br />
Seemin S. Ahmed, Reuben Matalon, Guangping Gao.<br />
36 4:00 PM<br />
Dramatic Phenotypic Improvement after Adeno-<br />
Associated Virus <strong>Gene</strong> <strong>Therapy</strong> in a Feline Model <strong>of</strong><br />
Sandh<strong>of</strong>f Disease<br />
Victoria J. McCurdy, Heather L. Gray-Edwards, Ashley N. Randle,<br />
Allison M. Bradbury, Aime K. Johnson, Patricia M. Beadlescomb,<br />
Nancy Morrison, Misako Hwang, Miguel Sena-Esteves, Douglas R.<br />
Martin.<br />
37 4:15 PM<br />
Preclinical Safety and Eficacy <strong>of</strong> Human CD34+ <strong>Cell</strong>s<br />
Transduced with Lentiviral Vector for the Treatment <strong>of</strong><br />
Adrenoleukodystrophy<br />
Lauryn Christiansen, Maria Denaro, John Pierciey, Anne Moser,<br />
Cynthia Bartholoma, Michael Rothe, Robert Kutner, Christopher<br />
Baum, Byoung Ryu, Manfred Schmidt, Garrett Heffner, Mitchell<br />
Finer, Gabor Veres.<br />
38 4:30 PM<br />
Preclinical Evaluation <strong>of</strong> Systemic Delivery <strong>of</strong> Blood-<br />
Brain Barrier-Targeted IDUA in Treating CNS Deicits <strong>of</strong><br />
Mice with Hurler Syndrome after Long Term Stem <strong>Cell</strong><br />
<strong>Gene</strong> <strong>Therapy</strong><br />
Salim S. El-Amouri, Mei Dai, Dao Pan.<br />
39 4:45 PM<br />
Broad Correction <strong>of</strong> Molecular Impairments by a<br />
Single Systemic rAAVrh74-hSGSH <strong>Gene</strong> Delivery in a<br />
Mouse Model <strong>of</strong> MPS IIIA: Links between the Brain and<br />
Peripheral Blood<br />
F. Jason Duncan, Bartholomew J. Naughton, Kimberly Zaraspe,<br />
Darren Murrey, Tierra Ware, Aaron Meadows, Haiyan Fu, Douglas<br />
McCarty.<br />
40 5:00 PM<br />
Amelioration <strong>of</strong> Neurologic Disease after AAV-<strong>Gene</strong><br />
<strong>Therapy</strong> in a Sheep Model <strong>of</strong> Tay-Sachs Disease<br />
H. L. Gray-Edwards, S. M. Hubbard, A. E. Randle, M. Hwang, A. M.<br />
Bradbury, V. J. McCurdy, D. U. Wilson, J. A. Hudson, B. K. Whitlock,<br />
J. L. Sartin, N. Salibi, R. J. Beyers, T. S. Denney, J. A. Johnson, K. P.<br />
Riddell, N. R. Cox, M. Sena-Estevez, D. R. Martin.<br />
Wednesday, May 15, 2013<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013<br />
89
Wednesday, May 15, 2013<br />
Abstract Directory, Wednesday, May 15, 2013<br />
Oral Abstract Session 135<br />
Chemical and Molecular Conjugates<br />
3:15 PM - 4:15 PM<br />
Room: 151 ABCG<br />
41 3:15 PM<br />
Optimized Polyplexes That Avoid Liver Uptake and<br />
Maintain Eficient <strong>Gene</strong> Transfer In Vivo<br />
Sanjib Khargharia, Koby Kizzire, Mark E. Ericson, Nick J.<br />
Baumhover, Kevin G. Rice.<br />
42 3:30 PM<br />
Synergistic Cancer <strong>Therapy</strong> by Simultaneous<br />
Expression and Silencing <strong>of</strong> Pro-Apoptotic and Pro-<br />
Survival <strong>Gene</strong>s Using Chimeric Nanoparticles<br />
Soo Kyung Cho, Jane Kim, Young Jik Kwon.<br />
43 3:45 PM<br />
Examining <strong>Cell</strong>-Penetrating Homochiral Cyclic Peptides<br />
as siRNA Non-Viral Transfection Agents<br />
Christiaan D. King, Ria L. Swanekamp, Bradley L. Nilsson, David A.<br />
Dean.<br />
44 4:00 PM<br />
Structure and Silencing Activity <strong>of</strong> Imidazole-<br />
Containing Peptide siRNA Polyplexes Are Dependent on<br />
Hydrogen Bonding<br />
Szu-Ting Chou, Kellie Hom, Lucas Tricoli, Jason Hustedt, Amy<br />
Lee, Joonil Seog, Jason Kahn, Qixin Leng, Daoning Zhang, Michael<br />
Shapiro, Archibald J. Mixson.<br />
Oral Abstract Session 136<br />
Infectious Diseases and Vaccines<br />
3:15 PM - 4:15 PM<br />
Room: 150 ABC<br />
45 3:15 PM<br />
Inducing Humoral and <strong>Cell</strong>ular Responses to Multiple<br />
Sporozoite and Liver-Stage Malaria Antigens Using<br />
pDNA<br />
Bernadette Ferraro, Kendra T. Talbot, Amritha Balakrishnan,<br />
Matthew P Morrow, Natalie A. Hutnick, Devin J. Myles, Devon J.<br />
Shedlock, Nyamekye Obeng-Adjei, Jian Yan, Rachel Shiver, Amir<br />
S. Khan, Maria Yang, Ami S. Brown, Ulrike Wille-Reece, Ashley<br />
Birkett, Niranjan Y. Sardesai, David B. Weiner.<br />
46 3:30 PM<br />
T-<strong>Cell</strong>s Edited To Express the C34 Peptide from gp41<br />
Heptad Repeat-2 Fused to CCR5 or CXCR4 Exhibit<br />
Robust Protection from Diverse HIV-1 Isolates<br />
Jianbin Wang, George J. Leslie, Jennifer Duong, Kevin L. Hua, Jenny<br />
J. Yan, Beth Haggarty, Andrea P. Jordon, Josephine Romano, Lei<br />
Zhang, Edward J. Rebar, Philip D. Gregory, James A. Hoxie, Michael<br />
C. Holmes.<br />
47 3:45 PM<br />
Integrase-Defective Lentiviral Vector-Induced Dendritic<br />
<strong>Cell</strong>s: Clinical Development and Validation <strong>of</strong> CD4+/<br />
CD8+ T <strong>Cell</strong> Responses for Combination with Adoptive<br />
CMV-Speciic T-<strong>Cell</strong> <strong>Therapy</strong><br />
Anusara Daenthanasanmak, Constanca Figueiredo, Eliana<br />
Ruggiero, Manfred Schmidt, Arnold Ganser, Renata Stripecke.<br />
48 4:00 PM<br />
Engineering Resistance to HIV-1 Infection with a Dual<br />
Therapeutic Lentiviral Vector<br />
Bryan P. Burke, Maureen P. Boyd, Michelle L. Millington, Helen<br />
Impey, Jane Zhang, Maria V. Carroll, Joanna Camba-Colon, Naomi<br />
Keech, Frederic Delebecque, Orit Wolstein, Annett Howe, Rachel<br />
Koldej, Tamara Nicolson, Bernard R. Levin, Valerie Rezek,<br />
Dimitrios N. Vatakis, Scott G. Kitchen, Louis R. Breton, Jeffrey S.<br />
Bartlett, Ge<strong>of</strong>f P. Symonds.<br />
Oral Abstract Session 137<br />
<strong>Cell</strong> Processing and Vector Production<br />
4:20 PM - 5:20 PM<br />
Room: 151 ABCG<br />
49 4:20 PM<br />
gc Cytokines IL7 and IL15 Expanded Chimeric Antigen<br />
Receptor-Redirected T <strong>Cell</strong>s (CAR-T) with Superior<br />
Antitumor Activity In Vivo<br />
Yang Xu, Ming Zhang, Aruna Mahendravada, Barbara Savoldo,<br />
Gianpietro Dotti.<br />
50 4:35 PM<br />
Large-Scale Clinical-Grade Retroviral Vector Production<br />
in iCELLis Nano Bioreactor<br />
Xiuyan Wang, Malgorzata Olzzewska, Jinrong Qu, Teresa<br />
Wasielewska, Shirley Bartido, Gregory Hermetet, Michel Sadelain,<br />
Isabelle Riviere.<br />
51 4:50 PM<br />
Large Scale Production <strong>of</strong> Lentiviral Vectors Using<br />
Serum Free Suspension <strong>Cell</strong> Culture System<br />
Amelia H. Thomas, Kevin Brown, Francis H. Pierciey, Jr, Gabor<br />
Veres, Byoung Y. Ryu.<br />
52 5:05 PM<br />
A Single Baculovirus for the Production <strong>of</strong> rAAV8 Viral<br />
Vectors<br />
Lionel Galibert, Aurélien Jacob, Bérangère Langlet-Bertin,<br />
Marjorie Boutin Fontaine, Delphine Bonnin, Christophe Lecomte,<br />
Christel Rivière, Otto W. Merten.<br />
90<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013
Abstract Directory, Wednesday, May 15, 2013<br />
Oral Abstract Session 138<br />
Sensory (Ophthalmic and Auditory) <strong>Gene</strong> &<br />
<strong>Cell</strong> <strong>Therapy</strong><br />
4:20 PM - 5:20 PM<br />
Room: 150 ABC<br />
53 4:20 PM<br />
Retinal <strong>Gene</strong> <strong>Therapy</strong> May Alter Connectivity <strong>of</strong> Visual<br />
Pathways<br />
M. Ashtari, L. Cyckowski, G. Zhang, P. Cook, K. Marshall, J. Wellman,<br />
J. Gee, A. Vossough, K. Shindler, A. Maguire, J. Bennett.<br />
Wednesday, May 15, 2013<br />
54 4:35 PM<br />
<strong>Gene</strong> <strong>Therapy</strong> for Wet-AMD: Progress Report on a<br />
Phase I/II Clinical Trial<br />
Elizabeth P. Rakoczy, May C. Lai, Cora Pierce, Aaron L. Magno,<br />
Richard J. Samulski, David J. Dismuke, Joshua C. Grieger, Thomas<br />
W. Chalberg, Mark S. Blumenkrnz, Martyn French, Ian J. Constable.<br />
55 4:50 PM<br />
Preclinical Safety Studies for AAV2-MerTK <strong>Gene</strong><br />
<strong>Therapy</strong> Vector for Retinitis Pigmentosa<br />
Thomas J. Conlon, Wen-Tao Deng, Renee C. Ryals, Kirsten E. Erger,<br />
Travis L. Cossette, Shannon E. Boye, Isaam McDoom, Nathalie<br />
Clemente, Brian Cleaver, Mark Potter, Corinne Abernathy, Sanford<br />
L. Boye, William H. Hauswirth.<br />
56 5:05 PM<br />
Glaucoma-GT, a Novel <strong>Gene</strong> <strong>Therapy</strong> Treatment for<br />
Primary Open-Angle Glaucoma<br />
Katie Binley, Scott Ellis, Vicky Scripps, Sharifah Iqball, Stuart<br />
Naylor, Kyriacos Mitrophanous.<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013<br />
91
Thursday, May 16, 2013<br />
Abstract Directory, Thursday, May 16, 2013<br />
Oral Abstract Session 230<br />
Clinical <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> Oral Abstract<br />
Session<br />
2:00 PM - 4:00 PM<br />
Room: Ballroom A<br />
57 2:00 PM<br />
Acid alpha-Glucosidase <strong>Gene</strong> Replacement <strong>Therapy</strong><br />
to the Diaphragm in Ventilator-Dependent Pompe<br />
Disease: One-Year Respiratory Motor Outcomes<br />
Barbara K. Smith, Daniel Martin, Lee Ann Lawson, Cathryn S. Mah,<br />
Saleem Islam, Thomas Conlon J. Conlon, Dawn Phillips, Shelley<br />
Collins, Barry J. Byrne.<br />
58 2:15 PM<br />
Long Term CD4 Reconstitution in HIV Subjects<br />
Receiving ZFN CCR5 Modiied CD4 T-<strong>Cell</strong>s (SB-728-T)<br />
May Be Attributed to the Sustained Durability <strong>of</strong> the<br />
Central Memory T-<strong>Cell</strong> Subset<br />
Gary K. Lee, Joumana Zeidan, Jay Lalezari, Ronald Mitsuyasu,<br />
Shelley Wang, Marty Giedlin, Ge<strong>of</strong>f Nichol, Winson Tang, Dale<br />
Ando, Raick-Pierre Sekaly.<br />
59 2:30 PM<br />
Cardiovascular Toxicity and Titin Cross-Reactivity <strong>of</strong><br />
Afinity-Enhanced TCR-Engineered T <strong>Cell</strong>s Against<br />
HLA-A1 Restricted MAGE-A3 Antigen<br />
Marcela V. Maus, Gerald P. Linette, Edward A. Stadtmauer, Aaron<br />
P. Rapoport, Bruce L. Levine, Lyndsey A. Emery, Leslie Litzky,<br />
Gwendolyn K. Binder-Scholl, Dominic P. Smethurst, Andrew B.<br />
Gerry, Nick J. Pumphrey, Alan Bennet, Joanna Brewer, Jane Harper,<br />
Namir Hassan, Bent K. Jakobsen, Michael Kalos, Carl H. June.<br />
60 2:45 PM<br />
<strong>Gene</strong> <strong>Therapy</strong> for Aromatic L-Amino Acid<br />
Decarboxylase Deiciency: Report <strong>of</strong> Treatment for 8<br />
Patients<br />
Wuh-Liang Hwu, Shin-ichi Muramatsu, Sheng-Hong Tseng, Kai-<br />
Yuan Tzen, Ni-Chung Lee, Yin-Hsiu Chien, Richard O. Snyder, Barry<br />
Byrne, Chun-Hwei Tai, Ruey-Meei Wu.<br />
61 3:00 PM<br />
Safe G-CSF Mobilization <strong>of</strong> Thalassemia Patient CD34+<br />
<strong>Cell</strong>s and Effective Globin <strong>Gene</strong> Transduction for<br />
Therapeutic Infusion<br />
Farid Boulad, Xiuyan Wang, Clare Taylor, Jinrong Qu, Leda Ferro,<br />
Garie Karponi, Shirley Bartido, Patricia Giardina, Glenn Heller,<br />
Susan E. Prockop, Aurelio Maggio, Michel Sadelain, Isabelle<br />
Riviere.<br />
62 3:15 PM<br />
Hematopoietic Stem <strong>Cell</strong> <strong>Gene</strong> <strong>Therapy</strong> with<br />
Lentiviral Vector in 4 Patients with Cerebral X-Linked<br />
Adrenoleukodystrophy: Long-Term Outcome and<br />
Comparison <strong>of</strong> Eficacy with Allogeneic Hematopoietic<br />
Stem <strong>Cell</strong> Transplantation<br />
Paul Orchard, Nathalie Cartier, Salima Hacein-Bey-Abina,<br />
Joanne Kurtzberg, Maria Escolar, Jakub Tolar, André Baruchel,<br />
Jean-Hughes Dalle, Gérard Michel, Stéphane Blanche, Cynthia<br />
Bartholomä, Manfred Schmidt, Pierre Bougnères, Christ<strong>of</strong> van<br />
Kalle, Marina Cavazzana-Calvo, Alain Fischer, Patrick Aubourg.<br />
676 3:30 PM<br />
Update on Toca 511 - a retroviral replicating vector in<br />
clinical trials for High Grade Glioma<br />
Douglas J Jolly, Joan M. Robbins, Derek Ostertag, Amy Lin, Carlos<br />
Ibanez, Harry E. Gruber, Noriyuki Kasahara, Timothy F. Cloughesy,<br />
Manish K. Aghi, Susan M. Chang, Michael A. Vogelbaum, Santosh<br />
Kesari, Tom Mikkelsen, Joseph Landoli, E. Antonio Chiocca, James<br />
Elder, Greg Foltz, Dan Pertschuk<br />
299 3:45 PM<br />
Clinical Responses in Patients Infused with<br />
T Lymphocytes Redirected To Target -Light<br />
Immunoglobulin Chain<br />
Carlos A. Ramos, Barbara Savoldo, Enli Liu, Adrian P. Gee, Zhuyong<br />
Mei, Bambi Grilley, Cliona M. Rooney, Helen E. Heslop, Malcolm K.<br />
Brenner, Gianpietro Dotti.<br />
Oral Abstract Session 231<br />
RNA Virus Vectors<br />
2:00 PM - 4:00 PM<br />
Room: Ballroom B<br />
63 2:00 PM<br />
Comprehensive Study <strong>of</strong> Lentiviral “Integrome” in WAS<br />
<strong>Gene</strong> <strong>Therapy</strong> Patients<br />
Luca Biasco, Cristina Baricordi, Francesca Dionisio, Andrea<br />
Calabria, Samantha Scaramuzza, Danilo Pellin, Clelia Di Serio,<br />
Christ<strong>of</strong> Von Kalle, Manfred Schmidt, Eugenio Montini, Luigi<br />
Naldini, Alessandro Aiuti.<br />
64 2:15 PM<br />
In Vivo Safety Evaluation <strong>of</strong> a Novel Insulated SIN LV<br />
Reveals Genotoxicity Escape-Mechanisms<br />
Monica Volpin, Daniela Cesana, Alexandre Artus, Caroline Duros,<br />
Fabrizio Benedicenti, Lucia Sergi Sergi, Luigi Naldini, Odile Cohen-<br />
Haguenauer, Eugenio Montini.<br />
65 2:30 PM<br />
No Impact <strong>of</strong> Lentiviral Transduction on Hematopoietic<br />
Stem and Progenitor <strong>Cell</strong> Telomere Length or <strong>Gene</strong><br />
Expression Proile in the Rhesus Macaque<br />
Stephanie E. Sellers, Bogden Dumitriu, Mary Morgan, Colin O. Wu,<br />
Nalini Raghavarchari, Yanqin Yang, Naoya Uchida, John F. Tisdale,<br />
Robert E. Donahue, Andre Larochelle, Cynthia E. Dunbar.<br />
92<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013
Abstract Directory, Thursday, May 16, 2013<br />
Oral Abstract Session 231, continued<br />
RNA Virus Vectors<br />
2:00 PM - 4:00 PM<br />
Room: Ballroom B<br />
66 2:45 PM<br />
Rescue <strong>of</strong> the X-CGD Phenotype by an Alpharetroviral<br />
Vector without Concomitant Induction <strong>of</strong> Aberrant<br />
Splicing<br />
Kerstin B. Kaufmann, Christian Brendel, Julia D. Suerth, Uta<br />
Müller-Kuller, Linping Chen-Wichmann, Joachim Schwäble,<br />
Shweta Pahujani, Hana Kunkel, Axel Schambach, Christopher<br />
Baum, Manuel Grez.<br />
67 3:00 PM<br />
<strong>Gene</strong> Transfer to Motor Neurons with Novel Targeted<br />
Lentiviral Vectors<br />
Ioanna Eleftheriadou, Antonio Trabalza, Stuart M. Ellison, Nicholas<br />
D. Mazarakis.<br />
68 3:15 PM<br />
NXF1 Controls Nuclear Export <strong>of</strong> Spliced and Unspliced<br />
Gammaretroviral Transcripts<br />
Toshie Sakuma, Yasuhiro Ikeda.<br />
69 3:30 PM<br />
Dual microRNA-Regulated Oncolytic Coxsackievirus B3<br />
Infection Displays Antitumor Activity with Attenuated<br />
Pathogenicity in Mice<br />
Shohei Miyamoto, Hiroyuki Inoue, Miyako Sagara, Beibei Wang,<br />
Koichi Takayama, Hiroyuki Shimizu, Yoichi Nakanishi, Kenzaburo<br />
Tani.<br />
70 3:45 PM<br />
Retroviral Replicating Vector (RRV)-Mediated Prodrug-<br />
Activator <strong>Gene</strong> <strong>Therapy</strong> Transcriptionally Targeted to<br />
Prostate Cancer<br />
Shuichi Kamijima, Takahiro Kimura, Kazunori Haga, Kei Hiraoka,<br />
Akihito Inagaki, Masamichi Takahashi, Christopher R. Logg,<br />
Chinghai Kao, Bernard H. Bochner, Noriyuki Kasahara.<br />
Oral Abstract Session 232<br />
AAV Vector Biology<br />
2:00 PM - 4:00 PM<br />
Room: Ballroom C<br />
71 2:00 PM<br />
The Structure <strong>of</strong> AAVrh32.33, a Novel <strong>Gene</strong> Delivery<br />
Vector<br />
Kyle Mikals, Hyun-Joo Nam, Kim Van Vliet, Luk H. Vandenberghe,<br />
Lauren E. Mays, Robert McKenna, James M. Wilson, Mavis<br />
Agbandje-McKenna.<br />
72 2:15 PM<br />
Identiication <strong>of</strong> Nucleolar Localization Sequences in<br />
the C-Terminal Region <strong>of</strong> Assembly-Activating Protein<br />
Lauriel F. Earley, Xiao-Xin Sun, Kei Adachi, Mushui Dai, Hiroyuki<br />
Nakai.<br />
73 2:30 PM<br />
Integrins Play a Pivotal Role in Transvascular<br />
Transport and Tissue Uptake <strong>of</strong> AAV9 In Vivo<br />
Shen Shen, Aravind Asokan.<br />
74 2:45 PM<br />
Adeno-Associated Virus Utilizes Host <strong>Cell</strong> Nuclear<br />
Import Machinery to Enter the Nucleus<br />
Sarah C. Nicolson, Richard J. Samulski.<br />
75 3:00 PM<br />
Functional Roles <strong>of</strong> the AAV2 Inverted Terminal Repeat<br />
in Messenger RNA Transport and Transgene Expression<br />
Yuan Wang, Yuan Lu, Lina Wang, Joon-Hyung Kim, George V.<br />
Aslanidi, Changquan Ling, Arun Srivastava, Chen Ling.<br />
76 3:15 PM<br />
Epigenetic Alterations in the Dystrophic Muscle<br />
Environment Down-Regulate rAAV-Mediated Transgene<br />
Expression<br />
Jean Baptiste Dupont, Benoit Tournaire, Christophe Georger,<br />
Laetitia Van Wittenberghe, Beatrice Marolleau, Laurence Dubreil,<br />
Emilie Lecomte, Fulvio Mavilio, Richard O. Snyder, Philippe<br />
Moullier, Adrien Leger.<br />
77 3:30 PM<br />
Sialic Acids and Erythrocyte Interactions Are Critical<br />
Determinants <strong>of</strong> Cardiopulmonary Tropism Displayed<br />
by AAV Serotype 4<br />
Shen Shen, Andrew Troupes, Nagesh Pulicherla, Aravind Asokan.<br />
78 3:45 PM<br />
Simultaneous Pharmacokinetic Proiling <strong>of</strong> Multiple<br />
AAV Serotypes and Mutants in a Non-Human Primate<br />
by AAV Barcode-Seq<br />
Kei Adachi, Yasuhiro Kawano, Gregory A. Dissen, Sergio R. Ojeda,<br />
Hiroyuki Nakai.<br />
Oral Abstract Session 233<br />
Stem <strong>Cell</strong> Engineering and <strong>Therapy</strong><br />
2:00 PM - 4:00 PM<br />
Room: Ballroom D<br />
79 Abstract Withdrawn from Presentation<br />
470 2:00 PM (Moved from Poster Session II)<br />
Human Parthenogenetic Stem <strong>Cell</strong>-Derived Hepatocytes<br />
for Liver Repopulation: <strong>Cell</strong> <strong>Therapy</strong> Demonstrated in a<br />
Pilot Study with Gunn Rats<br />
Larisa Agapova, Alina Ostrowska, Trudy Christiansen-Weber,<br />
Tiffany Chu, Ruslan Semechkin.<br />
80 2:15 PM<br />
Transplanted Mesenchymal Stem <strong>Cell</strong>s Provide Type<br />
VII Collagen in the Skin <strong>of</strong> Recessive Dystrophic<br />
Epidermolysis Bullosa<br />
Katsuto Tamai, Shin Iinuma, Eriko Ikawa, Yasuhi Kikuchi, Ryo<br />
Fujita, Masaru Ishii, John McGrath, Jouni Uitto, Yasuhumi Kaneda.<br />
Thursday, May 16, 2013<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013<br />
93
Abstract Directory, Thursday, May 16, 2013<br />
Oral Abstract Session 233, continued<br />
Stem <strong>Cell</strong> Engineering and <strong>Therapy</strong><br />
2:00 PM - 4:00 PM<br />
Room: Ballroom D<br />
Oral Abstract Session 234<br />
Musculo-skeletal <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> I<br />
2:00 PM - 4:00 PM<br />
Room: 150 DEFG<br />
Thursday, May 16, 2013<br />
81 2:30 PM<br />
“Semi-Universal Donor Stem <strong>Cell</strong>s” That Express a<br />
Single Class I Human Leukocyte Antigen (HLA) A*0201<br />
Can Present Peptides to Allorestricted CD8+ T-<strong>Cell</strong>s<br />
German Gornalusse, Marvin Festag, Roli Hirata, Laura Riolobos,<br />
Cameron Turtle, Stan Riddell, David W. Russell.<br />
82 2:45 PM<br />
Targeted Correction <strong>of</strong> X-CGD iPSCs by TALEN-Mediated<br />
<strong>Gene</strong> Transfer to the CYBB Locus<br />
Colin L. Sweeney, Uimook Choi, Jizhong Zou, Suk See DeRavin,<br />
Harry L. Malech.<br />
83 3:00 PM<br />
<strong>Gene</strong>tically Corrected Myoblasts Derived from<br />
Dystrophic hIPSCs Fused with Existing Muscle Fibers<br />
in Immunodeicient mdx Mice and Led to Expression <strong>of</strong><br />
Micro-Dystrophin and a-Sarcoglycan<br />
Chantale Maltais, Carl Lebel, Joël Rousseau, Jacques P. Tremblay.<br />
84 3:15 PM<br />
<strong>Gene</strong>ration <strong>of</strong> Functional Monocyte/Macrophages<br />
by <strong>Gene</strong>tic Correction <strong>of</strong> Patient-Speciic iPSC in<br />
Congenital Pulmonary Alveolar Proteinosis<br />
Nico Lachmann, Christine Happle, Doreen Lüttge, Mania<br />
Ackermann, Adele Mucci, Nicolaus Schwerk, Martin Wetzke, Sylvia<br />
Merkert, Axel Schambach, Gesine Hansen, Thomas Moritz.<br />
85 3:30 PM<br />
Clonogenic Hepatocyte Progenitors from the Liver,<br />
Pancreas and Gall Bladder<br />
Craig Dorrell, Meritxell Huch, Yuhan Wang, Hans Clevers, Markus<br />
Grompe.<br />
86 3:45 PM<br />
The Activation <strong>of</strong> the Wnt3a/b-Catenin Pathway<br />
Induced Maturation <strong>of</strong> Impaired Neutrophils as Well<br />
as the Endoplasmic Reticulum Stress in Induced<br />
Pluripotent Stem <strong>Cell</strong> Derived from a Severe Congenital<br />
Neutropenia Patient with ELANE Mutation<br />
Takafumi Hiramoto, Yasuhiro Ebihara, Yoko Mizoguchi, Kazuhiro<br />
Nakamura, Kiyoshi Yamaguchi, Kazuko Ueno, Naoki Nariai, Shinji<br />
Mochizuki, Shohei Yamamoto, Masao Nagasaki, Yoichi Furukawa,<br />
Kenzaburo Tani, Hiromitsu Nakauchi, Masao Kobayashi, Kohichiro<br />
Tsuji.<br />
87 2:00 PM<br />
The Effect <strong>of</strong> Neonatal <strong>Gene</strong> <strong>Therapy</strong> on Dysostosis<br />
Multiplex in Mucopolysaccaridosis VII Dogs after a<br />
Decade<br />
Katherine P. Ponder, Elizabeth M. Xing, Van W. Knox, Patricia A.<br />
O’Donnell, Yuli Liu, Susan Wu, Mark E. Haskins.<br />
88 2:15 PM<br />
scAAV Provides Sustained Expression <strong>of</strong> a Homologous<br />
Therapeutic Transgene in Large Mammalian Joints,<br />
with Enhanced Expression in Osteoarthritis<br />
Rachael S. Watson, Ted Broome, Andrew Smith, Patrick Colahan,<br />
Steven Ghivizzani.<br />
89 2:30 PM<br />
Dual Axis <strong>Gene</strong>-<strong>Therapy</strong> Using Stem <strong>Cell</strong>s<br />
Overexpressing TGF-b3 in Combination with IL-1b and<br />
TNF-a RNA Silencing for Osteoarthritis Control in a<br />
Large Animal Osteochondral Chip Fracture Model<br />
Alan J. Nixon, Ashlee E. Watts, Laila Begum.<br />
90 2:45 PM<br />
Respiratory Function in a Canine Model <strong>of</strong> X-Linked<br />
Myotubular Myopathy Following Regional Limb<br />
Infusion with Recombinant AAV8-MTM1<br />
Melissa Goddard, Romain Joubert, Karine Poulard, Robert Grange,<br />
Michael Lawlor, Christelle Moal, Thibaud Jamet, Nathalie Danièle,<br />
Samia Martin, Christel Rivière, Caroline Hammer, Laetitia Van<br />
Wittenberghe, Erin Mitchell, Alban Vignaud, Carole Masurier,<br />
Philippe Moullier, Alan Beggs, Anna Buj-Bello, Barbara Smith,<br />
Barry Byrne, Martin Childers.<br />
91 3:00 PM<br />
Vascular Delivery <strong>of</strong> AAV.Micro-Dystrophin Using<br />
Whole Limb Re-Circulation in the Non-Human Primate<br />
as a <strong>Therapy</strong> for DMD<br />
Louis G. Chicoine, Chrystal L. Montgomery, Danielle A. Grifin,<br />
Kristin N. Heller, Kim Shontz, Thomas J. Preston, William G.<br />
Bremer, Christopher M. Walker, K. R. Clark, Jerry R. Mendell,<br />
Louise R. Rodino-Klapac.<br />
92 3:15 PM<br />
Safety and Histological Signs <strong>of</strong> Eficacy Following<br />
Systemic AAV9-CMV-Microutrophin <strong>Gene</strong> <strong>Therapy</strong> in a<br />
Canine Model for Duchenne Muscular Dystrophy<br />
Alock Malik, Andrew Mead, Mike Petrov, Marilyn Mitchell, Leonard<br />
Su, Frederick Balzer, Jacqueline Farag, Janet Bogan, Dan Bogan, Joe<br />
Kornegay, Hansell Stedman.<br />
93 3:30 PM<br />
A Xenograft Model <strong>of</strong> Skeletal Muscle for Preclinical<br />
<strong>Gene</strong> <strong>Therapy</strong> Studies<br />
Kathryn R. Wagner, Tracy Y. Zhang, Terence A. Partridge, Takako<br />
Jone, Oliver King, Fedik Rahimov, Qin Su, Guangpin Gao, Louis M.<br />
Kunkel, Robert H. Brown, Jr, Miguel Sena-Esteves.<br />
94<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013
Abstract Directory, Thursday, May 16, 2013<br />
Oral Abstract Session 234, continued<br />
Musculo-skeletal <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> I<br />
2:00 PM - 4:00 PM<br />
Room: 150 DEFG<br />
94 3:45 PM<br />
Kidney Toxicity Caused by NEMO-Binding Domain<br />
(NBD) Peptide <strong>Therapy</strong> in a Murine Model <strong>of</strong> Muscular<br />
Dystrophy<br />
Paula R. Clemens, Daniel P. Reay, Katy Wack, Donna B. Stolz, Paul<br />
D. Robbins.<br />
Oral Abstract Session 235<br />
Cardiovascular <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong><br />
2:00 PM - 3:00 PM<br />
Room: 150 ABC<br />
95 2:00 PM<br />
FunSel: <strong>Gene</strong>ration <strong>of</strong> AAV-Based, Arrayed Libraries for<br />
In Vivo Functional Selection <strong>of</strong> Secreted Factors and<br />
miRNAs Against Degenerative Disorders<br />
Giulia Ruozi, Francesca Bortolotti, Luca Braga, Antonella Falcione,<br />
Matteo Dal Ferro, Serena Zacchigna, Lorena Zentilin, Mauro<br />
Giacca.<br />
96 2:15 PM<br />
Intra-Infarct Delivery <strong>of</strong> Microparticles Containing<br />
MAP4K4 siRNA Reduce Acute Post-Infarct<br />
Inlammatory Responses<br />
Matthew S. Weaver, Jun Luo, James E. Dennis, Myriam Aouadi,<br />
Timothy P. Fitzgibbons, Shinya U. Amano, Michael P. Czech,<br />
Margaret D. Allen.<br />
97 2:30 PM<br />
<strong>Gene</strong> Transfer Using a Snake Venom Derived<br />
Vascular Endothelial Growth Factor Induces Robust<br />
Angiogenesis and Improves Ventricular Function in<br />
Ischemic Porcine Myocardium<br />
Paavo J. Halonen, Jussi Nurro, Antti Kuivanen, Tuomas T. Rissanen,<br />
Seppo Ylä-Herttuala.<br />
98 2:45 PM<br />
Long-Term Robust Myocardial Transduction <strong>of</strong> the Dog<br />
Heart from Peripheral Vein by Adeno-Associated Virus<br />
Serotype-8 (AAV-8)<br />
Xiufang Pan, Yongping Yue, Keqing Zhang, William Lostal, Jin-Hong<br />
Shin, Dongsheng Duan.<br />
Oral Abstract Session 236<br />
Physical Methods <strong>of</strong> Delivery<br />
2:00 PM - 3:15 PM<br />
Room: 151 ABCG<br />
99 2:00 PM<br />
Effective <strong>Gene</strong> Electrotransfer Is Dependent on the<br />
Pulse Parameters <strong>of</strong> the Applied Electric Field<br />
Richard Heller, Cathryn Lundberg, Fanying Li, Niculina Burcus,<br />
Shawna Shirley.<br />
100 2:15 PM<br />
Targeted Modulation <strong>of</strong> BACE1 Expression and<br />
Electrical Activity in Neuronal Cultures Using Voltage<br />
Pre-Conditioning<br />
Arati M. Sridharan, Jit Muthuswamy.<br />
101 2:30 PM<br />
Ultrasound and Microbubbles Mediated Factor VIII<br />
Plasmid <strong>Gene</strong> <strong>Therapy</strong> in Hemophilia A Mice<br />
Shuxian Song, Misty L. Noble, Ryan Sun, Luping Fan, Carol H. Miao.<br />
102 2:45 PM<br />
Gadolinium Chloride Extends the Duration <strong>of</strong> <strong>Gene</strong><br />
Expression in Dogs Hydrodynamically Infused with<br />
Sleeping Beauty Transposons<br />
R. Scott McIvor, Myra C. Rusten, Erik R. Olson, Jason B. Bell, Elena<br />
L. Aronovich, Kendra A. Hyland, John R. Lake, Roland Gunther,<br />
Bryan M. Ladd, David W. Hunter, Perry B. Hackett.<br />
103 3:00 PM<br />
Non-Invasive In Vivo Imaging, Biodistribution and<br />
Clearance Analyses <strong>of</strong> Intravenously Administered<br />
Extracellular Vesicles<br />
Charles P. Lai, Osama Mardini, Casey A. Maguire, Bakhos T.<br />
Tannous, Xandra O. Breakeield.<br />
Oral Abstract Session 237<br />
Evolution <strong>of</strong> Pulmonary Treatments<br />
3:05 PM - 4:05 PM<br />
Room: 150 ABC<br />
104 3:05 PM<br />
Maternal Immunization with Chimpanzee Adenovirus<br />
Expressing RSV Fusion Protein Protects Against<br />
Neonatal RSV Pulmonary Infection<br />
Anurag Sharma, Rebecca Wendland, Biin Sung, Wendy Wu, Stefan<br />
Worgall.<br />
Thursday, May 16, 2013<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013<br />
95
Abstract Directory, Thursday, May 16, 2013<br />
Oral Abstract Session 237, continued<br />
Evolution <strong>of</strong> Pulmonary Treatments<br />
3:05 PM - 4:05 PM<br />
Room: 150 ABC<br />
Poster Session I<br />
4:00 PM - 6:00 PM<br />
Room: Exhibit Hall C/D<br />
AAV Vectors I<br />
Thursday, May 16, 2013<br />
105 Abstract Withdrawn from Presentation<br />
432 3:20 PM Moved from Poster Session II<br />
A Novel Cancer <strong>Cell</strong> Vaccine Using Induced Pluripotent<br />
Stem <strong>Cell</strong>s <strong>Gene</strong>tically Engineered To Produce GM-CSF<br />
Elicits Substantial Antitumor Immunity in a Syngeneic<br />
Mouse Model<br />
Hiroyuki Inoue, Ayumi Wanatabe, Chika Sakamoto, Megumi<br />
Narusawa, Shohei Miyamoto, Makoto Inoue, Koichi Takayama,<br />
Mamoru Hasegawa, Yoichi Nakanishi, Kenzaburo Tani.<br />
106 3:35 PM<br />
Safety, Toxicology and Eficacy <strong>of</strong> a Lung-Directed AAV-<br />
Based <strong>Gene</strong> <strong>Therapy</strong> for a1-Antitrypsin Deiciency<br />
Maria J. Chiuchiolo, Neil R. Hackett, Esther Z. Frenk, Yihharn<br />
Hwang, Jianping Qiu, Janet Benson, Gensheng Wang, Stephen M.<br />
Kaminsky, Ronald G. Crystal.<br />
107 3:50 PM<br />
Allele-Preferred Targeted Correction <strong>of</strong> CFTR <strong>Gene</strong> in<br />
Cystic Fibrosis Induced Pluripotent Stem <strong>Cell</strong>s<br />
Jacquelin H. Bui, Ana M. Crane, Xuan S. Li, Philipp Kramer, Manuel<br />
Gonzalez-Garay, Wei Liao, Jianbin Wang, Helena C. Sun, David E.<br />
Paschon, Dmitry Y. Guschin, Philip D. Gregory, Darrell N. Kotton,<br />
Michael C. Holmes, Brian R. Davis.<br />
Oral Abstract Session 238<br />
Immunologic & Host Responses in <strong>Gene</strong> & <strong>Cell</strong><br />
<strong>Therapy</strong><br />
3:20 PM - 4:05 PM<br />
Room: 151 ABCG<br />
108 3:20 PM<br />
Escaping Immune Activation through the Use <strong>of</strong> CpG-<br />
Depleted AAV Vectors<br />
Susan Faust, Peter Bell, Benjamin Cutler, Scott Ashley, Joseph<br />
Rabinowitz, James Wilson.<br />
109 3:35 PM<br />
Ex Vivo Expanded Treg Suppress Inhibitor Formation in<br />
Protein and <strong>Gene</strong> Therapies for Hemophilia<br />
Debalina Sarkar, David M. Markusic, Todd M. Brusko, Roland W.<br />
Herzog.<br />
110 3:50 PM<br />
Overcoming Pre-Existing Humoral Immunity to AAV<br />
Using Capsid Decoys<br />
Federico Mingozzi, Xavier M. Anguela, Giulia Pavani, Yifeng Chen,<br />
Robert H. Davidson, Daniel H. Hui, Mustafa Yazicioglu, Liron<br />
Elkouby, Christian J. Hinderer, Armida Faella, Carolann Howard,<br />
Alex Tai, Gregory M. Podsak<strong>of</strong>f, Margaret C. Ragni, Shangzhen<br />
Zhou, Etiena Basner-Tschakarjan, Fraser J. Wright, Katherine A.<br />
High.<br />
111<br />
Locoregional Intravenous Delivery <strong>of</strong> AAV Vector in<br />
Macaque Results in Persistent Transgene Expression<br />
Despite a Dedicated Humoral and <strong>Cell</strong>ular Immune<br />
Responses<br />
Gwladys Gernoux, Marie Devaux, Laurence Dubreil, Nicolas Jaulin,<br />
Mickaël Guilbaud, Jack-Yves Deschamps, Thibaut Larcher, Guigand<br />
Lydie, Maeva Dutilleul, Caroline Le Guiner, Yan Chérel, Philippe<br />
Moullier, Oumeya Adjali.<br />
112<br />
Global CNS <strong>Gene</strong> Delivery in Non-Human Primates<br />
by Intra-CSF Administration: Evasion <strong>of</strong> Anti-AAV<br />
Neutralizing Antibodies but Not a Transgene-Speciic<br />
CTL Response<br />
Steven J. Gray, Sahana Nagabhushan Kalburgi, Thomas J. McCown,<br />
R. Jude Samulski.<br />
113<br />
Increased Occurrence <strong>of</strong> Hepatocellular Carcinoma<br />
(HCC) in Mice after Neonatal Adeno-Associated Virus<br />
(AAV8) <strong>Gene</strong> Delivery Exhibits Dose-Dependent<br />
Tumorigenesis<br />
Randy J. Chandler, Charles P. Venditti.<br />
114<br />
A Novel Adeno-Associated Virus Serotype (AAV)-8<br />
Capsid Mutant Improves Human Coagulation Factor IX<br />
Expression In Vivo<br />
Dwaipayan Sen, Rupali A. Gadkari, Govindarajan Sudha, Nishanth<br />
Gabriel, V. Ramya, Sukesh C. Nair, N. Srinivasan, Alok Srivastava,<br />
Giridhara R Jayandharan.<br />
115<br />
New MiniPromoters with Restricted-Retinal Expression<br />
When Docked in the Mouse Genome Show the Same<br />
Expression When Delivered in AAV<br />
Elizabeth M. Simpson, Charles N. de Leeuw, Frank M. Dyka,<br />
Sanford L. Boye, Stephanie Laprise, Michelle Zhou, Alice Y. Chou,<br />
Lisa Borretta, Simone C. McInerny, Elodie Portales-Casamar,<br />
Magdalena I. Swanson, Steven J. M. Jones, Robert A. Holt, Daniel<br />
Goldowitz, William W. Hauswirth, Wyeth W. Wasserman.<br />
116<br />
AAV Serotypes 1, 8, and 9 Share a Conserved<br />
Mechanism for Axonal Transport but Exhibit<br />
Differences in Distal <strong>Gene</strong> Transfer In Vivo<br />
Michael J. Castle, April Giles, Erika L. F. Holzbaur, John H. Wolfe.<br />
117<br />
AAV6 Captron Vectors Facilitate Persistent Expression<br />
<strong>of</strong> Therapeutic Levels <strong>of</strong> Human a1-Antitrypsin in<br />
Canine Lungs<br />
Christine L. Halbert, David K. Madtes, A. D. Miller.<br />
96<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013
Abstract Directory, Thursday, May 16, 2013<br />
Poster Session I, continued<br />
4:00 PM - 6:00 PM<br />
Room: Exhibit Hall C/D<br />
AAV Vectors I<br />
118<br />
Productive Life Cycle <strong>of</strong> the AAV2 in the Complete<br />
Absence <strong>of</strong> a Conventional Polyadenylation Signal<br />
Lina Wang, Yuan Wang, Yuan Lu, George V. Aslanidi, Arun<br />
Srivastava, Changquan Ling, Chen Ling.<br />
119<br />
A Unique Biological Property <strong>of</strong> an AAV9 Mutant<br />
Showing Increased <strong>Cell</strong> Attachment in the Presence and<br />
Absence <strong>of</strong> AAV5<br />
Kei Adachi, Alex Summers, Hiroyuki Nakai.<br />
120<br />
Effective Transduction <strong>of</strong> Common Marmoset with rAAV<br />
1 and 9 To <strong>Gene</strong>rate NHP Model <strong>of</strong> Muscular Dystrophy<br />
Hironori Okada, Hidetoshi Ishibashi, Hiromi Hayashita-Kinoh,<br />
Tomoko Chiyo, Yuko Nitahara-Kasahara, Takashi Okada, Shin’ichi<br />
Takeda.<br />
121<br />
Precise Evaluation <strong>of</strong> NAb Status Against Adeno-<br />
Associated Viral Vectors and an Approach toward<br />
Managing Its Inhibitory Effect<br />
Hiroaki Mizukami, Jun Mimuro, Shuji Hishikawa, Tomokazu<br />
Ikemoto, Ryosuke Uchibori, Tomonori Tsukahara, Masashi Urabe,<br />
Akihiro Kume, Asuka Sakata, Tsukasa Ohmori, Seiji Madoiwa,<br />
Yoichi Sakata, Keiya Ozawa.<br />
122<br />
Dissection <strong>of</strong> Key Parameters Inluencing Ex Vivo AAV<br />
Library Selections<br />
Eike Kienle, Dirk Grimm.<br />
123<br />
Peptide-Mediated Retargeting <strong>of</strong> 12 AAV Serotypes<br />
Anna Sacher, Kathleen Börner, Eike Kienle, Hans-Georg<br />
Kraeusslich, Martin Mueller, Dirk Grimm.<br />
124<br />
Creation <strong>of</strong> Liver-Detargeting AAV2-Derived Mutants<br />
Based on the Knowledge <strong>of</strong> AAV9 Capsid Functions<br />
Kei Adachi, Michael Verga, Hiroyuki Nakai.<br />
125<br />
Cruciform Hairpin Structure <strong>of</strong> AAV Genome Is<br />
Speciically Subject to ATM-Dependent Silencing <strong>of</strong><br />
<strong>Gene</strong> Expression<br />
Marcela P. Cataldi, Kim Zaraspe, Haiyan Fu, Douglas M. McCarty.<br />
Adenovirus and Other DNA Virus Vectors I<br />
126<br />
Shielding the Hexon Hyper Variable Region 1 <strong>of</strong><br />
Ad5-Based <strong>Gene</strong> Transfer Vectors by PEGylation<br />
Decreases Sequestration by Erythrocytes and Increases<br />
Blood Coagulation Factor XMediated Hepatocyte<br />
Transduction<br />
Lea Krutzke, Jan-Michael Prill, Tatjana Engler, Stefan Kochanek,<br />
Florian Kreppel.<br />
127<br />
Therapeutic Eficacy in a Breast Cancer Model <strong>of</strong> Novel<br />
Conditionally Replicative Adenovirus (CRAd) Based<br />
upon the Deletion <strong>of</strong> Minor Core Protein <strong>Gene</strong> V<br />
Hideyo Ugai, George C. Dobbins, Minghui Wang, David T. Curiel.<br />
128<br />
No Recombinant Vector Sequences Detected in<br />
Tumor Tissues from Two Research Subjects Received<br />
Adenovirus Ornithine Transcarbamylase <strong>Gene</strong> <strong>Therapy</strong><br />
Li Zhong, Shaoyong Li, Mengxin Li, Yu Zhang, Brendan Lee, Mark L.<br />
Batshaw, James M. Wilson, Guangping Gao.<br />
129<br />
Oncolytic Adenovirus Expressing IL-23 and p35<br />
Elicits IFN-g- and TNF-a-Co-Producing T <strong>Cell</strong>-Mediated<br />
Antitumor Immunity<br />
Il-Kyu Choi, Yan Li, Eonju Oh, Chae-Ok Yun.<br />
130<br />
Wnt Decoy Receptor (sLRP6E1E2) Induces Antiibrotic<br />
Effect Via Inhibition <strong>of</strong> Wnt Signaling in Keloid Scars<br />
Jung-Sun Lee, Won Jai Lee, Chae-Ok Yun.<br />
131<br />
Versican Stimulates Adenoviral Transgene Expression<br />
in a JAK Dependent Manner<br />
Patricia Y. Akinfenwa, Wesley S. Bond, Mary Y. Hurwitz, Richard L.<br />
Hurwitz.<br />
132<br />
Replication <strong>of</strong> Human Adenoviruses in Non-Human<br />
<strong>Cell</strong>s In Vitro and In Vivo<br />
Catherine M. Crosby, Mallory A. Turner, Michael A. Barry.<br />
133<br />
Adenovirus Serotypes Differ in Their Susceptibility to<br />
Neutralization by Mouse Serum: Role <strong>of</strong> Coagulation<br />
Factor X<br />
Jie Tian, Zhili Xu, Andrew P. Byrnes.<br />
134<br />
Non-Covalent Coating <strong>of</strong> Adenoviral Vectors with<br />
PAMAM Dendrimer Conjugates Allows for CAR<br />
Independent Virus Uptake and Targeting to the EGF<br />
Receptor<br />
Alexandra Vetter, Kulpreet S. Virdi, Sigrid Espenlaub, Wolfgang<br />
Rödl, Ernst Wagner, Florian Kreppel, Christine Spitzweg, Manfred<br />
Ogris.<br />
Thursday, May 16, 2013<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013<br />
97
Abstract Directory, Thursday, May 16, 2013<br />
Thursday, May 16, 2013<br />
Poster Session I, continued<br />
4:00 PM - 6:00 PM<br />
Room: Exhibit Hall C/D<br />
Adenovirus and Other DNA Virus Vectors I<br />
135<br />
A <strong>Cell</strong> Line for Large-Scale Production <strong>of</strong><br />
Nondisseminative Adenoviral Vectors (Deleted<br />
<strong>of</strong> Protease [PS] <strong>Gene</strong>) for Cancer <strong>Therapy</strong> and<br />
Vaccination<br />
Rénald Gilbert, Claire Guilbault, Mélanie Simoneau, Seyyed Mehdy<br />
Elahi, Bernard Massie.<br />
136<br />
Inhibition <strong>of</strong> Breast Cancer Malignant Progression by<br />
Neutralizing Hypoxia-Induced <strong>Cell</strong> Motility<br />
Yi Lu, Liyuan Li, Jun Zhang.<br />
137<br />
HATHFUN: A Protein-Based Alternative to Viral<br />
Reprogramming<br />
Trudy Christiansen-Weber, Rodolfo Gonzalez, Alexa Dillberger,<br />
Tiffany Chu, Larisa Agapova, Alina Ostrowska, Ruslan Semechkin.<br />
138<br />
hChonJb#7 <strong>Cell</strong>s (Chondrocytes Expressing TGF-b1)<br />
Reduced Pain in a Rat Osteoarthritis Model<br />
Kyoung Baek Choi, Hyeon Youl Lee, Dae Wook Kim, Su Jeong Kim.<br />
561 (Moved from Poster Session III)<br />
Lister Strain Vaccinia Virus Demonstrates Favourable<br />
Anti-Tumor Eficacy and Tumor-Selectivity Compared<br />
to Western Reserve Strain<br />
Jonathan Hughes, Pengju Wang, Ghassan Alusi, Vipul Bhakta,<br />
Yongchao Chu, Hailiang Shi, Iain McNeish, Andrea McCart, Istvan<br />
Fodor, Nicholas Lemoine, Yaohe Wang.<br />
Physical Methods <strong>of</strong> Delivery<br />
139<br />
Electro-Chemo-<strong>Gene</strong> <strong>Therapy</strong> for Long-Term<br />
Management <strong>of</strong> Cancer in Canines<br />
Jeffry Cutrera, Xueqing Xia, Glenn King, Pamela Jones, Kristin<br />
Kicenuik, Shulin Li.<br />
140<br />
Engineering Novel Transducers for Effective<br />
Ultrasound-Mediated <strong>Gene</strong> <strong>Therapy</strong><br />
Misty L. Noble, Shuxian Song, Ryan R. Sun, Kyle P. Morrison,<br />
George W. Keilman, Luping Fan, Marla Paun, Keith R. Loeb, Carol<br />
H. Miao.<br />
141<br />
<strong>Gene</strong> Transfer Mediated by First-<strong>Gene</strong>ration Plasmids<br />
Induce Large Scale Proteomic Changes to <strong>Cell</strong>ular<br />
<strong>Program</strong>ming That Are Minimized by Elimination <strong>of</strong><br />
Prokaryotic Sequences<br />
Ramaz Geguchadze, Zhimin Wang, Lee Zourelias, Laurie Machen,<br />
Michael J. Passineau.<br />
142<br />
Development <strong>of</strong> pDNA-Loaded Bubble Liposomes for<br />
a Potential Theranostic Approach: Nano-Sized Agents<br />
Containing Cationic Lipid for Ultrasound Imaging and<br />
<strong>Gene</strong> Delivery<br />
Yoichi Negishi, Yoko Endo-Takahashi, Daichi Suzuki, Ukai Saori,<br />
Norio Takagi, Ryo Suzuki, Kazuo Maruyama, Yukihiko Aramaki.<br />
143<br />
Intracarotid Injection <strong>of</strong> Adipose Tissue-Derived<br />
Platelet-Derived Growth Factor Receptor b-Positive<br />
<strong>Cell</strong>s in Rat and Canine Model: Feasibility <strong>of</strong> <strong>Cell</strong><br />
Delivery under Mannitol-Induced Blood-Brain Barrier<br />
Opening<br />
Jung-Ick Byun, Keun-Hwa Jung, Kon Chu, Sung Won Yoon, Jong<br />
Young Lee, Soon-Tae Lee, Jeong-Min Kim, Manho Kim, Sang Kun<br />
Lee, Moon-Hee Han, Jae-Kyu Roh.<br />
144<br />
Measuring Intracellular Degradation <strong>of</strong> Polylactide-<br />
Based Magnetic Nanoparticles Using FRET<br />
Jillian E. Tengood, Alferiev S. Ivan, Fishbein Ilia, Robert J. Levy,<br />
Michael Chorny.<br />
145<br />
<strong>Gene</strong> Delivery System for Liver by Sonoporation with<br />
Nanobubble<br />
Ryo Suzuki, Yusuke Oda, Daiki Omata, Yoshikazu Sawaguchi, Yoichi<br />
Negishi, Kazuo Maruyama.<br />
146<br />
Exploring Therapeutic Applications <strong>of</strong> Pseudouridine-<br />
Containing mRNA<br />
Katalin Karikó, Hiromi Muramatsu, Norbert Pardi, Drew<br />
Weissman.<br />
147<br />
Transfection <strong>of</strong> Pseudouridine-Containing mRNA<br />
Encoding CPD-Photolyase into UVB-Irradiated Human<br />
Keratinocytes Results in Rapid Repair <strong>of</strong> DNA Damage<br />
Gábor Boros, Edit Miko, Eszter Emri, Gijsbertus T. J. van der Horst,<br />
Iren Horkay, Gabriella Emri, Eva Remenyik, Hiromi Muramatsu,<br />
Drew Weissman, Katalin Karikó.<br />
148<br />
Improvement <strong>of</strong> Both <strong>Cell</strong> Survival and Eficacy for<br />
Large Plasmids Electrotransfer in MSC<br />
Léa L. Lesueur, Lluis M. Mir, Franck M. Andre.<br />
149<br />
Minicircles Mediate Superior Ultrasound-Assisted<br />
<strong>Gene</strong> Transfer Relative to First-<strong>Gene</strong>ration Plasmids at<br />
Eqimolar Concentrations<br />
Zhimin Wang, Ramaz Geguchadze, Lee Zourelias, Laurie Machen,<br />
Michael J. Passineau.<br />
98<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013
Abstract Directory, Thursday, May 16, 2013<br />
Poster Session I, continued<br />
4:00 PM - 6:00 PM<br />
Room: Exhibit Hall C/D<br />
Physical Methods <strong>of</strong> Delivery<br />
150<br />
Multifunctional Cationic Lipid-Based Magnetic<br />
Nanoparticles for Enhanced <strong>Gene</strong> Transfection and<br />
Magnetic Resonance Imaging Applications<br />
Srinath Govindarajan, Kumiko Kitaura, Makoto Takafuji, Hirotaka<br />
Ihara, K. S. Varadarajan, Anant B. Patel, Vijaya Gopal.<br />
158<br />
Anionic, Self-Assembling Nanocomplexes for Improved<br />
Receptor-Targeted Transfection In Vitro and In Vivo<br />
Aristides D. Tagalakis, Gavin D. Kenny, Alison S. Bienemann, John<br />
A. T. Pugh, Mark F. Lythgoe, Cameron W. McLeod, Edward A. White,<br />
Stephen L. Hart.<br />
159<br />
Prostate Cancer <strong>Cell</strong>-Speciic siRNA and Drug Co-<br />
Delivery with Aptamer-Functionalized Liposomes<br />
(Aptamosomes)<br />
Gwang-Hyun Lee, Hwa Yeon Jeong, Yong Serk Park, Dong-Eun Kim,<br />
Keun-Sik Kim.<br />
151<br />
Non-Viral Enteral Hormone Gastrin <strong>Gene</strong> <strong>Therapy</strong><br />
Improves the Function and Stimulates the Proliferation<br />
<strong>of</strong> Pancreatic beta <strong>Cell</strong>s<br />
Jeonghyun Park, Hyesook Jung, Na Han, Taekyun Kim, Minjung<br />
Kwon, Soonhee Lee, Byungdoo Rhee, Mikyung Kim.<br />
152<br />
Initial Toxicology <strong>of</strong> High Dose GNE Plasmid in Rodent<br />
and Canine Animal Species<br />
Yadira Valles-Ayoub, Rosangela Carbajo, Lucia Sandoval, Zeshan<br />
Khokher, Jorge Garcia-Figueroa, Sarah Stein, Daniel No, Babak<br />
Darvish, Daniel Darvish.<br />
153<br />
Elucidating the Expression Kinetics and Iniltration<br />
Resulting from <strong>Gene</strong> Delivery Enhanced by Dermal<br />
Electroporation<br />
Janess M. Mendoza, Gleb Kichaev, Dinah Amante, Christine Knott,<br />
Trevor R. F. Smith, Niranjan Y. Sardesai, Kate E. Broderick.<br />
154<br />
The Optimization <strong>of</strong> Electroporation Parameters To<br />
Enhance the Eficiency <strong>of</strong> Plasmid <strong>Gene</strong> Delivery in<br />
Muscle<br />
Ningqing Wang, Yong Diao.<br />
160<br />
Large <strong>Gene</strong> Non-Viral Transfer Mediated by Chitosan<br />
Nanoparticles and Integrase<br />
Daniel C. Chung, Anna V. Oliviera, Hadassah Janumala, Gabriela<br />
Silva.<br />
161<br />
Co-Delivery <strong>of</strong> siRNA and pDNA for Increased<br />
Transfection Eficiency<br />
Laura Rose, Michael Hogarth, Hasan Uludag.<br />
162<br />
Stability <strong>of</strong> Polymer/Plasmid DNA Complexes In Vitro<br />
and In Vivo<br />
Laura Rose, Jeremy Fife, Cezary Kucharski, Hasan Uludag.<br />
163<br />
When Nucleic Acids Meet Cationic Polymers: Effects <strong>of</strong><br />
Mixing Sequence on Complexation and Transfection<br />
Eficiencies<br />
Soo Kyung Cho, Xi Wang, Regina Ragan, Young Jik Kwon.<br />
164<br />
Bioreducible and Acid-Labile Poly(Amido Amine)s for<br />
Eficient <strong>Gene</strong> Delivery<br />
Qinghui Zhou, Yezhi You, Zhiqiang Yu, Junjie Yan.w<br />
Thursday, May 16, 2013<br />
Chemical and Molecular Conjugates<br />
155<br />
A High Throughput Screening Approach To Identify<br />
<strong>Gene</strong> Transfer Peptides In Vitro<br />
Jing Li, Jason Duskey, Kevin G. Rice.<br />
156<br />
Targeted Delivery <strong>of</strong> Short Interfering RNA by Human<br />
TAR RNA-Binding Protein-Afibody Chimera<br />
Gulam H. Dar, Erica Nathan, Nalam M. Rao, Vijaya Gopal.<br />
157<br />
Oligopeptide Comb Architectures for Controlled DNA<br />
Binding and Non-Viral Transfection<br />
Sangram S. Parelkar, Rachel Letteri, Delphine Chan-Seng, Todd<br />
Emrick.<br />
165 Abstract Withdrawn from Presentation<br />
Musculo-skeletal <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> I<br />
166<br />
Micro-Dystrophin Ameliorates Muscular Dystrophy in<br />
the Canine Model <strong>of</strong> Duchenne Muscular Dystrophy<br />
Jin-Hong Shin, Xiufang Pan, Chady H. Hakim, Hsiao T. Yang,<br />
Yougping Yue, Keqing Zhang, Ronald L. Terjung, Dongsheng Duan.<br />
167<br />
Expression <strong>of</strong> Minidystrophin from the Dystrophin<br />
Locus by TALEN Mediated <strong>Gene</strong> Targeting<br />
David G. Ousterout, Pablo Perez-Pinera, Pratiksha I. Thakore, Xiao<br />
Xiao, Charles A. Gersbach.<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013<br />
99
Abstract Directory, Thursday, May 16, 2013<br />
Thursday, May 16, 2013<br />
Poster Session I, continued<br />
4:00 PM - 6:00 PM<br />
Room: Exhibit Hall C/D<br />
Musculo-skeletal <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> I<br />
168<br />
Activation, Expansion, and In Vivo Regenerative<br />
Potential <strong>of</strong> Pax3-Transduced Satellite <strong>Cell</strong>s: A Potential<br />
<strong>Cell</strong>-Based <strong>Therapy</strong> for Duchenne Muscular Dystrophy<br />
Antonio Filareto, Robert W. Arpke, Darko Bosnakovski, Radbod<br />
Darabi, Fabrizio Rinaldi, Auston Miller, Michael Kyba, Rita C. R.<br />
Perlingeiro.<br />
169<br />
<strong>Gene</strong>tic Correction <strong>of</strong> Facioscapulohumeral Dystrophy<br />
Using Telomere Insertion: Potential for Therapeutic<br />
Application<br />
Fabrizio Rinaldi, Linn Hartweck, Darko Bosnakovski, Erik<br />
Toso, Abhijit Dandapat, Radbod Darabi, Thomas Zwaka, Simon<br />
Nargarkhan, Rita Perlingeiro, Michael Kyba.<br />
170<br />
Enhanced Myogenic Differentiation Induced by<br />
Engineered MyoD Transcription Factors<br />
Ami M. Kabadi, Pratiksha I. Thakore, Charles A. Gersbach.<br />
171<br />
Viral Mediated Acute Mouse Model for Myotonic<br />
Dystrophy Type1<br />
Syed-Rehan A. Hussain, Lindsay Wallace, Danielle Grifin, Chrystal<br />
Montgomery, Scott Q. Harper, Zarife Sahenk, K. Reed Clark.<br />
172<br />
Pre-Clinical Eficacy Study <strong>of</strong> Beta-Sarcoglycan <strong>Gene</strong><br />
Transfer<br />
Eric R. Pozsgai, William E. Grose, Danielle A. Grifin, Kristin N.<br />
Heller, Jerry R. Mendell, Louise R. Rodino-Klapac.<br />
173<br />
The Cardiac Alkali Myosin Light Chain Can Restore<br />
Skeletal Muscle Function in a Mouse Model <strong>of</strong> Nemaline<br />
Myopathy<br />
Johan M. Lindqvist, Julien Ochala.<br />
176<br />
Engraftment <strong>of</strong> Mesenchymal Stromal <strong>Cell</strong>s That Can<br />
Differentiate To Form Myogenic <strong>Cell</strong>s Is Enhanced<br />
by Expressing IL-10 in Dog with Duchenne Muscular<br />
Dystrophy<br />
Yuko N. Kasahara, Hiromi H. Kinoh, Tomoko Chiyo, Hironori<br />
Okada, Takashi Okada, Shin’ichi Takeda.<br />
177<br />
Modulating <strong>Cell</strong> Cycle Inhibitor Expression To Enhance<br />
Skeletal Myoblast Engraftment<br />
Pratiksha I. Thakore, David G. Ousterout, Charles A. Gersbach.<br />
178<br />
Human Muscle Derived Stem <strong>Cell</strong>s Exhibit Bone<br />
Regeneration Capacity Similar to Bone Marrow<br />
Mesenchymal Stem <strong>Cell</strong>s When <strong>Gene</strong>tically Modiied To<br />
Express BMP2 Both In Vitro and In Vivo<br />
Xueqin Gao, Arvydas Usas, Ying Tang, Aiping Lu, Jian Tan, Johannes<br />
Schneppendahl, Nick Oyster, Bing Wang, Rocky Tuan, Johnny<br />
Huard.<br />
179<br />
Rapid Progression <strong>of</strong> Muscular Dystrophy in<br />
Dystrophin/Utrophin-/- Mice Is Associated with Muscle<br />
Progenitor <strong>Cell</strong> Exhaustion<br />
Aiping Lu, Minakshi Poddar, Ying Tang, Jonathan D. Proto,<br />
Xiaodong Mu, Bing Wang, Johnny Huard.<br />
180<br />
A New Mouse Model <strong>of</strong> DMD: A Tool for Therapeutic<br />
Development Directed at Exon Duplications<br />
Adeline Vulin, Nicolas Wein, Yuuki J. Kaminoh, Andrew R. Findlay,<br />
Kevin M. Flanigan.<br />
181<br />
Investigation <strong>of</strong> Dystromirs in the mdx Mouse<br />
Thomas C. Roberts.<br />
182<br />
Dose-Dependent Toxicity <strong>of</strong> Humanized Renilla<br />
Reniformis GFP (hrGFP) Limits Its Utility as a Reporter<br />
<strong>Gene</strong> in Mouse Muscle<br />
Lindsay M. Wallace, Andrew Moreo, K. Reed Clark, Scott Q. Harper.<br />
174<br />
<strong>Gene</strong>rating Dystrophin+ Myogenic <strong>Cell</strong>s from mdx<br />
Fibroblasts by Using a Triple-Recombinase iPS <strong>Cell</strong>-<br />
Based Strategy<br />
Jonathan M. Geisinger, Chunli Zhao, Alfonso P. Farrugio,<br />
Christopher L. Chavez, Christopher R. Bjornson, Tawny L. Neal,<br />
Michele P. Calos.<br />
175<br />
Alpha7 Integrin Partially Preserves the Mechanical<br />
Function <strong>of</strong> the Extensor Digitorum Longus Muscle in<br />
Dystrophin-Deicient Mice<br />
Chady H. Hakim, Dean J. Burkin, Dongsheng Duan.<br />
Infectious Diseases and Vaccines<br />
183<br />
Electroporation <strong>of</strong> ZFN mRNA Enables Eficient CCR5<br />
<strong>Gene</strong> Disruption in Mobilized Blood Hematopoietic<br />
Stem <strong>Cell</strong>s at Clinical Scale<br />
Paula M. Cannon, Jill E. Henley, Travis Wood, Lynn Truong, Jianbin<br />
Wang, Kenneth Kim, Jenny Yan, Philip D. Gregory, Gary Lee,<br />
Michael C. Holmes.<br />
100<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013
Abstract Directory, Thursday, May 16, 2013<br />
Poster Session I, continued<br />
4:00 PM - 6:00 PM<br />
Room: Exhibit Hall C/D<br />
Infectious Diseases and Vaccines<br />
184<br />
Next <strong>Gene</strong>ration TALENs Mediate Eficient Disruption <strong>of</strong><br />
the CCR5 <strong>Gene</strong> in Human HSCs<br />
Nick Llewellyn, Jeffrey C. Miller, John Campo, Colin Exline, Orla<br />
Mulhern, Jill Henley, Jianbin Wang, Dmitry Guschin, Danny Xia,<br />
Irina Ankoudinova, Philip D. Gregory, Lei Zhang, Edward J. Rebar,<br />
Michael C. Holmes, Paula M. Cannon.<br />
185<br />
Enhanced Neutralising Antibody Response to Bovine<br />
Viral Diarrhoea Virus (BVDV) Induced by BVDV DNA<br />
Vaccine Co-Expressing RIG-I Agonist in Cattle<br />
Laila M. El-Attar, Olivia Anstaett, Carole Thomas, Jeremy Luke,<br />
James Williams, Joe Brownlie.<br />
186<br />
Preclinical Safety Evaluation <strong>of</strong> an Antibiotic-Free<br />
LAMP-1 Plasmid Allergy Immunotherapy<br />
Teri L. Heiland, James A. Williams, William G. Hearl.<br />
187<br />
Inhibition <strong>of</strong> HBV Replication through ZFN-Mediated<br />
Disruption <strong>of</strong> cccDNA<br />
Daniel Stone, Nick Weber, Harshana De Silva Feelixge, Keith R.<br />
Jerome.<br />
188<br />
Development <strong>of</strong> Designer Homing Endonucleases for<br />
Inactivation <strong>of</strong> the HIV Provirus<br />
Esther Mintzer, Jordan Jarjour, Andy Scharenberg, Barry Stoddard,<br />
David Baker, Nick Weber, Daniel Stone, Keith R. Jerome.<br />
189<br />
Whole <strong>Cell</strong>-SELEX Derived Anti-CD7 Aptamers for the<br />
Delivery <strong>of</strong> Anti-CCR5 siRNA: A Prohylactic Approach to<br />
HIV <strong>Therapy</strong><br />
Laura Millroy, Daniel Lazar, Makobetsa Khati, Marc S. Weinberg.<br />
190<br />
Capsid Modiied AAV2 Vectors Are Capable <strong>of</strong><br />
<strong>Gene</strong>rating Functional Cytotoxic T <strong>Cell</strong>s in a Prime-<br />
Boost Vaccine Protocol<br />
Brad E. H<strong>of</strong>fman, Irene Zolotukhin, Roland W. Herzog, Ou Cao.<br />
191<br />
The Anti-Genomic (Negative) Strand <strong>of</strong> Hepatitis C<br />
Virus Is Not Targetable by shRNA<br />
Leszek Lisowski, Menashe Elazar, Kirk Chu, Jeffrey S. Glenn, Mark<br />
A. Kay.<br />
192<br />
Development <strong>of</strong> the Oral Universal Type A Inluenza<br />
Vaccine Using Biidobacterium Displaying Inluenza-<br />
M2e Protein<br />
Naoya Morishita, Suzuka Ozaki, Koichi Kitagawa, Chihiro<br />
Shirosaki, Hiroki Saito, Katsumi Shigemura, Masato Fujisawa,<br />
Masato Kawabata, Toshiro Shirakawa.<br />
193<br />
Enzyme Engineering <strong>of</strong> Homing Endonucleases To<br />
Target HBV cccDNA<br />
Nicholas D. Weber, Ryo Takeuchi, Jordan Jarjour, Daniel Stone,<br />
Barry L. Stoddard, Keith R. Jerome.<br />
194<br />
In Vivo Electroporation <strong>of</strong> Minicircle DNA as a Noval<br />
Method <strong>of</strong> Vaccine Delivery<br />
Wenlin Huang, Qingtao Wang, Changchuan Pan, Shijuan Gao, Wei<br />
Jiang.<br />
195<br />
The Potential Therapeutic Role <strong>of</strong> Secreted Antiviral<br />
Entry Inhibitory (SAVE) Peptides Expressed by<br />
Transduced MSCs To Block Viral Replication<br />
Narae Lee, Edith Walker, Wenpeng Li, Lisa Egerer, Dorothee von<br />
Laer, Bruce A. Bunnell, Stephen E. Braun.<br />
Cancer Immunotherapy I<br />
196 Abstract Withdrawn<br />
197<br />
Dual Transgenesis <strong>of</strong> T <strong>Cell</strong>s with a CD44v6-Speciic<br />
CAR and a Suicide <strong>Gene</strong> for the Safe Eradication <strong>of</strong><br />
Myeloid Leukemia and Myeloma<br />
Monica Casucci, Benedetta Nicolis di Robilant, Laura Falcone,<br />
Barbara Camisa, Pietro Genovese, Bernhard Gentner, Claudio<br />
Bordignon, Barbara Savoldo, Fabio Cicieri, Luigi Naldini,<br />
Gianpietro Dotti, Chiara Bonini, Attilio Bondanza.<br />
198<br />
Capitalizing on Epitope Escape To Avoid T <strong>Cell</strong><br />
Fratricide without Compromising Targeting <strong>of</strong> CD56+<br />
Tumors<br />
Denise L. Crossland, Sonny Ang, Simon Olivares, Kirsten Switzer,<br />
Tiejuan Mi, Helen Huls, Laurence J. N. Cooper.<br />
199<br />
Phase II Study <strong>of</strong> FANG (Autologous Tumor <strong>Cell</strong><br />
– GMCSF/pbi-shRNA Furin DNA Plasmid) Vaccine in<br />
Advanced Melanoma<br />
John Nemunaitis, Neil Senzer, Minal Barve, Padmasini Kumar,<br />
Donald D. Rao, Gladice Wallraven, Beena O. Pappen, Jospeh Kuhn,<br />
Peter Beitsch, Robert Steckler, Brian M. Gogel, Walton Taylor,<br />
Phillip B. Maples.<br />
200<br />
Renal <strong>Cell</strong> Tumor-Mediated Reprogramming <strong>of</strong> Natural<br />
Killer <strong>Cell</strong>s by Soluble Immune Modulators<br />
Yue Guan, Purba Singh, Trenae Mann, Christopher B. Chambers,<br />
Donald S. Torry, Andrew Wilber.<br />
Thursday, May 16, 2013<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013<br />
101
Abstract Directory, Thursday, May 16, 2013<br />
Thursday, May 16, 2013<br />
Poster Session I, continued<br />
4:00 PM - 6:00 PM<br />
Room: Exhibit Hall C/D<br />
Cancer Immunotherapy I<br />
201<br />
A New Epithelial Junction Opener for Cancer <strong>Therapy</strong><br />
Ines Beyer, Hua Cao, Jonas Persson, Roma Yumul, Andre Lieber.<br />
202<br />
A Chemotherapy-Selective Method for Expansion <strong>of</strong><br />
Transgenic T <strong>Cell</strong>s<br />
David Rushworth, Laurence Cooper.<br />
203<br />
Phase I Study <strong>of</strong> the “Triad” Autologous (FANG)<br />
Vaccine, Incorporating Bifunctional shRNAfurin and<br />
GMCSF Transgene Expression, in Advanced Ewing’s<br />
Sarcoma: Preliminary Data in Pediatrics Patients<br />
Maurizio Ghisoli, Carl Lenarsky, Gladice Wallraven, Padmasini<br />
Kumar, Phillip B. Maples, John Nemunaitis.<br />
204<br />
Dissecting the Human T <strong>Cell</strong> Receptor Repertoire<br />
in Different Immunological Conditions by Deep<br />
Sequencing<br />
Eliana Ruggiero, Jan P. Nicolay, Anne Arens, Raffaele Fronza, Anna<br />
Paruzynski, Ali Nowrouzi, Gökçe Ürenden, Sergij Goerdt, Hanno<br />
Glimm, Peter H. Krammer, Manfred Schmidt, Christ<strong>of</strong> von Kalle.<br />
205<br />
Aptamer Targeted Antigen Delivery<br />
Matthew Levy, Brian C. Wengerter, Debborah Palliser, Joseph<br />
Katakowski.<br />
206<br />
FANG Cytokine Expression Analyses Update<br />
Padmasini Kumar, Connor Phalon, Beena O. Pappen, Gladice<br />
Wallraven, Yang Yu, Fabienne Norvell, Christopher M. Jay, Zhaohui<br />
Wang, Donald D. Rao, John Nemunaitis, Neil Senzer, Phillip B.<br />
Maples.<br />
207<br />
Effect <strong>of</strong> Interleukin-15 and Dendritic <strong>Cell</strong>s on the<br />
Phenotype and Cytotoxicity <strong>of</strong> Cytokine-Induced Killer<br />
<strong>Cell</strong>s<br />
Xianghui He, Zhaohui Lai, Na Zhao, Liwei Zhu.<br />
208<br />
Investigating Macrophage Induction <strong>of</strong> Metastasis: A<br />
Precursor To Identify Therapeutic Targets<br />
Trevor Memmott, Evita Weagel, Justin Livingston, Pingguo Liu, Atif<br />
Elnaggar, Richard Robison, Kim O’Neill.<br />
209 Abstract Withdrawn from Presentation<br />
Cancer-Targeted <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>: Viral<br />
Vector / siRNA-Mediated Therapies<br />
210<br />
Modelling Viral Insertion Sites Via Network Based<br />
Frameworks<br />
Raffaele Fronza, Cynthia Bartholomae, Annette Deichmann,<br />
Stephanie Laufs, Christ<strong>of</strong> von Kalle, Manfred Schmidt.<br />
211<br />
rAAV Mediated Modulation <strong>of</strong> miR-1 Inluences the<br />
Progression <strong>of</strong> Hepatocellular Carcinoma in a Mouse<br />
Model <strong>of</strong> Alpha-1 Antitrypsin<br />
Florie Borel, Qiushi Tang, Nancy Marcus, Jeffery Teckman, Terence<br />
Flotte, Christian Mueller.<br />
212<br />
AAV Mediated IFNb <strong>Gene</strong> <strong>Therapy</strong> <strong>of</strong> Orthotopic<br />
Xenograft Mouse Model <strong>of</strong> Invasive Glioblastoma<br />
multiforme<br />
Dwijit GuhaSarkar, James Neiswender, Miguel Sena-Esteves.<br />
213<br />
Response <strong>of</strong> Drug Sensitive and Resistant Breast Cancer<br />
<strong>Cell</strong>s to Combinatorial siRNA <strong>Therapy</strong><br />
Hamidreza Montazeri Aliabadi, Parvin Mahdipoor, Hasan Uludag.<br />
214<br />
Combination <strong>Therapy</strong> with Novel Nanoparticle,<br />
SNS01-T, and Bortezomib Results in Synergistic<br />
Cytotoxicity In Vitro and In Vivo in Multiple Myeloma<br />
Catherine Taylor, Qifa Zheng, Zhongda Liu, Terence Tang, Sarah<br />
Francis, Richard Dondero, John Thompson.<br />
215<br />
Rapamycin Enhances Adenovirus-Mediated Cancer<br />
Diagnosis in Naive and Pre-Immunized Hosts<br />
Ziyue Karen Jiang, Mai Johnson, Diana Moughon, Jennifer Kuo,<br />
Makoto Sato, Lily Wu.<br />
216<br />
Anti-Tumor Effects <strong>of</strong> Ad-p53 on INK4A/ARF-Defective<br />
Mesothelioma Are Inluenced Positively by Small G<br />
Protein Inhibitors but Negatively by Heat Shock Protein<br />
90 Inhibitors<br />
Masatoshi Tagawa, Kuan Chai, Yuanyuan Jiang, Kiyoko Kawamura,<br />
Shan Yang, Atsushi Ogino, Naoto Yamaguchi, Kazunori Kato, Yuji<br />
Tada, Yuichi Takiguchi, Koichiro Tatsumi, Hideaki Shimada, Kenzo<br />
Hiroshima, Shuji Kubo.<br />
217<br />
Lentiviral shRNA Targeting NANOG/NANOGP8<br />
Enhances Topotecan Chemotherapy in Human<br />
Colorectal Carcinomas<br />
John M. Jessup, Jingyu Zhang, Luis A. Espinoza, Abid Mattoo,<br />
Snorri S. Thorgeirsson.<br />
102<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013
Abstract Directory, Thursday, May 16, 2013<br />
Poster Session I, continued<br />
4:00 PM - 6:00 PM<br />
Room: Exhibit Hall C/D<br />
Cancer-Targeted <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>: Viral<br />
Vector / siRNA-Mediated Therapies<br />
218<br />
Comparative Evaluation <strong>of</strong> the Antitumor Activity<br />
<strong>of</strong> Anti-Angiogenic Proteins Delivered by Lentivirus<br />
Vector in Human Malignant Mesothelioma Model<br />
Misato Takagi-Kimura, Tomoko Hashimoto-Tamaoki, Noriyuki<br />
Kasahara, Shuji Kubo.<br />
219<br />
Clinical Development <strong>of</strong> “Bifunctional” shRNA<br />
Knockdown <strong>of</strong> Acid Ceramidase<br />
James Norris, Xiang Liu, Donald D. Rao, Zhaohui Wang, Neil Senzer,<br />
John Nemunaitis.<br />
220<br />
Photochemical Internalization (PCI)-Enhanced<br />
Nonviral <strong>Gene</strong>-Directed Enzyme Prodrug Cancer<br />
<strong>Therapy</strong><br />
Henry Hirschberg, Young Jik Kwon.<br />
221<br />
TRAIL-R2 and HRK Mediate Angiotensin Type 2<br />
Receptor-Induced Apoptosis <strong>of</strong> Human Prostate Cancer<br />
<strong>Cell</strong>s<br />
Nana Pei, Yanling Zhang, Feilong Jie, Jie Luo, Xinglu Chen, Jinlong<br />
Li, Hongwei Li.<br />
225<br />
In Vitro Repair <strong>of</strong> Rhodopsin Mutation by Spliceosome-<br />
Mediated RNA Trans-Splicing<br />
Adeline Berger, Stéphanie Lorain, Charlène Joséphine, Mélissa<br />
Desrosiers, Peggy Fabre, Cecile Peccate, Thomas Voit, Luis Garcia,<br />
José-Alain Sahel, Alexis-Pierre Bemelmans.<br />
226<br />
Muller <strong>Cell</strong> Reactivity Despite Neuronal Preservation<br />
after Systemic <strong>Gene</strong> <strong>Therapy</strong><br />
Tonia S. Rex.<br />
227<br />
<strong>Gene</strong> Replacement <strong>Therapy</strong> for Choroideremia<br />
Aaron D. Black, Vidyullatha Vasireddy, Rajashekhar Gaddameedi,<br />
Daniel C. Chung, Albert M. Maguire, Jeannette L. Bennicelli, Tanya<br />
Tolmachova, Miguel C. Seabra, Jean Bennett.<br />
228<br />
Adeno-Associated Vector (Type 8)-Mediated Expression<br />
<strong>of</strong> siRNA Targeting Vascular Endothelial Growth Factor<br />
Eficiently Inhibits Neovascularization in a Murine<br />
Choroidal Neovascularization Model<br />
Tsutomu Igarashi, Koichi Miyake, Noriko Miyake, Osamu Iijima,<br />
Chiemi Yaguchi, Takashi Shimada, Hiroshi Takahashi.<br />
229<br />
AAV-Mediated Expression <strong>of</strong> Secreted Human Ciliary<br />
Neurotropic Factor (hCNTF) Leads to Long-Term<br />
Preservation <strong>of</strong> Cone Photoreceptors in an Autosomal<br />
Recessive Model <strong>of</strong> Retinitis Pigmentosa<br />
Daniel M. Lipinski, Alun R. Barnard, Mandeep S. Singh, Edward<br />
Lee, Robert E. MacLaren.<br />
Thursday, May 16, 2013<br />
Sensory (Ophthalmic and Auditory) <strong>Gene</strong> &<br />
<strong>Cell</strong> <strong>Therapy</strong><br />
222<br />
CEP290 Directly Anchors Microtubules to the<br />
Membrane and Its N- and C- Termini Inhibit Protein<br />
Function and Ciliogenesis<br />
Theodore G. Drivas, Jean Bennett.<br />
223<br />
Dual Adeno-Associated Virus Vectors for Delivery <strong>of</strong><br />
Large <strong>Gene</strong>s to the Retina<br />
Frank M. Dyka, Sanford L. Boye, Shannon E. Boye, William W.<br />
Hauswirth.<br />
224<br />
Induced Pluripotent Stem <strong>Cell</strong>s as a Model To Test <strong>Gene</strong><br />
Augmentation <strong>Therapy</strong> for Choroideremia<br />
Vidyullatha Vasireddy, Jason A. Mills, Rajashekhar Gaddameedi,<br />
Etiena Basner-Tschakarjan, Monica Kohnke, Aaron H. Black, Krill<br />
Alexandrov, Albert M. Maguire, Daniel Chung, Helen Mac, Lisa<br />
Sullivan, Paul Gadue, Jeannette L. Bennicelli, Deborah L. French,<br />
Jean Bennett.<br />
230<br />
The Anti-Angiogenic Capacity <strong>of</strong> POMC <strong>Gene</strong> Delivery:<br />
From Cancer to Blindness<br />
Guei-Sheung Liu, Ming-Hong Tai, Gregory J. Dusting.<br />
231<br />
Genotype to Phenotype Relationship in CEP290<br />
Mutations: Improving <strong>Gene</strong> <strong>Therapy</strong> Strategies for the<br />
Most Common Form <strong>of</strong> Leber’s Congenital Amaurosis<br />
Adam P. Wojno, Theodore G. Drivas, Jean Bennett.<br />
232<br />
The LentiVector® <strong>Gene</strong> <strong>Therapy</strong> Platform for Ocular<br />
Disease: A Clinical Update<br />
Yatish Lad, Kyriacos Mitrophanous, Scott Ellis, James Miskin, Katie<br />
Binley, Michelle Kelleher, Cherry Lucas, Stuart Naylor.<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013<br />
103
Abstract Directory, Thursday, May 16, 2013<br />
Poster Session I, continued<br />
4:00 PM - 6:00 PM<br />
Room: Exhibit Hall C/D<br />
<strong>Gene</strong> Regulation<br />
233<br />
Lentiviral Vector-Based Insertional Mutagenesis<br />
Identiies New Oncogenes and Molecular Networks<br />
That Control Hepatocyte Transformation and<br />
Metabolism<br />
Marco Ranzani, Daniela Cesana, Cynthia Bartholomae, Francesca<br />
Sanvito, Stefano Annunziato, Michela Riba, Fabrizio Benedicenti,<br />
Pierangela Gallina, Claudio Doglioni, Christ<strong>of</strong> von Kalle, Yoon Jun<br />
Kim, Elia Stupka, Manfred Schmidt, Giovanni Tonon, Luigi Naldini,<br />
Eugenio Montini.<br />
240<br />
Spacio-Temporal Mutagenesis in Muscle by Using AAV-<br />
Cre Vectors: The Example <strong>of</strong> the Mtm1 <strong>Gene</strong><br />
Romain Joubert, Alban Vignaud, Mickaël Le, Christelle Moal, Nadia<br />
Messaddeq, Anna Buj-Bello.<br />
241<br />
Synergistic and Tunable <strong>Gene</strong> Activation by<br />
Combinations <strong>of</strong> Synthetic Transcription Factors<br />
Pablo Perez-Pinera, David G. Ousterout, Jonathan M. Brunger,<br />
Alicia M. Farin, Katherine A. Glass, Farshid Guilak, Gregory E.<br />
Crawford, Alexander J. Hartemink, Charles A. Gersbach.<br />
242<br />
A Novel Enhancer Increases the Level and Duration <strong>of</strong><br />
Transgene Expression in Mice<br />
Chunbo Zhang, Leping Li, Dexi Liu.<br />
Thursday, May 16, 2013<br />
234<br />
Detection <strong>of</strong> RNA Interference (RNAi) Mediated mRNA<br />
Cleavage in Fresh Injected Tumor Tissue from Patients<br />
a Phase I Trial <strong>of</strong> an Intratumoral pbi-shRNA Lipoplex<br />
Targeting Stathmin-1<br />
Zhaohui Wang, Donald D. Rao, Phillip B. Maples, Neil Senzer, John<br />
Nemunaitis.<br />
235<br />
LncRNA Saf Interacts with SPF45 To Promote Fas<br />
Receptor Alternative Splicing in Maturing Erythroid<br />
<strong>Cell</strong>s<br />
Olga Villamizar, Christopher B. Chambers, Janice M. Riberdy, Derek<br />
A. Persons, Andrew Wilber.<br />
236<br />
Stable and Selective Production <strong>of</strong> Therapeutic Protein<br />
in the Breastmilk <strong>of</strong> Non-Transgenic Mice Via Early<br />
Gestational Intra-Amniotic <strong>Gene</strong> Transfer<br />
Jesse D. Vrecenak, Miroslaw Kozlowski, Carlyn A. Todorow,<br />
Antoneta Radu, Alan W. Flake.<br />
237<br />
Improved Engraftment <strong>of</strong> Lentivirally Transduced<br />
Adipose Tissue-Derived Stem <strong>Cell</strong>s for Hemophilia B<br />
<strong>Therapy</strong> Using Bioengineered <strong>Cell</strong> Sheet Technology<br />
Inkyong Shim, Kazuo Ohashi, Kohei Tatsumi, Natsumi Watanabe,<br />
Yuji Kashiwakura, Tsukasa Ohmori, Yoichi Sakata, Makoto Inoue,<br />
Mamoru Hasegawa, Teruo Okano.<br />
238<br />
Targeted PLGA Nanoparticles Encapsulating Histone<br />
Deacetylase Inhibitor, Panobinostat Effectively Control<br />
T <strong>Cell</strong> Leukemia<br />
Boyoung Choi, Jangwook Lee, Jeong-A. Shin, Dahye Lee, Kuen Yong<br />
Lee, Priti Kumar, Sang-Kyung Lee.<br />
239<br />
Radiosensitivity <strong>of</strong> Human Induced Pluripotent Stem<br />
<strong>Cell</strong>s (hiPSC) Detectable by <strong>Cell</strong> Cycle Analysis<br />
Frank Houghton, Michael W. Epperly, Xichen Zhang, Vishwajit<br />
Nimgaonkar, Joel S. Greenberger.<br />
243<br />
Design and Screening <strong>of</strong> Custom Zinc Finger<br />
Transcription Factors To Modulate Expression <strong>of</strong> <strong>Gene</strong>s<br />
Controlling <strong>Cell</strong> Fate<br />
Ashley L. Fischer, Qingzhou Ji, Erik R. Eastlund, Mark A. Gerber,<br />
Carol A. Kreader, Scott W. Knight.<br />
244<br />
TaqMan® Assays for Assessment <strong>of</strong> Genome,<br />
Epigenome and Quality <strong>of</strong> Pluripotent Stem <strong>Cell</strong>s<br />
Andrew Fontes, Rene H. Quintanilla, Jeffrey Fergus, Uma<br />
Lakshmipathy.<br />
245<br />
An ‘Open-Source’ Approach to Simple and Precise<br />
Genome Editing with rAAV<br />
Rob Howes, Alex Rieck, Eric Rhodes.<br />
246<br />
UV Light Modulates Transcriptional Activity <strong>of</strong> Egr-1<br />
Promoter on Human Primary Tenocytes<br />
Francisco Martinez-F, Araceli Barrera-Lopez, Hugo Sandoval-<br />
Zamora, David T. Curiel, Juan A. Madinaveitia-V, Rebecca E.<br />
Franco-Bourland.<br />
Fetal and Adult Stem <strong>Cell</strong>s<br />
247<br />
Embryonic Renal Progenitor <strong>Cell</strong>s Activate the<br />
b-Catenin/TCF Signaling Pathway during Integration<br />
into Damaged Adult Renal Tubules<br />
Paul Goodyer, Zhao Zhang, Diana Iglesias.<br />
248<br />
Preclinical Investigations <strong>of</strong> Therapeutic Beneits and<br />
Biodistribution from Stress Erythropoiesis on Minimal<br />
Dose <strong>of</strong> Lysosomal Enzyme Required for Phenotypic<br />
Correction in Mice with Hurler Syndrome<br />
Jingfen Han, Mei Dai, Salim S. Elamouri, Phuong Cao, Dao Pan.<br />
104<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013
Abstract Directory, Thursday, May 16, 2013<br />
Poster Session I, continued<br />
4:00 PM - 6:00 PM<br />
Room: Exhibit Hall C/D<br />
Fetal and Adult Stem <strong>Cell</strong>s<br />
249<br />
Evidence <strong>of</strong> a Functional Role <strong>of</strong> Co-Culture in Delta-<br />
Mediated Expansion<br />
Korashon L. Watts, Colleen Delaney, Hans-Peter Kiem.<br />
250<br />
Rapidly Adhering <strong>Cell</strong>s (RACs) Residing in Skeletal<br />
Muscle Contribute to Ectopic Adipose <strong>Cell</strong> Formation/<br />
Accumulation in Skeletal Muscle <strong>of</strong> Dystrophin/<br />
Utrophin-/- Mice<br />
Jihee Sohn, Aiping Lu, Ying Tang, Bing Wang, Johnny Huard.<br />
251<br />
PGE2 Increases Lentiviral Vector Transduction<br />
Eficiency <strong>of</strong> Human HSC<br />
Garrett C. Heffner, Lauryn Christiansen, Francis J. Pierciey, Jr,<br />
Robert Kutner, Mitchell Finer, Gabor Veres.<br />
252 Abstract Withdrawn from Presentation<br />
253<br />
Amniotic Fluid Stem <strong>Cell</strong>s for the Treatment <strong>of</strong><br />
Hemophilia A<br />
Chung-Jung Kuo, Saloomeh Mokhtari, Melisa Soland, Shay Soker,<br />
James Yoo, John Owen, Mary Ann Knovich, Anthony Atala, Graca<br />
Almeida-Porada, Christopher Porada.<br />
254<br />
Characterization <strong>of</strong> Tumor Suppressive Effect Mediated<br />
by Human Bone Marrow-Derived Mesenchymal Stem<br />
<strong>Cell</strong>s<br />
Yulyana Yulyana, Ivy A. W. Ho, Jerry K. Y. Chan, Paula YP Lam.<br />
255<br />
Non-Invasive High-Resolution Imaging <strong>of</strong> Muscle<br />
Regeneration with a New Red-Absorbing Fluorescent<br />
Protein<br />
Jun Chu, Russell D. Haynes, Stephane Y. Corbel, Pengpeng Li,<br />
Emilio Gonzalez-Gonzalez, Palula J. Cranill, Michelle Baird,<br />
Michael W. Davidson, Christopher H. Contag, Kang Shen, Helen M.<br />
Blau, Michael Z. Lin.<br />
256<br />
Effects <strong>of</strong> Nano-Pillar Arrays on Differentiation <strong>of</strong><br />
Human Mesenchymal Stem <strong>Cell</strong>s<br />
Hyuk Sang Yoo, Hyesung Kim.<br />
257<br />
Integrating Non-Viral Sleeping Beauty Vector<br />
Delivery <strong>of</strong> Sox Transcription Factors Enhances MSC<br />
Chondrogenesis<br />
Laila Begum, Alan J. Nixon.<br />
258<br />
Variables That Affect Transduction <strong>of</strong> CD34+<br />
Hematopoietic Stem <strong>Cell</strong>s<br />
Francis J. Pierciey, Jr, Lauryn M. Christiansen, Robert Kutner,<br />
Garrett C. Heffner, Gabor Veres.<br />
<strong>Cell</strong> Processing and Vector Production<br />
259<br />
Assembly-Activating Protein Is Highly Tolerant To Point<br />
Mutations without Loss <strong>of</strong> Function: A High-Throughput<br />
Mutagenesis Screen Utilizing DNA Barcoding<br />
Lauriel Earley, Kei Adachi, Hiroyuki Nakai.<br />
260<br />
Development <strong>of</strong> a Validated Method Offering a<br />
Potentially Standardized Biological Assay for the<br />
Titration <strong>of</strong> HIV-1 Based Lentiviral Vectors<br />
Christian Skorik, Wesley Gorman, Mitch Finer, Mark D. Angelino,<br />
Robert H. Kutner.<br />
261<br />
Technology Transfer <strong>of</strong> a High Yield Fermentation<br />
Process and cGMP Production <strong>of</strong> Antibiotic-Free<br />
Plasmid DNA Vaccines<br />
Philippe Ledent, Olivier Geurten, Laurent Jost, Marc Daukandt,<br />
James A. Williams, Aaron Carnes.<br />
262<br />
Development <strong>of</strong> a Scale-Down Manufacturing Model for<br />
<strong>Gene</strong>ration <strong>of</strong> HIV-1 Based Lentiviral Vectors<br />
Wesley C. Gorman, Christian Skorik, Mitch Finer, Mark D. Angelino,<br />
Robert H. Kutner.<br />
263<br />
Antibiotic-Free Production <strong>of</strong> a Herpes Simplex Virus 2<br />
DNA Vaccine in a High Yield cGMP Process<br />
Jared Nelson, Ying Cai, Stephen Rodriguez, Neil Finlayson, James<br />
A. Williams, Aaron E. Carnes.<br />
264<br />
Characterization Technique for Drug and <strong>Gene</strong> Delivery<br />
Nanoparticles in Complex/Biological Media<br />
Sonja Capracotta, Duncan Grifiths, Phil Vincent, William Bernt,<br />
Patrick Hole.<br />
265<br />
Molecular and <strong>Cell</strong>ular Analyses <strong>of</strong> Platelet-Rich<br />
Plasma Preparations<br />
Roberta S. Stilhano, Priscila M. A. Denapoli, Sheila J. M. Ingham,<br />
Renne J. Abdalla, Sang W. Han.<br />
Late Breaking Abstracts I<br />
677<br />
RNA-guided Genome Editing Using CRISPR-Cas Systems<br />
Feng Zhang<br />
Thursday, May 16, 2013<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013<br />
105
Abstract Directory, Thursday, May 16, 2013<br />
Thursday, May 16, 2013<br />
Poster Session I, continued<br />
4:00 PM - 6:00 PM<br />
Room: Exhibit Hall C/D<br />
Late Breaking Abstracts I<br />
678<br />
Rescue <strong>of</strong> T-cell deiciency in Prkdc scid mice by<br />
transplantation <strong>of</strong> gene-repaired haematopoietic stem<br />
cells<br />
Hayder H Abdul-Razak, Céline Rocca, Steven J Howe, María<br />
Eugenia Alonso-Ferrero, Richard Gabriel, Cynthia C Bartholomae,<br />
Chih Hao V Gan, Marina I Garín, Francisco Javier Molina Estévez,<br />
Alison Roberts, Michael Blundell, Guillermo Güenechea, Jianbin<br />
Wang, Michael C Holmes, Philip D Gregory, Christine Kinnon,<br />
Christ<strong>of</strong> von Kalle, Manfred Schmidt, Juan Antonio Bueren, Adrian<br />
J Thrasher and Rafael J Yáñez-Muñoz<br />
679<br />
A microRNA-based system for selective gene expression<br />
in tumor-associated macrophages after adenovirus<br />
mediated gene transfer<br />
Michelle Giovani, Kamola Saydaminova, Hongjie Wang, Jonas<br />
Persson, Hua Cao, Erini Papapetrou, Il-Kyu Choi, Chae-Ok Yun,<br />
Andre Lieber<br />
680<br />
Overcoming problems in the generation <strong>of</strong> adenovirus<br />
vectors for genome editing in human hematopoietic<br />
stem cells<br />
Kamola Saydaminova, Xun Ye, Hongjie Wang, Akseli Hemminki,<br />
Eirini Papapetrou, Andre Lieber<br />
681<br />
Scalable contaminant-free production <strong>of</strong> adenoassociated<br />
virus vectors utilizing cytoplasmic helper<br />
carrier<br />
Biao Dong, Andrea R. Moore, Jihong Dai, Sean Roberts, Kirk Chu,<br />
Philipp Kapranov, Bernard Moss, Weidong Xiao<br />
682<br />
A novel RNA oligonucleotide improves liver function<br />
and inhibits liver carcinogenesis in vivo<br />
V. Reebye, P. Sætrom, P.J. Mintz, K.W. Huang, P. Swiderski, L. Peng,<br />
C. Liu, X.X. Liu, S. Jensen, D. Zacharoulis, N. Kostomitsopoulos, N.<br />
Kasahara, J.P. Nicholls, L.R. Jiao, M. Pai, M. Mizandari, T. Chikovani,<br />
M.M. Emara, A. Haoudi, D.A. Tomalia, J.J. Rossi, N.A. Habib<br />
683<br />
Pre-T cell receptor for improved expansion <strong>of</strong> T cells in<br />
the absence <strong>of</strong> TCR alpha<br />
Roman Galetto, Céline Lebuhotel, Laurent Poirot, Cécile Schiffer<br />
Mannioui, Julianne Smith, Andrew Scharenberg<br />
685<br />
Human Erythropoietin <strong>Gene</strong> Delivery for Cardiac<br />
Remodeling <strong>of</strong> Myocardial Infarction in Rats<br />
Youngsook Lee, Arlo N. McGinn, Curtis D. Olsen, Kihoon Nam,<br />
Minhyung Lee, Sug Kyun Shin, Sung Wan Kim<br />
686<br />
MGN1703 – structure <strong>of</strong> agonist determines cellular<br />
responses to TLR9 activation<br />
Kerstin Kapp, Christiane Kleuss, Manuel Schmidt, Matthias<br />
Schr<strong>of</strong>f, Burghardt Wittig<br />
687 Abstract Withdrawn from Presentation<br />
688<br />
Mini-LCC DNA Vector: The Gold Standard <strong>of</strong> <strong>Gene</strong><br />
Delivery in Molecular Medicine<br />
Naiseh Naissi, Roderick Slavcev<br />
689<br />
Overcoming physical barriers in oncolytic virotherapy<br />
using a serotype 3-based oncolytic adenovirus<br />
expressing relaxin<br />
Joung-Woo Choi, Il-Kyu Choi, Andre Lieber, Chae-Ok Yun<br />
690<br />
Design <strong>of</strong> oncolytic HSV1 that express cellular gene<br />
moieties that improve antiglioma eficacy and normal<br />
cell safety<br />
Hiroshi Nakashima, Tran T. Nguyen, William F. Goins, Ennio A.<br />
Chiocca<br />
691<br />
Polymeric Carriers for the Delivery <strong>of</strong> Neurotrophins to<br />
the Brain<br />
Lei Miao, Devika S-Manickam, Zhijian He,Yuhang Jiang, Alexander<br />
V. Kabanov<br />
692<br />
A Lentiviral System for Analysis <strong>of</strong> Signaling Pathways<br />
Involved in Reprogramming <strong>of</strong> Induced Pluripotent<br />
Stem <strong>Cell</strong>s<br />
Ashley Fritz, Sunnie Mao, David Schaffer<br />
693<br />
Pluronic block copolymers for gene delivery<br />
Vivek Mahajan, Alexander Kabanov<br />
694<br />
Gold nanoparticles assisted laser optoporation <strong>of</strong> cells:<br />
a novel physical method for eficient, safe and versatile<br />
gene delivery<br />
W. Ding, E. Bergeron, J. Baumgart, M-O. Lapointe, L. Humbert, J-J.<br />
Lebrun, M. Meunier<br />
684<br />
An inducible Caspase-9 delivery-system using<br />
Mesenchymal Stromal <strong>Cell</strong>s for lung cancer treatment<br />
Miki Ando, Valentina Hoyos, Shigeki Yagyu, Carlos Ramos, Antonio<br />
Di Stasi, Gianpietro Dotti, Lisa Bouchier-Hayes, Malcolm Brenner<br />
695<br />
A potential therapeutic role for survivin in mitigating<br />
the harmful effects <strong>of</strong> ionizing radiation<br />
G Metzger, E Choi, K Carruthers, E Kocak<br />
106<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013
Abstract Directory, Thursday, May 16, 2013<br />
Poster Session I, continued<br />
4:00 PM - 6:00 PM<br />
Room: Exhibit Hall C/D<br />
Late Breaking Abstracts I<br />
696<br />
HIV Latency: Activating Virus Expression with<br />
Combined <strong>Gene</strong>tic and Chemical Tools<br />
Suresh K. Arya, PhD, Agnes Holczbauer, MD<br />
697<br />
Sensitisation <strong>of</strong> neuroblastoma cells for TRAIL-induced<br />
apoptosis by downregulated XIAP<br />
Ahmet Cingoz, Yavuz Taga, Betul Catalgol<br />
698 Abstract Withdrawn from Presentation<br />
699<br />
Culture and cryopreservation <strong>of</strong> human epidermal<br />
melanocytes (MC) for preclinical trials<br />
Kim Jin Tac, Pak Seoung Hoon, Shin Back Soo, Han Su-Youne, Lee<br />
Hyeon Kyeong, Lee Hyun Woo, Kim Ji Hyang, Lee Ai-Young, Do<br />
Byung-Rok<br />
700<br />
Universal Stem <strong>Cell</strong> <strong>Gene</strong> <strong>Therapy</strong> Platform: Upgrading<br />
the Long-Term <strong>Gene</strong> <strong>Therapy</strong> Arm by shifting from<br />
Analogic to Breakthrough Digital Endonuclease-<br />
Boosted <strong>Gene</strong> Targeting and Focussing on Next<br />
<strong>Gene</strong>ration Ex Vivo Protocols<br />
Roger Bertolotti<br />
Thursday, May 16, 2013<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013<br />
107
Friday, May 17, 2013<br />
Abstract Directory, Friday, May 17, 2013<br />
Oral Abstract Session 320<br />
DNA Vectorology & <strong>Gene</strong> Targeting<br />
1:00 PM - 3:00 PM<br />
Room: Ballroom A<br />
266 1:00 PM<br />
<strong>Gene</strong> Targeting at Silent Loci with Adeno-Associated<br />
Virus Vectors<br />
Li B. Li, David R. Deyle, David W. Russell.<br />
267 1:15 PM<br />
Development <strong>of</strong> Novel Adeno-Associated Virus Variants<br />
Targeting HIV-Infected T <strong>Cell</strong>s<br />
Dawn P. Wooley, John Weinstein, Poornima K. L. Narayan, Priyanka<br />
Sharma, David V. Schaffer, Katherine J. D. A. Exc<strong>of</strong>fon.<br />
268 1:30 PM<br />
DNA Hairpins Stimulate Eficient <strong>Gene</strong> Correction Via<br />
Double Strand Break Repair<br />
Matthew L. Hirsch, Richard H. Samulski.<br />
269 1:45 PM<br />
A Transposon-Based Inducible System for Improved<br />
Expression In Vivo<br />
Joseph E. Doherty, Lauren E. Woodard, Adham S. Bear, Aaron<br />
Foster, Matthew H. Wilson.<br />
270 2:00 PM<br />
Minicircle DNA Is Superior to Plasmid DNA in Eliciting<br />
Antigen-Speciic CD8+ T <strong>Cell</strong> Responses and <strong>Gene</strong>rating<br />
Protective Immunity<br />
Nicole E. B. Skinner, Wynette M. Dietz, Sara E. Hamilton, Michelle<br />
D. Jund, Suzanne Heitfeld, Adam J. Litterman, Patrick Hwu, Zhi-<br />
Ying Chen, Andres M. Salazar, John R. Ohlfest, Bruce R. Blazar,<br />
Christopher A. Pennell, Mark J. Osborn.<br />
271 2:15 PM<br />
A Mini-Intronic Plasmid (MIP): A Novel Robust<br />
Transgene Expression Vector In Vivo and In Vitro<br />
Jiamiao Lu, Feijie Zhang, Mark A. Kay.<br />
272 2:30 PM<br />
Vertically-Integrated Translational Studies <strong>of</strong> PDX1 as<br />
a Therapeutic Target for Pancreatic Cancer Via a Novel<br />
Bifunctional RNAi Platform<br />
F. Charles Brunicardi, Juehua Yu, Shi-he Liu, Guisheng Zhou, Robbi<br />
Sanchez, Christopher M. Jay, Padmasini Kumar, Phillip B. Maples,<br />
Donald D. Rao, Neil Senzer, John Nemunaitis.<br />
273 2:45 PM<br />
Phase I Trial <strong>of</strong> Escalating Doses <strong>of</strong> SGT-53 Infusion in<br />
Combination with Docetaxel in Advanced Solid Tumor<br />
Patients<br />
John Nemunaitis, Neil Senzer, Cynthia Bedell, Derek Nemunaitis,<br />
Meghan Degele, Gerald Edelman, Minal Barve, Robert Nunan,<br />
Kathleen F. Pirollo, Antonina Rait, Esther H. Chang.<br />
Oral Abstract Session 321<br />
<strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> <strong>of</strong> Diabetes, Metabolic<br />
and <strong>Gene</strong>tic Diseases I<br />
1:00 PM - 3:00 PM<br />
Room: Ballroom B<br />
274 1:00 PM<br />
AAV Integrates Randomly into the Nuclear and<br />
Mitochondrial Genome after LPLD <strong>Gene</strong> <strong>Therapy</strong><br />
Christine Kaeppel, Stuart Beattie, Raffaele Fronza, Richard van<br />
Logtenstein, Florence Salmon, Sabine Schmidt, Stephan Wolf, Ali<br />
Nowrouzi, Hanno Glimm, Christ<strong>of</strong> von Kalle, Harald Petry, Daniel<br />
Gaudet, Manfred Schmidt.<br />
275 1:15 PM<br />
Ex Vivo Lentiviral <strong>Gene</strong> <strong>Therapy</strong> as a Cure for Murine<br />
Pompe Disease<br />
Merel Stok, Helen de Boer, Marshall W. Huston, Edwin H. Jacobs,<br />
Onno Roovers, Holger Jahr, Dirk J. Duncker, Trudi P. Visser,<br />
Ans T. van der Ploeg, Arnold J. J. Reuser, Nico P. van Til, Gerard<br />
Wagemaker.<br />
276 1:30 PM<br />
Pathogenesis <strong>of</strong> Growth Failure in Murine and Canine<br />
Glycogen Storage Disease-Ia and Partial Reversal <strong>of</strong><br />
Growth Failure Using Adeno-Associated Viral Vector<br />
<strong>Therapy</strong><br />
Elizabeth J. Brooks, Dianne Little, Ramamani Arumugam, Baodong<br />
Sun, Sarah Curtis, Amanda DeMaster, Michael Maranzano, Mark W.<br />
Jackson, Priya Kishnani, Michael S. Freemark, Dwight D. Koeberl.<br />
277 1:45 PM<br />
AAV-Mediated Direct Somatic <strong>Gene</strong>tic Correction <strong>of</strong><br />
Epidermolysis Bullosa<br />
Sandra Melo, Leszek Lisowski, Lisa Bashkirova, Mark A. Kay,<br />
Anthony E. Oro.<br />
278 2:00 PM<br />
Correction <strong>of</strong> Mitochondrial Cardiomyopathy<br />
Associated with Friedreich Ataxia in a Transgenic<br />
Model by <strong>Gene</strong> <strong>Therapy</strong><br />
Brahim Belbellaa, Morgane Perdomini, Laurent Monassier,<br />
Laurence Reutenauer, Nadia Messaddeq, Ronald G. Crystal, Patrick<br />
Aubourg, Hélène Puccio.<br />
279 2:15 PM<br />
Integration Proile <strong>of</strong> Lentiviral Vectors in <strong>Gene</strong><br />
<strong>Therapy</strong> for X-Adrenoleukodystrophy<br />
Cynthia C. Bartholomae, Nathalie Cartier, Salima Hacein-Bey-<br />
Abina, Ina Kutschera, Alain Fischer, Marina Cavazzana-Calvo,<br />
Patrick Aubourg, Manfred Schmidt, Christ<strong>of</strong> von Kalle.<br />
280 2:30 PM<br />
A Partial Deiciency Model <strong>of</strong> MUT Methylmalonic<br />
Acidemia (MMA) Displays Diet Inducible Disease and<br />
Sensitivity to Acetaminophen (APAP)<br />
Julien S. Senac, Vijay H. Aswani, Justin R. Sysol, Irini Manoli,<br />
Charles P. Venditti.<br />
108<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013
Abstract Directory, Friday, May 17, 2013<br />
Oral Abstract Session 321, continued<br />
<strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> <strong>of</strong> Diabetes, Metabolic<br />
and <strong>Gene</strong>tic Diseases I<br />
1:00 PM - 3:00 PM<br />
Room: Ballroom B<br />
281 2:45 PM<br />
AAV-Based over Expression <strong>of</strong> PEPCK-C Re-Patterns<br />
Mouse Muscle Energy Metabolism and Prevents High-<br />
Fat Diet Induced Obesity<br />
Daibang Nie, Sheri Wang, Ying Tang, Dong Wei, William Witt, Allan<br />
Zhao, Yifan Dai, Freddie Fu, Johnny Huard, Bing Wang.<br />
Oral Abstract Session 322<br />
Neurological <strong>Therapy</strong>: Neurodegeneration<br />
and Imaging<br />
1:00 PM - 3:00 PM<br />
Room: 150 ABC<br />
287 2:15 PM<br />
Fusion <strong>of</strong> Either Gap-43 or Tau to the Clostridial<br />
Tetanus Toxin Light Chain Improves Synaptic Inhibition<br />
in the Rat Spinal Cord Following Adenoviral Vector<br />
<strong>Gene</strong> Delivery<br />
Eleanor M. Donnelly, Chalonda R. Handy, Marie Kim, Jeremiah<br />
Huang, Thais Federici, Nicholas M. Boulis.<br />
288 2:30 PM<br />
MR-Guided Delivery <strong>of</strong> AAV2-hAADC into the Midbrain<br />
<strong>of</strong> Macaques Results in a Safe and Eficient Axonal<br />
Transport <strong>of</strong> AADC into the Striatum<br />
Kryst<strong>of</strong> S. Bankiewicz, Adrian P. Kells, Lluis Samaranch, Waldy San<br />
Sebastian, Nitasha Sharma, John Bringas, Phillip Pivirotto, John<br />
Forsayeth.<br />
289 2:45 PM<br />
Noninvasive, Quantitative PET Imaging <strong>of</strong> AAV-<br />
Mediated D2R80A Reporter <strong>Gene</strong> Expression in Live<br />
Animal Brains<br />
Sea Young Yoon, Datta Ponde, Harish Poptani, Jessica Bagel,<br />
Patricia A. O’Donnell, Charles H. Vite, John H. Wolfe.<br />
282 1:00 PM<br />
Silencing Mutant Htt by AAV-Mediated Expression <strong>of</strong><br />
RNAi Ameliorates Disease Manifestations in the YAC128<br />
Mouse Model <strong>of</strong> Huntington’s Disease<br />
Lisa M. Stanek, Lamya S. Shihabuddin, Seng H. Cheng.<br />
283 1:15 PM<br />
<strong>Gene</strong> Transfer <strong>of</strong> Human Apoe e2 Alleviate the<br />
Progression <strong>of</strong> Amyloid Deposition In Vivo<br />
Eloise Hudry, Jonathan Dashk<strong>of</strong>f, Allyson Lee, Shuko Takeda,<br />
Robert M. K<strong>of</strong>ie, Tadafumi Hashimoto, Maria Scheel, Tara Spires-<br />
Jones, Michal Arbel-Ornath, Rebecca A. Betensky, Beverly L.<br />
Davidson, Bradley T. Hyman.<br />
284 1:30 PM<br />
Vascular Delivery <strong>of</strong> AAV9-RNAi as a Potential <strong>Therapy</strong><br />
for Huntington’s Disease<br />
Brett Dufour, Catherine Smith, Randall Clark, Timothy Walker, Jodi<br />
McBride.<br />
285 1:45 PM<br />
Transcriptional Targeting <strong>of</strong> HSV Vector-Mediated<br />
Transgene Expression to Speciic Sensory Neuron<br />
Subtypes<br />
Mark F. Doyal, Mingdi Zhang, George Huang, Justus Cohen, Nicole<br />
Scheff, Michael Gold, William F. Goins, Joseph C. Glorioso.<br />
Oral Abstract Session 323<br />
Musculo-skeletal <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> II<br />
1:00 PM - 3:00 PM<br />
Room: 150 DEFG<br />
290 1:00 PM<br />
<strong>Gene</strong> <strong>Therapy</strong> Combined with NF-kappaB Inhibition for<br />
Duchenne Muscular Dystrophy<br />
Ying Tang, Robert Kang, Kara Imbrogno, Arvydas Usas, Qing Yang,<br />
Aiping Lu, Freddie Fu, Johnny Huard, Bing Wang.<br />
291 1:15 PM<br />
AAV Mediated Overexpression <strong>of</strong> Human a7 Integrin<br />
Leads to Histological and Functional Improvement in<br />
Dystrophic Mice<br />
Kristin N. Heller, Chrystal L. Montgomery, Kimberly M. Shontz,<br />
Paul M. L. Janssen, K. Reed Clark, Jerry R. Mendell, Louise R.<br />
Rodino-Klapac.<br />
292 1:30 PM<br />
<strong>Gene</strong>tic Correction <strong>of</strong> Duchenne Muscular Dystrophy<br />
Using Zinc Finger Nucleases<br />
David G. Ousterout, Pablo Perez-Pinera, Pratiksha I. Thakore,<br />
Matthew T. Brown, Charles A. Gersbach.<br />
Friday, May 17, 2013<br />
286 2:00 PM<br />
Mechanisms <strong>of</strong> Caspase-1 Mediated Schwannoma<br />
Regression<br />
Mehran Taherian, Shilpa Prabhakar, Giulia Fulci, Miguel Sena-<br />
Esteves, Xandra O. Breakeield, Gary J. Brenner.<br />
293 1:45 PM<br />
New TALENs To Correct the Reading Frame <strong>of</strong> Exon 54<br />
<strong>of</strong> the Dystrophin <strong>Gene</strong><br />
Joel Rousseau, Jean-Paul Iyombe, Charles A. Gersbach, Jacques P.<br />
Tremblay.<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013<br />
109
Friday, May 17, 2013<br />
Abstract Directory, Friday, May 17, 2013<br />
Oral Abstract Session 323, continued<br />
Musculo-skeletal <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> II<br />
1:00 PM - 3:00 PM<br />
Room: 150 DEFG<br />
294 2:00 PM<br />
Restriction <strong>of</strong> Calpain3 Expression to the Skeletal<br />
Muscle Prevents Cardiac Toxicity and Corrects<br />
Pathology in a Murine Model <strong>of</strong> Limb-Girdle Muscular<br />
Dystrophy<br />
Carinne Roudaut, Florence Le Roy, Laurence Suel, Isabelle Richard.<br />
295 2:15 PM<br />
RNAi <strong>Therapy</strong> for Limb Girdle Muscular Dystrophy<br />
Type 1A<br />
Jian Liu, Lindsay Wallace, Sara Garwick-Coppens, Carol Davis,<br />
Chady Hakim, Susan Guckes, Michael Hauser, Jerry Mendell, Susan<br />
Brooks, Scott Harper.<br />
296 2:30 PM<br />
Expression <strong>of</strong> FKRP Consequently Restored Functional<br />
Glycosylation <strong>of</strong> a-DG in the Skeletal and Cardiac<br />
Muscles in FKRP Mutant Mice<br />
Qi Long Lu, Lei Xu, Pei Juan Lu, Chi-Hsien Wang, Elizabeth<br />
Keramaris, Chunping Qiao, Bin Xiao, Derek J. Blake, Xiao Xiao.<br />
297 2:45 PM<br />
Minimally Invasive Implantation <strong>of</strong> Autologous<br />
Chondrocytes Transduced with rAAV5-IGF-I Improves<br />
Longterm Cartilage Repair in Full-Thickness Chondral<br />
Defects in the Equine Model<br />
Kyla Ortved, Alan J. Nixon.<br />
Oral Abstract Session 324<br />
Cancer-Targeted <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong><br />
1:00 PM - 3:00 PM<br />
Room: Ballroom C<br />
298 1:00 PM<br />
Safety and Correlates <strong>of</strong> Clinical Response in an Early<br />
Phase Clinical Trial in Multiple Myeloma Patients<br />
Post Auto-SCT and Adoptive Immunotherapy with<br />
Engineered T <strong>Cell</strong>s Expressing an HLA-A2 Restricted<br />
Afinity-Enhanced TCR for LAGE-1 and NY-ESO-1<br />
Bruce L. Levine, Aaron P. Rapoport, Edward A. Stadtmauer, Dan<br />
T. Vogl, Brendan Weiss, Gwendolyn K. Binder-Scholl, Dominic P.<br />
Smethurst, Jeffrey Finkelstein, Irina Kulikovskaya, Minnal Gupta,<br />
Erica Suppa, Joanna E. Brewer, Alan D. Bennett, Andrew B. Gerry,<br />
Nick J. Pumphrey, Helen K. Tayton-Martin, Daniel Williams,<br />
Zhaohui Zheng, Ashraf Z. Badros, Saul Yanovich, Gorgon Akpek,<br />
Michael Kalos, Bent K. Jakobsen, Carl H. June.<br />
299 Abstract Moved to Oral Abstract Session 230<br />
648 1:15 PM<br />
Neural Stem <strong>Cell</strong>s Expressing a Modiied Human<br />
Carboxylesterase Potentiate Anti-Cancer Activity <strong>of</strong><br />
Irinotecan in Murine Glioma Model<br />
Karen S. Aboody, Lucy Y. Ghoda, Margarita Gutova, Megan<br />
Gilchrist, Christine Brown, Vazgen Kankaldyyan, Elizabth Garcia,<br />
Lauren H<strong>of</strong>fman, Rex A. Moats, Phil Potter, Timothy W. Synold,<br />
Michael Barish.<br />
300 1:30 PM<br />
Virus-Speciic Cytotoxic T Lymphocytes (CTLs)<br />
Redirected with a Chimeric Antigen Receptor (CAR)<br />
and Engineered To Respond to IL7 Resist Regulatory T<br />
<strong>Cell</strong> (Treg) Inhibition<br />
Serena K. Perna, Daria Pagliara, Aruna Mahendravada, Hao Liu,<br />
Malcolm K. Brenner, Barbara Savoldo, Gianpietro Dotti.<br />
301 1:45 PM<br />
Engineering Hematopoiesis for Tumor-Targeted<br />
Delivery <strong>of</strong> IFNa by Tumor-Iniltrating Macrophages<br />
Achieves Safe and Effective Inhibition <strong>of</strong> Breast<br />
Cancer and Its Metastases in Both Murine and Human<br />
Hematochimeric Models<br />
Giulia Escobar, Davide Moi, Anna Ranghetti, Mario L. Squadrito,<br />
Michele De Palma, Roberta Mazzieri, Luigi Naldini.<br />
302 2:00 PM<br />
Myeloid Suppressor <strong>Cell</strong> Blockade Potentiates Anti-<br />
Tumor Responses <strong>of</strong> Adoptive T <strong>Cell</strong> Transfer <strong>Therapy</strong><br />
Steven Mok, Christopher Tsui, Thinle Chodon, Antoni Ribas,<br />
Richard C. Koya.<br />
303 2:15 PM<br />
Afinity-Enhanced Epithelial Junction Openers Improve<br />
Cancer Chemotherapy<br />
Roma Yumul, Hua Cao, Maximillian Richter, Julie Gralow, Andre<br />
Lieber.<br />
304 2:30 PM<br />
Glioblastoma Transfer miRNAs into Microglial <strong>Cell</strong>s<br />
through Extracellular Vesicles<br />
Erik R. Abels, Kristan E. van der Vos, Johan Skog, Suzanne E.<br />
Hickman, Joseph El Khoury, Xandra O. Breakeield.<br />
305 2:45 PM<br />
ab-T <strong>Cell</strong> Receptor-Based <strong>Gene</strong> <strong>Therapy</strong> Targeting<br />
the Common Tumor-Antigen Survivin for <strong>Therapy</strong> <strong>of</strong><br />
Hematological Malignancies<br />
Caroline Arber, Harshal Abhyankar, Helen E. Heslop, Gianpietro<br />
Dotti, Barbara Savoldo.<br />
110<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013
Abstract Directory, Friday, May 17, 2013<br />
Oral Abstract Session 325<br />
<strong>Gene</strong> <strong>Therapy</strong> for Hematologic Diseases: From<br />
Models to Clinical Results<br />
1:00 PM - 3:00 PM<br />
Room: 151 ABCG<br />
306 1:00 PM<br />
Lentiviral Vector-Based <strong>Gene</strong> <strong>Therapy</strong> for Wiskott-<br />
Aldrich Syndrome: Preliminary Results from the<br />
French Center<br />
Salima Hacein-Bey-Abina, Johanna Blondeau, Laure Caccavelli,<br />
Sabine Charrier, Capucine Picard, Liliane Dal-Cortivo, Guillem<br />
Cross, Stephane Blanche, Frederic Bushman, Nirav Malani, Alain<br />
Fischer, Adrian Thrasher, Anne Galy, Marina Cavazzana-Calvo.<br />
307 1:15 PM<br />
Phase II Clinical <strong>Gene</strong> <strong>Therapy</strong> Trial for Adenosine<br />
Deaminase-Deicient Severe Combined Immune<br />
Deiciency (ADA-SCID)<br />
Kit L. Shaw, Robert Sokolic, Suparna Mishra, Satiro de Oliveira,<br />
Christopher Silvin, Geiger-Schredelseker Sabine, Brown<br />
Berkley, Garabedian Elizabeth, Monika Smogorzewska, Denise<br />
A. Carbonaro, Pei-yu Fu, Alejandra Davila, Jayashree Jagadeesh,<br />
Michael Hershield, Alan Wayne, Neena Kapoor, Theodore Moore,<br />
Fabio Candotti, Donald B. Kohn.<br />
311 2:15 PM<br />
Expansion <strong>of</strong> <strong>Gene</strong>-Modiied T <strong>Cell</strong>s Following<br />
Direct Intravenous Injection <strong>of</strong> Therapeutic Foamy<br />
Virus Vector in a Canine Model <strong>of</strong> Severe Combined<br />
Immunodeiciency<br />
Christopher R. Burtner, Brian C. Beard, Douglas R. Kennedy, Troy<br />
R. Torgerson, Andrew M. Scharenberg, David J. Rawlings, Grant D.<br />
Trobridge, Peter J. Felsburg, Hans-Peter Kiem.<br />
312 2:30 PM<br />
Adeno-Associated Virus-Mediated Expression <strong>of</strong> a<br />
C-Terminal Truncated ADAMTS13 Variant Prevents<br />
Shigatoxin-Induced Thrombotic Thrombocytopenic<br />
Purpura<br />
Sheng-Yu Jin, Juan Xiao, Jialing Bao, Shangzhen Zhou, Fraser J.<br />
Wright, X. Long Zheng.<br />
313 2:45 PM<br />
Self-Inactivating Lentiviral Vectors for Correction <strong>of</strong><br />
Rag1 Severe Combined Immunodeiciency<br />
Karin Pike-Overzet, Anna-Sophia Wiekmeijer, Miranda R. M. Baert,<br />
Chantal Lagresle-Peyrou, Axel Schambach, Fang Zhang, Rob C.<br />
Hoeben, Salima Hacein-Bey-Abina, Arjen C. Lankester, Robbert G.<br />
M. Bredius, Gertjan J. A. Driessen, Adrian J. Thrasher, Christopher<br />
Baum, Marina Cavazzana-Calvo, Jacques J. M. van Dongen, Frank J.<br />
T. Staal.<br />
308 1:30 PM<br />
Lentiviral <strong>Gene</strong> Transfer for Treatment <strong>of</strong> Children<br />
>2 Years Old with X-Linked Severe Combined<br />
Immunodeiciency<br />
Suk See De Ravin, Uimook Choi, Narda Theobald, Janet Lee,<br />
Hongmei Wang, Xiaolin Wu, Geraldine O’Connor, Daniel W.<br />
McVicar, Elizabeth Kang, John T. Gray, Brian P. Sorrentino, Harry L.<br />
Malech.<br />
309 1:45 PM<br />
Integration Site Analysis in a Clinical Trial <strong>of</strong> Lentiviral<br />
Vector Based Hematopoietic Stem <strong>Cell</strong> <strong>Gene</strong> <strong>Therapy</strong><br />
for Metachromatic Leukodystrophy. An 18 Months<br />
Follow-Up<br />
Eugenio Montini, Alessandra Bifi, Andrea Calabria, Luca Biasco,<br />
Martina Cesani, Fabrizio Benedicenti, Tiziana Plati, Simone Leo,<br />
Victor Neduva, Gianluigi Zanetti, Alessandro Aiuti, David Dow,<br />
Christ<strong>of</strong> von Kalle, Manfred Schmidt, Maria Sessa, Luigi Naldini.<br />
310 2:00 PM<br />
Comparison <strong>of</strong> Integration Site Proiles between<br />
SCID-X1 <strong>Gene</strong> <strong>Therapy</strong> Trials Using Gammaretroviral<br />
Vectors with Intact or Deleted (SIN) LTRs<br />
Salima Hacein-Bey-Abina, Laure Caccavelli, Nirav Malani, Charles<br />
Berry, Frances Male, Frederic Bushman, Alain Fischer, Marina<br />
Cavazzana-Calvo.<br />
Oral Abstract Session 326<br />
<strong>Gene</strong> Regulation and Animal Models<br />
1:00 PM - 3:00 PM<br />
Room: Ballroom D<br />
314 1:00 PM<br />
Pristimerin Enhances Recombinant Adeno-Associated<br />
Virus Serotype 2 Vector-Mediated Transgene<br />
Expression Both In Vitro and In Vivo<br />
Lina Wang, Yuan Wang, George V. Aslanidi, Arun Srivastava,<br />
Changquan Ling, Chen Ling.<br />
315 1:15 PM<br />
Transcriptional and Post-Transcriptional Regulation To<br />
Overcome Immunological Responses to <strong>Gene</strong> <strong>Therapy</strong><br />
for Hemophilia A<br />
Stefania E. Cannizzo, Simone Merlin, Antonia Follenzi.<br />
316 1:30 PM<br />
Selective Expansion <strong>of</strong> Transduced Hepatocytes: A<br />
Mechanism for Modulating Transgene Levels In Vivo<br />
Sean C. Nygaard, Annelise Haft, Milton Finegold, Markus Grompe.<br />
317 1:45 PM<br />
Multiplex Bi-Functional Short-Hairpin RNA (shRNA)<br />
Targeting Single-Nucleotide KRAS Mutations in<br />
Pancreatic Cancer<br />
Donald D. Rao, Zhaohui Wang, Padmasini Kumar, Neil Senzer, John<br />
Nemnaitis.<br />
Friday, May 17, 2013<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013<br />
111
Abstract Directory, Friday, May 17, 2013<br />
Oral Abstract Session 326, continued<br />
<strong>Gene</strong> Regulation and Animal Models<br />
1:00 PM - 3:00 PM<br />
Room: Ballroom D<br />
318 2:00 PM<br />
Engineering an XID-Site Speciic I-AniI Homing<br />
Endonuclease through Combination <strong>of</strong> Rational Design<br />
and Directed Evolution<br />
Yupeng Wang, Jordan Jarjour, Sandrine Boissel, Summer Thyme,<br />
Iram Khan, Joseph Pangallo, David Baker, Barry Stoddard, Andrew<br />
M. Scharenberg, David J. Rawlings.<br />
319 2:15 PM<br />
LMO2 Perturbs Human T <strong>Cell</strong> Development In Vivo by<br />
Two Different Mechanisms<br />
Anna-Sophia Wiekmeijer, Karin Pike-Overzet, Martijn H. Brugman,<br />
Willem E. Fibbe, Frank J. T. Staal.<br />
325 5:10 PM<br />
Targeting B <strong>Cell</strong> Precursor Acute Lymphoblastic<br />
Leukemia (ALL) with Chimeric Antigen Receptors<br />
(CARs) Speciic for CD19 or CD22<br />
Rimas J. Orentas, Waleed Haso, Daniel W. Lee, Terry J. Fry, Alan S.<br />
Wayne, Crystal L. Mackall.<br />
Poster Session II<br />
5:30 PM - 7:30 PM<br />
Room: Exhibit Hall C/D<br />
AAV Vectors II<br />
326<br />
Retro-Orbital Injection <strong>of</strong> RAAVs to Neonatal Mice<br />
Dramtically Enhances <strong>Gene</strong> Delivery to the CNS<br />
Jia Li, Li Zhong, Shaoyong Li, Mengxin Li, Su Qin, Ran He, Flotte R.<br />
Terence, Guangping Gao.<br />
Friday, May 17, 2013<br />
320 2:30 PM<br />
Tightly Regulated Doxycycline (Dox)-Inducible<br />
Lentiviral Vectors for Human Myeloprotective <strong>Gene</strong><br />
<strong>Therapy</strong>: In Vitro and CD34+Xenotransplant Studies<br />
Nico Lachmann, Roman Hilje, Sebastian Brennig, Julia Dahlmann,<br />
Niels Heinz, Ina Gruh, Bernd Schiedlmeier, Christopher Baum,<br />
Thomas Moritz.<br />
321 2:45 PM<br />
Transcription Factor Activity Analysis Using a<br />
Functional Multiplex Gaussia Luciferase-Based<br />
Reporter Assay<br />
Sjoerd van Rijn, Jonas Nilsson, David P. Noske, W. Peter Vandertop,<br />
Bakhos A. Tannous, Thomas Würdinger.<br />
Presidential Symposium 330<br />
Presidential Symposium<br />
3:15 PM - 5:30 PM<br />
Room: Ballroom E-J<br />
322 4:10 PM<br />
Kinetics and Epigenetics <strong>of</strong> Retroviral Silencing in<br />
Mouse Embryonic Stem <strong>Cell</strong>s Deined by Deletion <strong>of</strong> a<br />
D4Z4 Barrier Element<br />
Sylvie Rival-Gervier, Mandy Lo, Shahryar Khattak, Peter Pasceri,<br />
Matthew Lorincz, James Ellis.<br />
323 4:30 PM<br />
Liver-Directed, Insulin-Based <strong>Gene</strong> <strong>Therapy</strong> Protects<br />
NOD Mice from Diabetes<br />
Mahzad Akbarpour, Kevin S. Goudy, Andrea Annoni, Francesca<br />
Sanvito, Luigi Naldini, Maria Grazia Roncarolo.<br />
324 4:50 PM<br />
On-Target Cleavage and Off-Target Activity <strong>of</strong> TALEN<br />
‘Nickases’<br />
Thomas J. Cradick, Christopher J. Antico, Eli J. Fine, Cory R.<br />
Lindsay, Eric Kildebeck, David B. Roth, Matthew H. Porteus.<br />
327<br />
AAV2H22, a Vector Selected by Directed Evolution,<br />
Transduces More Eficiently from the Basolateral<br />
Surface Despite Apical Selective Pressure<br />
Benjamin R. Steines, David D. Dickey, Katherine J. Exc<strong>of</strong>fon, David<br />
V. Schaffer, Joseph Zabner.<br />
328<br />
High-Eficiency Transduction <strong>of</strong> Primary Human<br />
Hematopoietic Stem <strong>Cell</strong>s and Erythroid Lineage-<br />
Restricted Expression by Optimized scAAV6 Serotype<br />
Vectors In Vitro and in a Murine Xenograft Model In<br />
Vivo<br />
Liujiang Song, Xiaomiao Li, Giridhara R. Jayandharan, Yuan Wang,<br />
George V. Aslanidi, Chen Ling, Li Zhong, Guangping Gao, Mervin C.<br />
Yoder, Changquan Ling, Mengqun Tan, Arun Srivastava.<br />
329 Abstract Withdrawn from Presentation<br />
330<br />
Comparing Dual AAV Vector Strategy Eficiencies for<br />
<strong>Gene</strong> <strong>Therapy</strong> In Vitro and In Vivo<br />
Heikki T. Turunen, María Victoria Luna Velez, Rajani Shelke, Ru<br />
Xiao, Jean Bennett, Luk H. Vandenberghe.<br />
331<br />
Regulation <strong>of</strong> Host <strong>Cell</strong>ular DNA Binding Proteins<br />
Augment the <strong>Gene</strong> Transfer Eficiency <strong>of</strong> Adeno-<br />
Associated Virus (AAV) Vectors<br />
Akshaya Krishnagopal, Sangeetha Hareendran, Dwaipayan Sen,<br />
Arun Srivastava, Alok Srivastava, Giridhara R Jayandharan.<br />
332<br />
Differences in AAV Serotype Dependent Distribution<br />
and Tropism Exist within the CNS <strong>of</strong> Wild Type and<br />
Mutant Mice in the MPSIIIB Mouse Model<br />
Janine A. Gilkes, Matthew D. Bloom, Andrew R. Kolarich, Coy D.<br />
Heldermon.<br />
112<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013
Abstract Directory, Friday, May 17, 2013<br />
Poster Session II, continued<br />
5:30 PM - 7:30 PM<br />
Room: Exhibit Hall C/D<br />
AAV Vectors II<br />
333<br />
High-Eficiency Transduction <strong>of</strong> Primary Human<br />
Monocyte-Derived Dendritic <strong>Cell</strong>s by Recombinant<br />
AAV6 Vectors Containing Mutations in Surface-Exposed<br />
Serine and Threonine Residues<br />
George Aslanidi, Angela Rivers, Luis Ortiz, Jhell Pandya, Chen Ling,<br />
Kim Van Vliet, Mavis Agbandje-McKenna, Arun Srivastava.<br />
334<br />
Optimization <strong>of</strong> the Capsid <strong>of</strong> Recombinant Adeno-<br />
Associated Virus 2 Vectors: The <strong>Final</strong> Threshold<br />
George Aslanidi, Angela Rivers, Luis Ortiz, Chen Ling, Kim Van<br />
Vliet, Lakshmanan Govindasamy, Mavis Agbandje-McKenna, Arun<br />
Srivastava.<br />
335<br />
Directed Evolution <strong>of</strong> AAV Capsid for Targeted Cancer<br />
<strong>Therapy</strong><br />
Damien Marsic, Yuan Lu, Chunxia Cao, Dane Phelan, Lakshmanan<br />
Govindasamy, Frederic J. Kaye, Mavis Agbandje-McKenna, Steve<br />
Ghivizzani, Sergei Zolotukhin.<br />
336<br />
Structures <strong>of</strong> the Retargeted AAV-DJ Vector in the<br />
Presence and Not <strong>of</strong> Receptor-Analog Sucrose<br />
Octasulfate<br />
Qing Xie, Nancy Meyer, Thomas F. Lerch, Dustin McCraw, Michael<br />
Spillman, Scott Stagg, Michael S. Chapman.<br />
337<br />
Improved AAV1 and AAV9 Vectors for <strong>Gene</strong> Delivery<br />
Balaji Balakrishnan, Akshaya Krishnagopal, Dwaipayan Sen, Alok<br />
Srivastava, Giridhara r Jayandharan.<br />
338<br />
Plasmid Integration within AAVS1 in AAV Producer <strong>Cell</strong><br />
Lines<br />
Amy Frederick, Yuxia Luo, John Martin, Jeffery Ardinger, Donna<br />
Armentano, Sam Wadsworth, Karen Vincent.<br />
339<br />
Short Hairpin RNA Delivered by Adeno-Associated<br />
Virus Vector Eficiently Reduced Target <strong>Gene</strong><br />
Expression in Mouse Primary Cortical Neurons<br />
Yun Lin, Holly Carlisle, Tom Wolfe, Danny Ortuno, Trish Amarante,<br />
Huiren Zhao, Steve Wood, Cherylene Plewa, Mike Wyszynski, Paul<br />
Kassner, Ki Jeong Lee.<br />
340<br />
AAV Vectors Puriied from Culture Medium with a<br />
Simple and Rapid Protocol Transduce Eficiently<br />
Murine Liver, Muscle and Retina<br />
Monica Doria, Antonella Ferrara, Alberto Auricchio.<br />
DNA Vectorology & <strong>Gene</strong> Targeting I<br />
341<br />
Minimally Invasive Electroporation <strong>of</strong> Synthetic DNA<br />
Vaccines for the Induction CTL and Functional Humoral<br />
Immune Responses Against HIV<br />
Megan Wise, Karuppiah Muthumani, Kate E. Broderick, Amir S.<br />
Khan, Janess Mendoza, Maria Yang, Jian Yan, Natalie Hutnick,<br />
Niranjan Y. Sardesai, David B. Weiner.<br />
342<br />
An E3-14.7K Peptide That Promotes Microtubules-<br />
Mediated Transport <strong>of</strong> Plasmid DNA Increases<br />
Polyplexes Transfection Eficiency<br />
Lucie Pigeon, Cristine Gonçalves, David Gosset, Chantal Pichon,<br />
Patrick Midoux.<br />
343<br />
Benchtop DNA Synthesizer: Oligo-Templated<br />
Polymerization (OTP)<br />
Adi Barzel, Mark A. Kay.<br />
344<br />
Nanoparticle-Mediated <strong>Gene</strong> Delivery for Rhodopsin-<br />
Associated Retinitis Pigmentosa<br />
Zongchao Han, Rasha Makkia, Junjing Guo, Miles J. Merwin, Mark J.<br />
Cooper, Muna I. Naash.<br />
345<br />
Hybrid Flp-TALE and Cre-TALE Recombinases with<br />
Designed Target Speciicity as Genome Engineering<br />
Tools<br />
Feng Li, Riddhi Shah, Eugenia Voziyanova, Yuri Voziyanov.<br />
346<br />
Permanent <strong>Gene</strong>tic Modiication <strong>of</strong> Dividing <strong>Cell</strong>s Using<br />
Episomally Maintained S/MAR DNA Vectors and the<br />
Correction <strong>of</strong> a Cancer Phenotype in Renal Tumour<br />
<strong>Cell</strong>s<br />
Suet Ping Wong, Richard P. Harbottle.<br />
347<br />
Collagen VII <strong>Gene</strong> Delivery Via Sleeping Beauty<br />
Transposon in COL7A1-Deicient Keratinocytes from<br />
Epidermolysis Bullosa Patients<br />
Maria Carmela Latella, Fabienne Cocchiarella, Giandomenico<br />
Turchiano, Zsuzsanna Izsvak, Zoltan Ivics, Manuel Gonçalves,<br />
Fulvio Mavilio, Fernando Larcher, Alessandra Recchia.<br />
348<br />
DNAJB4, a Heat Shock Rotein hsp40 Homolog, Activates<br />
Dendritic <strong>Cell</strong>s and Functions as a Molecular Adjuvant<br />
When Incorporated into Cancer DNA Vaccine<br />
Hsing-Yu Chen, Jeremy. J.W. Chen, Chi-Chen Lin.<br />
349<br />
Surface-Stabilized Calcium Phosphate Nano-Crystals<br />
for Eficient <strong>Gene</strong> Delivery<br />
Min Sang Lee, Kyuri Lee, Nak Won Kim, Ji Hoon Jeong.<br />
Friday, May 17, 2013<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013<br />
113
Abstract Directory, Friday, May 17, 2013<br />
Friday, May 17, 2013<br />
Poster Session II, continued<br />
5:30 PM - 7:30 PM<br />
Room: Exhibit Hall C/D<br />
DNA Vectorology & <strong>Gene</strong> Targeting I<br />
350<br />
Polymeric Micellar Nanocarriers for <strong>Gene</strong> and<br />
Oligonucleotide Delivery<br />
Kazunori Kataoka.<br />
351<br />
Methods <strong>of</strong> Facilitating Patient Participation in DNA<br />
Based Experimental Therapeutic Phase I Cancer<br />
Studies<br />
Wanda Strange, Cynthia Bedell, Shannon Cagnina, Jeanne Jones,<br />
Patricia Brown, Gladice Wallraven, John Nemunaitis.<br />
352<br />
Manufacturing Developments for Scale-Up <strong>of</strong><br />
Autologous Phase II Ovarian Tumor Tissue FANG<br />
Vaccine<br />
Nicolas Taquet, John Nemunaitis, Phillip B. Maples.<br />
<strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> <strong>of</strong> Diabetes, Metabolic<br />
and <strong>Gene</strong>tic Diseases I<br />
353<br />
Ultrasound-Guided Hepatic Injections <strong>of</strong> HDAd Vector<br />
Are More Eficient and Safer Than Systemic Intravenous<br />
Injections for Liver-Directed <strong>Gene</strong> <strong>Therapy</strong> <strong>of</strong> Crigler-<br />
Najjar Syndrome Type I<br />
Nunzia Pastore, Edoardo Nusco, Pasquale Piccolo, Sigismondo<br />
Castaldo, Francesco Vetrini, Donna Palmer, Libor Vitek, Philip Ng,<br />
Nicola Brunetti-Pierri.<br />
354<br />
<strong>Gene</strong> <strong>Therapy</strong> for Liver Diseases Using Non-Viral<br />
Minicircle-DNA Vector<br />
Hiu Man Viecelli, Richard P. Harbottle, Suet Ping Wong, Marinne<br />
Chuah, Thierry VandenDriessche, Cary O. Harding, Beat Thony.<br />
355<br />
Hepatic Delivery <strong>of</strong> <strong>Gene</strong>s Encoding Therapeutic<br />
Proteins or Mirnas Using Hyperactive Transposons: A<br />
Platform Technology for <strong>Gene</strong> <strong>Therapy</strong> and Functional<br />
Genomics In Vivo<br />
Mario Di Matteo, Pieter De Bleser, Ermira Samara-Kuko, Marinee<br />
Chuah, Thierry VandenDriessche.<br />
356<br />
Liver Progenitor <strong>Cell</strong>s Isolated from Adult Mice Fed a<br />
Choline-Deicient, Ethionine-Supplemented Diet Can<br />
Engraft, Expand and Mature in the Injured Liver <strong>of</strong> the<br />
FRG Mouse Model<br />
Gustavo de Alencastro, Grant J. Logan, Allison P. Dane, Sophia H. Y.<br />
Liao, George C. T. Yeoh, Ian E. Alexander.<br />
357<br />
Long-Term Phenotypic Correction <strong>of</strong> Hemophilia A by<br />
Non-Viral PiggyBac Transposon Vector<br />
Hideto Matsui, Mitsuhiko Sugimoto, Midori Shima, Naoko<br />
Fujimoto, Akitsu Hotta.<br />
358<br />
Long-Term Secretion <strong>of</strong> Arylsulfatase A without<br />
Antibody Formation after AAV Mediated Neonatal <strong>Gene</strong><br />
Transfer into MLD Model Mice<br />
Noriko Miyake, Koichi Miyake, Motoko Yamamoto, Takashi<br />
Shimada.<br />
359<br />
In Vitro and In Vivo Characterization <strong>of</strong> an Engineered<br />
Arylsulfatase B for <strong>Therapy</strong> <strong>of</strong> Mucopolysaccharidosis<br />
VI<br />
Rita Ferla, Pamela Claudiani, Gabriella Cotugno, Aaron Petrey,<br />
Richard Steet, Alberto Auricchio.<br />
360<br />
<strong>Gene</strong> <strong>Therapy</strong> for MPS VI Is Effective in Cats without<br />
Pre-Existing Immunity to AAV8<br />
Rita Ferla, Thomas O’Malley, Roberto Calcedo, Patricia O’Donnell,<br />
Ping Wang, Gabriella Cotugno, Pamela Claudiani, James M. Wilson,<br />
Mark Haskins, Alberto Auricchio.<br />
361<br />
Correlation <strong>of</strong> <strong>Gene</strong> Transfer and Transduction to<br />
Chloride Current Defect from rAAV2/5 Vector Derived<br />
Transgene DCFTR in Murine CF Nasal Airway<br />
Gang Shi, Kelvin D. MacDonald, Christian Mueller, Terence R.<br />
Flotte, Anne C. Fischer.<br />
362<br />
Helper-Dependent Adenoviral Vectors for Liver-<br />
Directed <strong>Gene</strong> <strong>Therapy</strong> <strong>of</strong> Primary Hyperoxaluria Type<br />
1<br />
Raffaele Castello, Stefania D”Aria, Roberta Borzone, Pasquale<br />
Piccolo, Patrizia Annunziata, Nicola Brunetti-Pierri.<br />
363<br />
Fetal <strong>Gene</strong> <strong>Therapy</strong> at 0.4G with AAV-hFIX or AAV-hFX<br />
Mediates Therapeutic Transgene Expression Despite<br />
Growth and a Positive Immune Response in a Non-<br />
Human Primate Model<br />
Citra Mattar, Cecilia Rosales, Rowell Fragata, Nuryanti Johana,<br />
Simon Waddington, Arijit Biswas, Mahesh Choolani, Amit<br />
Nathwani, Jerry Chan.<br />
364<br />
AAV Vector-Mediated Iduronidase <strong>Gene</strong> Delivery in<br />
a Murine Model <strong>of</strong> Mucopolysaccharidosis Type I:<br />
Comparing Different Routes <strong>of</strong> Delivery to the CNS<br />
Lalitha Belur, Zhenhong Nan, Kelly M. Podetz-Pedersen, Carolyn<br />
A. Fairbanks, Leah R. Hanson, William H. Frey, Karen Kozarsky,<br />
Walter C. Low, R. Scott McIvor.<br />
114<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013
Abstract Directory, Friday, May 17, 2013<br />
Poster Session II, continued<br />
5:30 PM - 7:30 PM<br />
Room: Exhibit Hall C/D<br />
<strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> <strong>of</strong> Diabetes, Metabolic<br />
and <strong>Gene</strong>tic Diseases I<br />
365<br />
Enzyme Replacement <strong>Therapy</strong> Enhanced a Therapeutic<br />
Eficacy <strong>of</strong> Bone Marrow Transplantation in MPS II<br />
Mouse<br />
Kazumasa Akiyama, Yohta Shimada, Takashi Higuchi, Sayoko<br />
Iizuka, Makoto Otsu, Hiromitsu Nakauchi, Jillian R. Brown, Brett<br />
E. Crawford, Takahiro Fukuda, Hiroshi Kobayashi, Yoshikatsu Eto,<br />
Hiroyuki Ida, Toya Ohashi.<br />
Cardiovascular <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong><br />
366<br />
Ex-Vivo Transduced Monocytes as <strong>Gene</strong> Delivery<br />
Vehicles to Restenotic Arteries<br />
Ilia Fishbein, Richard F. Adamo, Jeanne M. Connolly, Shira Redlich,<br />
Katherine A. Clark, Ricardo A. Corrales, Michael Chorny, Scott P.<br />
Forbes, Robert J. Levy.<br />
367<br />
AAV9-Mediated miR-499 Expression Is Suficient To<br />
Induce Progressive Heart Failure in Mice<br />
Ru-Hang Tang, Bin Xiao, Zhenhua Yuan, Chunping Qiao, Jianbin Li,<br />
Bo He, Juan Li, Xiao Xiao.<br />
368<br />
Eficacy <strong>of</strong> Various Populations <strong>of</strong> Murine Mesenchymal<br />
Stem <strong>Cell</strong>s for the Treatment <strong>of</strong> Critical Limb Ischemia<br />
Francesca Bortolotti, Laura Ukovich, Vahid Razban, Valentina<br />
Martinelli, Giulia Ruozi, Franca Dore, Mauro Giacca, Serena<br />
Zacchigna.<br />
369<br />
Functional Impairment <strong>of</strong> Circulating Angiogenic <strong>Cell</strong>s<br />
for Treatment <strong>of</strong> Myocardial Infarction Is Differentially<br />
Driven by Both Advanced Age and Coronary Artery<br />
Disease, and Is Improved or Further Impaired by<br />
Modulating Nitric Oxide Synthase Expression<br />
Qiumei Chen, Monika Varga, Xiaoyin Wang, Daniel J. Haddad,<br />
Songtao An, Lejla Medzikovic, Yan Zhang, Brian T. Clifford, Wenhui<br />
Gong, Jeffrey R. Fineman, Christian Heiss, Kirstin A. Aschbacher,<br />
Yerem Yeghiazarians, Matthew L. Springer.<br />
370<br />
Dobutamine Stress Enhances Echocardiography<br />
Detection in mdx Mouse Heart and Reveals Therapeutic<br />
Beneits by Minidystrophin <strong>Gene</strong> <strong>Therapy</strong><br />
Chunping Qiao, Chunxia Zhao, Chi-Hsien Wang, Zhenhua Yuan,<br />
Jianbin Li, Juan Li, Xiao Xiao.<br />
371<br />
Ixmyelocel T: A <strong>Cell</strong>ular <strong>Therapy</strong> for the Treatment <strong>of</strong><br />
Severe, Chronic Cardiovascular Disease<br />
Ronnda L. Bartel.<br />
372<br />
Prevention <strong>of</strong> Restenosis through Ad-iNOS <strong>Gene</strong><br />
<strong>Therapy</strong> Combined with Supplementation <strong>of</strong><br />
Tetrahydrobiopterin and Arginine<br />
Scott P. Forbes, Ivan Alferiev, Richard F. Adamo, Michael Chorny,<br />
Robert J. Levy, Ilia Fishbein.<br />
373<br />
Safety <strong>of</strong> Direct Administration <strong>of</strong> AdVEGF-All6A+, an<br />
Adenovirus Vector Expressing a cDNA/Genomic Hybrid<br />
<strong>of</strong> Human Vascular Endothelial Growth Factors, to the<br />
Ischemic Myocardium <strong>of</strong> Rats<br />
Stephen M. Kaminsky, Lucy Quach, Stacey Chen, Lorraine Pierre-<br />
Destine, Benjamin Van de Graaf, Sébastien Monette, Jonathan B.<br />
Rosenberg, Dolan Sondhi, Neil R. Hackett, Ronald G. Crystal.<br />
374<br />
A Novel Anti-Inlammatory Formulation Approach<br />
To Enhance Myocardial <strong>Gene</strong> Transfer: An In-Vitro<br />
Assessment <strong>of</strong> Aspirin Loaded Poly-Lactic Glycolic Acid<br />
Nanoparticles<br />
Anthony S. Fargnoli, Anbin Mu, Michael G. Katz, Richard D.<br />
Williams, Marina V. Sumaroka, Robert Levy, David B. Weiner, Scott<br />
L. Diamond, Kenneth B. Margulies, Shu Yang, Charles R. Bridges.<br />
375<br />
Development <strong>of</strong> In Vitro and In Vivo Evaluation<br />
Methods for <strong>Gene</strong> <strong>Therapy</strong> Products<br />
Il Ung Oh, Ki Dae Park, Soyeong Kang, Ju Hyun Choi, Sung Yeol<br />
Han, Min Jeong Choi, Sun-Young Baek.<br />
Neurologic <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> I<br />
376<br />
Restoration <strong>of</strong> the Motor and Behavior Phenotype<br />
with AAV9 in Aromatic L-Amino Acid Decarboxylase<br />
Deiciency Mice<br />
Ni-Chung Lee, Yin-Hsiu Chien, Shin-ichi Muramatsu, Barry J.<br />
Byrne, Wuh-Liang Hwu.<br />
377<br />
Signiicant Therapeutic Beneit after Post-Symptomatic<br />
<strong>Gene</strong> <strong>Therapy</strong> in a Feline Model <strong>of</strong> GM2 Gangliosidosis<br />
Victoria J. McCurdy, Ashley N. Randle, Heather L. Gray-Edwards,<br />
Allison M. Bradbury, Aime K. Johnson, Patricia M. Beadlescomb,<br />
Miguel Sena-Esteves, Douglas R. Martin.<br />
378<br />
Tight Regulation <strong>of</strong> Transgene Expression by<br />
Mifepristone-Inducible System after In Vitro Delivery<br />
by Lentiviral Vectors to Neural Progenitor <strong>Cell</strong>s<br />
Christopher Grunseich, Yijun Shi, Michael Mooney, Kenneth H.<br />
Fischbeck, Tyler M. Pierson.<br />
Friday, May 17, 2013<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013<br />
115
Abstract Directory, Friday, May 17, 2013<br />
Friday, May 17, 2013<br />
Poster Session II, continued<br />
5:30 PM - 7:30 PM<br />
Room: Exhibit Hall C/D<br />
Neurologic <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> I<br />
379<br />
Intravenous AAV9 Based <strong>Gene</strong> <strong>Therapy</strong> Corrects a<br />
Mouse Model <strong>of</strong> Sandh<strong>of</strong>f Disease<br />
Jagdeep S. Walia, Alexander Bello, Naderah Altaleb, Christa Kruck,<br />
Richard Hemming, Gary P. Kobinger, Barbara Triggs-Raine.<br />
380<br />
A Speciic Cleavage <strong>of</strong> Amyloid-b by a Viral Protease<br />
NIa Prevents the Amyloid-b-Mediated Mitochondrial<br />
Dysfunction<br />
Woo Jin Park, Bae-Hyun Shin, Hye-Eun Han, Sang Min Park.<br />
381<br />
Viral Vector Mediated Overexpression <strong>of</strong> Pleiotrophin<br />
for Disease Modiication in Parkinson’s Disease<br />
S. E. Gombash, F. P. Manfredsson, C. J. Kemp, S. L. Wohlgenant, N. C<br />
Kuhn, S. M. Fleming, R. J. Mandel, T. H. Collier, C. E. Sortwell.<br />
382<br />
Infrared Fluorescent Protein (iRFP) as a Reporter for<br />
Monitoring and Modulating Neurons<br />
Christopher T. Richie, Lowella V. Fortuno, Douglas B. Howard,<br />
Leslie R. Whitaker, Bruce T. Hope, Brandon K. Harvey.<br />
383<br />
Fetal Brain-Directed AAV5 and AAV9 <strong>Gene</strong> <strong>Therapy</strong><br />
Results in Rapid, Robust, and Persistent Transgene<br />
Expression in Mouse Choroid Plexus Epithelia<br />
Marie Reine Haddad, Eun-Young Choi, Anthony Donsante, Patricia<br />
M. Zerfas, Stephen G. Kaler.<br />
384<br />
Intravenous Administration <strong>of</strong> AAV9-mbgal in a GM1<br />
Gangliosidosis Mouse Model Expresses Enzyme in the<br />
CNS and Delays Disease Onset with Gender Differences<br />
Cara M. Weismann, Jennifer Ferreira, Stacy Maitland, Quin Su,<br />
Guangping Gao, Miguel Sena-Esteves.<br />
385<br />
<strong>Gene</strong> Delivery <strong>of</strong> ATP6 by a Mitochondrial Targeting<br />
Sequence Modiication <strong>of</strong> AAV9 Capsid VP2 Rescues<br />
<strong>Cell</strong>s with Mutated T8993G MtDNA Responsible for<br />
Neuropathy Ataxia and Retinitis Pigmentosa and<br />
Expresses in the Mouse CNS<br />
John Guy, Huijan Yuan.<br />
386<br />
rAAV2/5-Mediated <strong>Gene</strong> Transfer Is Less Eficient<br />
in the Aged Rat Brain: Examination <strong>of</strong> a-Synuclein-<br />
Mediated Neurodegeneration in the Nigrostriatal<br />
System<br />
Sara E. Gombash, Fredric P. Manfredsson, Christopher H. Kemp,<br />
David L. Fischer, Nathan C. Kuhn, Allyson Cole-Strauss, Ronald J.<br />
Mandel, Sheila M. Fleminig, Timothy J. Collier, Jack W. Lipton, Caryl<br />
E. Sortwell.<br />
387<br />
Safety Study <strong>of</strong> Intracranial AAVrh8-Mediated <strong>Gene</strong><br />
Transfer <strong>of</strong> beta-Hexosaminidase in Non-Human<br />
Primates<br />
Diane Golebiowski, Imramsjah M. J. van der Bom, Churl-Su Kwon,<br />
Stacy Maitland, Anna Luisa Kühn, Nina Bishop, Elizabeth Curren,<br />
Nilsa Silva, Andrew Miller, Susan Westmoreland, Matthew Gounis,<br />
Douglas R. Martin, Wael Asaad, Miguel Sena-Esteves.<br />
388<br />
Targeting Human SOD1 Using AAV Mediated RNAi in a<br />
Mouse Model <strong>of</strong> Amyotrophic Lateral Sclerosis<br />
Lorelei I. Stoica, Gabriella Toro, Robert Brown, Christian Mueller,<br />
Miguel Sena-Esteves.<br />
389<br />
TALEFrat#8–VP64 Increases Frataxin <strong>Gene</strong> and Protein<br />
Expression in Fibroblasts <strong>of</strong> Friedreich Patients and <strong>of</strong><br />
Mouse Model<br />
Jacques P. Tremblay, Pierre Chapdelaine, Jean-Paul Iyombe, Joël<br />
Rousseau, Zoé Coulombe.<br />
390<br />
Transduction <strong>of</strong> the Central Nervous System Following<br />
Intra-Cerebroventricular Administration <strong>of</strong> Adeno-<br />
Associated Viral Vectors to Neonatal and Juvenile Mice<br />
Shervin Gholizadeh, Sujeenthar Tharmalingam, Margarita E.<br />
MacAldaz, David R. Hampson.<br />
391<br />
Serial and Endpoint Biomarkers for Cardiorespiratory<br />
Function in a Canine Model <strong>of</strong> DMD Provide an<br />
Integrative View <strong>of</strong> Disease Pathogenesis and Response<br />
to <strong>Therapy</strong><br />
Andrew Mead, Alock Malik, Mihail Petrov, Martin Childers, Janet<br />
Bogan, Benjamin Kozyak, Hansell Stedman.<br />
590 (Moved from Poster Session III)<br />
Intracranial AAV <strong>Gene</strong> <strong>Therapy</strong> Extends the Life Span<br />
<strong>of</strong> GM2 Gangliosidosis Cats >Four-Fold with No Clinical<br />
Evidence <strong>of</strong> Vector Toxicity<br />
Allison M. Bradbury, Victoria J. McCurdy, Aime K. Johnson, Heather<br />
Gray-Edwards, Brandon L. Brunson, Ashley N. Randle, Nancy R.<br />
Cox, Miguel Sena-Esteves, Douglas R. Martin.<br />
116<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013
Abstract Directory, Friday, May 17, 2013<br />
Poster Session II, continued<br />
5:30 PM - 7:30 PM<br />
Room: Exhibit Hall C/D<br />
Cancer Immunotherapy II<br />
392<br />
Restored Expression <strong>of</strong> Heparanase in Tumor<br />
Speciic T <strong>Cell</strong>s Enhances Their Antitumor Effects in<br />
Neuroblastoma<br />
Ignazio Caruana, Valentina Hoyos, Gerrit Weber, Barbara Savoldo,<br />
Dario Marchetti, Gianpietro Dotti.<br />
393<br />
Single-<strong>Cell</strong> Imaging Reveals That Subsets <strong>of</strong> T <strong>Cell</strong>s<br />
Expressing a CD19-Speciic Chimeric Antigen Receptor<br />
Differ in Effector Function<br />
Harjeet Singh, Ivan Liadi, Gabrielle Romain, Navin Varadarajan,<br />
Laurence J. N. Cooper.<br />
394<br />
MCSP-Chimeric Antigen Receptor (CAR)-Redirected T<br />
<strong>Cell</strong>s Target Multiple Solid Tumors<br />
Claudia Arambula, Barbara Savoldo, Valentina Hoyos, Eric Yvon,<br />
Michele Del Vecchio, Soldano Ferrone, Gianpietro Dotti.<br />
395<br />
Tumor Recurrences Share Immunogenic Antigens<br />
across Both Tumor Types and Primary Treatments<br />
Which Can Be Therapeutically Targeted with VSV-cDNA<br />
Libraries<br />
Nicolas Boisgerault, Jose Pulido, Timothy Kottke, Oliver Donnelly,<br />
Esteban Celis, Jill Thompson, Rosa Diaz, Kevin Harrington, Hardev<br />
Pandha, Peter Selby, Alan Melcher, Richard Vile.<br />
396<br />
BARF1-Speciic T <strong>Cell</strong>s for the Adoptive Immunotherapy<br />
<strong>of</strong> EBV-Positive Nasopharyngeal Carcinoma<br />
Mamta Kalra, Minhtran C. Ngo, Ulrike Gerdemann, Ann Leen,<br />
Chrystal Louis, Cliona M. Rooney, Stephen Gottschalk.<br />
397<br />
IL-12 Increases Immune Responses Induced by a Novel<br />
pDNA Prostate Cancer Immunotherapy Approach in<br />
Non-Human Primates<br />
Bernadette Ferraro, Amritha Balakrishnan, Jewell N. Walters,<br />
Devin J. Myles, Jian Yan, Amir S. Khan, Niranjan Y. Sardesai, David<br />
B. Weiner.<br />
398<br />
Transgenic Expression <strong>of</strong> a Novel Immunosuppressive<br />
Signal Converter on T <strong>Cell</strong>s<br />
Norihiro Watanabe, Usanarat Anurathapan, Malcolm K. Brenner,<br />
Helen E. Heslop, Ann M. Leen, Cliona M. Rooney, Juan F. Vera.<br />
399<br />
Hypomethylating Agent 5-aza Cytidine Exposure To<br />
Improve AP1903 Treatment for Apoptosis Induction <strong>of</strong><br />
iCasp9/DCD19 <strong>Gene</strong> Modiied T <strong>Cell</strong>s<br />
Elodie Bole-Richard, Jean-Marie Certoux, Idir Idirene, Laurent<br />
Jossot, Eric Deconinck, Fabrice Larosa, Etienne Daguindau,<br />
Christophe Borg, Pierre Tiberghien, Christophe Ferrand, Marina<br />
Deschamps.<br />
400<br />
Adapting the Spontaneous Canine Osteosarcoma Model<br />
for T-<strong>Cell</strong> <strong>Therapy</strong><br />
Melinda Mata, Sunitha Kakarla, Juan Vera, Lisa Wang, Cliona<br />
Rooney, Heather Wilson, Stephen Gottschalk.<br />
401<br />
T-<strong>Cell</strong> Engineering for Adoptive Immunotherapy Using<br />
TAL-Effector Nucleases (TALENTM)<br />
Laurent Poirot Poirot, Cécile Schiffer-Mannioui, Agnes Gouble,<br />
Roman Galetto, Stephanie Grosse, Isabelle Chion-Sotinel, Diane Le<br />
Clerre, Laetitia Lemaire, Céline Lebuhotel, Justin Eyquem, Sylvain<br />
Arnould, Julianne Smith, Andrew Scharenberg, Sophie Derniame.<br />
402<br />
Randomized Phase II Trial <strong>of</strong> Post-Operative Adjuvant<br />
Chemotherapy ±FANG Autologous Tumor <strong>Cell</strong> Vaccine<br />
in Colorectal Carcinoma with Liver Metastases<br />
Joseph Kuhn, Neil Senzer, Minal Barve, G. Thomas Shires,<br />
Padmasini Kumar, Donald D. Rao, Gladice Wallraven, Beena O.<br />
Pappen, Phillip B. Maples, John Nemunaitis.<br />
403<br />
The Potential <strong>of</strong> Pro-Apoptotic PAC-1 Treatment<br />
for Augmentation <strong>of</strong> IL-2 Mediated Breast Cancer<br />
Immunotherapy<br />
Shyambabu Chaurasiya, Paggy Hew, Paul J. Hergenrother, David<br />
Sharon, Mary Hitt.<br />
404<br />
Effect <strong>of</strong> IL-15 <strong>Gene</strong> <strong>Therapy</strong> and mTOR Inhibitor<br />
Everolimus on Experimental Metastatic Breast Cancer<br />
Growth<br />
Na Zhao, Xiaodan Li, Feng Qi, Yujie Qiu, Xianghui He, Liwei Zhu.<br />
Cancer-Oncolytic Viruses I<br />
405<br />
Measles Virus for Immunovirotherapy <strong>of</strong> Cancer<br />
Christian Grossardt, Christine E. Engeland, Sascha Bossow, Silke<br />
Hamzaoui-Nord, Niels Halama, Dirk Jäger, Christ<strong>of</strong> von Kalle, Guy<br />
Ungerechts.<br />
406<br />
Oncolytic Herpes Simplex Viruses Encoding an<br />
Antibody Retargeted C5a Complement Protein for<br />
Multiple Myeloma <strong>Therapy</strong><br />
Hongtao Li, Stephen J. Russell.<br />
Friday, May 17, 2013<br />
407<br />
Mengovirus <strong>Therapy</strong> for Multiple Myeloma<br />
Autumn Ruiz, Elizabeth M. Hadac, Stephen J. Russell.<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013<br />
117
Abstract Directory, Friday, May 17, 2013<br />
Friday, May 17, 2013<br />
Poster Session II, continued<br />
5:30 PM - 7:30 PM<br />
Room: Exhibit Hall C/D<br />
Cancer-Oncolytic Viruses I<br />
408<br />
Systemic Administration <strong>of</strong> Fiber Redesigned<br />
Infectivity-Selective Oncolytic Adenovirus for Cancer<br />
Treatment<br />
Yoshiaki Miura, Joohee Han, Julia Davydova, Masato Yamamoto.<br />
409<br />
Therapeutic Eficacy <strong>of</strong> Oncolytic Herpes Virus and<br />
Chemotherapy for Treatment <strong>of</strong> Late-Stage Ovarian<br />
Cancer<br />
Chelsea M. Bolyard, Balveen Kaur.<br />
410<br />
T-<strong>Cell</strong> Engager-Armed Oncolytic Vaccinia Virus<br />
Signiicantly Enhances Antitumor <strong>Therapy</strong><br />
Feng Yu, Stephen M. Gottschalk, Xiao-Tong Song.<br />
411<br />
Improving the Oncolytic Activity <strong>of</strong> Measles Virus<br />
by Enhancing the Neutrophil-Mediated Response to<br />
Infected Tumor <strong>Cell</strong>s<br />
Aditi Dey, Yu Zhang, Anna Z. Castleton, Bella Patel.<br />
412<br />
Directed Evolution <strong>of</strong> Oncolytic HSV To Overcome<br />
Resistance to Virotherapy Due to Low Entry Receptor<br />
Expression<br />
Pin-Yi Wang, Pearlly Yan, Ralf Bundschuh, Hok-Hei Tam, Timothy<br />
P. Cripe.<br />
413<br />
VSV Pseudotyped with Maraba Virus G Glycoprotein<br />
Escapes Serum Neutralization<br />
Mulu Z. Tesfay, Arun Ammayappan, Mark J. Federspiel, Glen N.<br />
Barber, David F. Stojdl, Kah-Whye Peng, Stephen J. Russell.<br />
414<br />
Oncolytic Adenovirus-Mediated Interferon<br />
Expression Augments Chemoradiation <strong>Therapy</strong> in<br />
Immunocompetent Pancreatic Cancer Model<br />
Joohee Han, Yoshi Miura, Ryan Shanley, Xianghua Luo, Kazunori<br />
Aoki, Selwyn M. Vickers, Masato Yamamoto, Julia Davydova.<br />
415<br />
High-Resolution SPECT/CT Imaging Elucidates<br />
Molecular Determinants <strong>of</strong> Viral Oncolysis in Primary<br />
and Metastatic Lymphoma<br />
Tanner S. Miest, Marie Frenzke, Roberto Cattaneo.<br />
416<br />
Enhanced GBM <strong>Therapy</strong> Using a New Class <strong>of</strong> HSV<br />
Oncolytic Vector<br />
Lucia Mazzacurati, Marco Marzulli, Bonnie Reinhart, Justus B.<br />
Cohen, Glorioso C. Joseph, Grandi Paola.<br />
417<br />
<strong>Gene</strong> Expression Proiling Identiies Predictive Markers<br />
<strong>of</strong> Response to Oncolytic Measles Virus <strong>Therapy</strong><br />
Ianko Iankov, Cory Allen, Ian Parney, Caterina Giannini, Evanthia<br />
Galanis.<br />
Hematologic and Immunologic <strong>Gene</strong> & <strong>Cell</strong><br />
<strong>Therapy</strong> I<br />
418<br />
Enhanced Engraftment <strong>of</strong> Hematopoietic Stem<br />
<strong>Cell</strong>s (HSCs) by Inhibition <strong>of</strong> Mammalian Target <strong>of</strong><br />
Rapamycin<br />
Emily A. Partridge, Jesse Vrecenak, Merek Kozlowski, Alan W.<br />
Flake.<br />
419<br />
Improved Expansion <strong>of</strong> Primitive Erythroid/<br />
Megakaryocyte Precursors from Cord Blood CD34+<br />
<strong>Cell</strong>s with Hyperactive Variants <strong>of</strong> Pharmacologically-<br />
Regulated Mpl<br />
Eyayu Belay, Christophre P. Miller, Amanda Kortum, Beverly Torok-<br />
Storb, C. Anthony Blau, David W. Emery.<br />
420<br />
The Challenge <strong>of</strong> HSCs Procurement for <strong>Gene</strong><br />
<strong>Therapy</strong> <strong>of</strong> beta-Thalassemia: Exploring Plerixafor as<br />
Mobilization Agent<br />
Maria Rosa Lidonnici, Annamaria Aprile, Marta Frittoli, Giacomo<br />
Mandelli, Bernhard Gentner, Laura Bellio, Elena Cassinerio, Laura<br />
Zanaboni, Silvano Rossini, Maria Domenica Cappellini, Fabio<br />
Ciceri, Sarah Marktel, Giuliana Ferrari.<br />
421<br />
Leukocyte Telomere Maintenance after Transplant and<br />
In Vivo Chemoselection <strong>of</strong> Mutant MGMTP140K <strong>Gene</strong>-<br />
Modiied Hematopoietic <strong>Cell</strong>s<br />
Christopher R. Burtner, Devikha Chandrasekaran, Brian C. Beard,<br />
Hans-Peter Kiem, Jennifer E. Adair.<br />
422<br />
Assessing Clonal Composition <strong>of</strong> Human Hematopoiesis<br />
in an NSG Transplantation Model after In Vitro<br />
Expansion <strong>of</strong> Transduced Cord Blood CD34 <strong>Cell</strong>s<br />
Reinhard Haemmerle, Ruhi Phaltane, Michael Rothe, Thomas<br />
Moritz, Ute Modlich.<br />
423<br />
Targeted Expansion <strong>of</strong> Individual Hematopoietic<br />
Lineages by Regulated Mpl Signaling<br />
David W. Emery, Eyayu Belay, Christopher P. Miller, Beverly Torok-<br />
Storb, C. Anthony Blau.<br />
424<br />
Correction <strong>of</strong> Murine Sickle <strong>Cell</strong> Disease Using Non-<br />
Ablative Conditioning and Autologous Bone Marrow<br />
<strong>Cell</strong>s Transduced with a Gamma Globin Lentiviral<br />
Vector<br />
Tamara I. Pestina, Phillip W. Hargrove, Hui Fen Zhao, Derek A.<br />
Persons.<br />
118<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013
Abstract Directory, Friday, May 17, 2013<br />
Poster Session II, continued<br />
5:30 PM - 7:30 PM<br />
Room: Exhibit Hall C/D<br />
Hematologic and Immunologic <strong>Gene</strong> & <strong>Cell</strong><br />
<strong>Therapy</strong> I<br />
425<br />
Evaluating a LM-PCR and Pyrosequencing Method<br />
for the Detection <strong>of</strong> Clonal Contribution in Polyclonal<br />
Retrovirally Transduced Samples<br />
Martijn H. Brugman, Julia D. Suerth, Michael Rothe, Sebastian<br />
Suerbaum, Axel Schambach, Ute Modlich, Olga S. Kustikova,<br />
Christopher Baum.<br />
426<br />
Disease Modeling Using Induced Pluripotent Stem <strong>Cell</strong>s<br />
Derived from Severe Congenital Neutropenia Patients<br />
Lisa Trump, Ramesh Nayak, Jose Cancelas, Carolyn Lutzko.<br />
427<br />
Enhanced Engraftment <strong>of</strong> Murine and Human<br />
Hematopoietic Stem <strong>Cell</strong>s Mobilized Using the<br />
Plerixafor+G-CSF Combination<br />
Nikoleta Psatha, Garyfalia Karponi, Eleni Sgouramali, Athanasios<br />
Siametis, Panayotis Baliakas, Isabelle Riviere, Michel Sadelain,<br />
Achilles Anagnostopoulos, George Stamatoyannopoulos, Thalia<br />
Papayannopoulou, Evangelia Yannaki.<br />
428<br />
Evaluation <strong>of</strong> Engraftment and Tolerance Induction<br />
after Reduced Intensity Conditioning for Hematopoietic<br />
Stem <strong>Cell</strong> Transplantation in the Rhesus <strong>Gene</strong> <strong>Therapy</strong><br />
Model<br />
Naoya Uchida, Molly Evans, Richard Weitzel, Aylin Bonifacino,<br />
Sandra Price, Allen Krouse, Mark Metzger, Matthew Hsieh, Robert<br />
Donahue, John Tisdale.<br />
429<br />
Myeloprotective Properties <strong>of</strong> Inducible (shRNA-<br />
Mediated) Knock-Down <strong>of</strong> Deoxycytidine-Kinase (dCK)<br />
in Human Hematopoietic In Vitro Models<br />
Nico Lachmann, Kevin Czarnecki, Miriam Hetzel, Sebastian<br />
Brennig, Niels Heinz, Bernd Schiedlmeier, Thomas Moritz.<br />
430<br />
Comparision <strong>of</strong> In-Utero <strong>Gene</strong> <strong>Therapy</strong> and In-Utero<br />
Hemopoietic Stem <strong>Cell</strong> <strong>Therapy</strong> Approaches in Severe<br />
Models <strong>of</strong> Murine beta Thalassemia<br />
Niraja Dighe, Citra Mattar, Lay Geok Tan, Daren Sandikin, Simon<br />
Waddington, Michael Antoniou, Mahesh Choolani, Jerry Chan.<br />
431<br />
Regulatory T <strong>Cell</strong>s Suitable for Clinical Use Can Be<br />
Propagated on Artiicial Antigen Presenting <strong>Cell</strong>s<br />
Pappanaicken Kumaresan, Chetan Dhamne, Sourindra Maiti,<br />
Janani Krishnamoorthy, Radhika Thokala, Helen Huls, Champlin<br />
Richard, Dean Lee, Dat Tran, Laurence Cooper.<br />
<strong>Gene</strong> and <strong>Cell</strong> <strong>Therapy</strong> for Pulmonary<br />
Diseases<br />
432 Abstract Moved to Oral Abstract Session 237<br />
433<br />
Eficient <strong>Gene</strong> Delivery to Pig Airway Epithelia<br />
and Submucosal Glands Using Helper-Dependent<br />
Adenoviral Vectors<br />
Huibi Cao, Tiago N. Machuca, Jonathan Yeung, Jing Wu, Kai Du,<br />
Cathleen Duan, Riccardo Bonato, Allan L. Coates, Kitty Leung,<br />
Herman Yeger, Ernest Cutz, Mingyao Liu, Shaf Keshavjee, Jim Hu.<br />
434<br />
Minicircle Plasmids Result in Enhanced and Prolonged<br />
Transgene Expression <strong>of</strong> Airway Epithelial <strong>Cell</strong>s Both In<br />
Vitro and In Vivo<br />
Mustafa M. Munye, Rachel E. Brown, Aristides D. Tagalakis,<br />
Josephine L. Barnes, Rupinder K. Ghatrora, Martin Pule, Mark F.<br />
Lythgoe, Robin J. McAnulty, Steven J. Howe, Stephen L. Hart.<br />
435<br />
Novel Human Bronchial Epithelial <strong>Cell</strong> Lines for the<br />
Study <strong>of</strong> <strong>Gene</strong> <strong>Therapy</strong> Approaches To Treat Primary<br />
Ciliary Dyskinesia<br />
Mustafa M. Munye, Claire M. Smith, Rob A. Hirst, Amelia Shoemark,<br />
Juliette M. Delhove, Tristan R. Mckay, Claire Hogg, Christopher<br />
O’Callaghan, Steven J. Howe, Stephen L. Hart.<br />
436<br />
Upregulation <strong>of</strong> a Cluster <strong>of</strong> microRNAs on Mouse<br />
Chromosome 12qF1 in a Kras Mutant Mouse Model <strong>of</strong><br />
Lung Adenocarcinoma<br />
Paul N. Valdmanis, Biswajoy Roy-Chaudhuri, Hak Kyun Kim,<br />
Leanne C. Sayles, Yanyan Zheng, Chen-Hua Chuang, Debbie<br />
Caswell, Monte Winslow, E. A. Sweet-Cordero, Mark A. Kay.<br />
437<br />
Lentiviral-Mediated Functional Correction <strong>of</strong><br />
Hermansky-Pudlak Syndrome Type 1 Melanocytes<br />
Yasuhiro Ikawa, Richard Hess, Heidi Dorward, Bernadette<br />
Gochuico, William A. Gahl, Fabio Candotti.<br />
438<br />
Development <strong>of</strong> a Myeloid <strong>Cell</strong>-Binding Adenovirus for<br />
the Potential <strong>Gene</strong> <strong>Therapy</strong> <strong>of</strong> Alpha-1 Anti-Trypsin<br />
Deiciency<br />
Maurizio Buggio, Christopher Towe, Michael O. Alberti, Justin C.<br />
Roth, Anand C. Annan, David T. Curiel.<br />
439<br />
MicroRNAs as Potential Targets for <strong>Therapy</strong> <strong>of</strong><br />
Pulmonary Hypertension<br />
Igor B. Gubrij, Amanda K. Pangle, Li Pang, Larry G. Johnson.<br />
440<br />
CVB3 Infection Elicits Potent Oncolytic Activity Against<br />
Lung Cancer Stem <strong>Cell</strong>s<br />
Miyako Sagara, Hiroyuki Inoue, Shohei Miyamoto, Chika Sakamoto,<br />
Yuki Nakano, Koichi Takayama, Hiroyuki Shimizu, Yoichi<br />
Nakanishi, Kenzaburo Tani.<br />
Friday, May 17, 2013<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013<br />
119
Abstract Directory, Friday, May 17, 2013<br />
Friday, May 17, 2013<br />
Poster Session II, continued<br />
5:30 PM - 7:30 PM<br />
Room: Exhibit Hall C/D<br />
Immunologic & Host Responses in <strong>Gene</strong> & <strong>Cell</strong><br />
<strong>Therapy</strong><br />
441<br />
Liver-Directed <strong>Gene</strong> <strong>Therapy</strong> with Lentiviral Vector<br />
Reverts Pre-Existing Immunity to Coagulation Factor IX<br />
and Rescues Hemophilia B Mice from the Disease<br />
Andrea Annoni, Alessio Cantore, Kevin Goudy, Mahzad Akbarpour,<br />
Fabio Russo, Sara Bartolaccini, Luigi Naldini, Maria Grazia<br />
Roncarolo.<br />
442<br />
Combination Use <strong>of</strong> TLR7 Ligand with GM-CSF <strong>Gene</strong>-<br />
Transduced Tumor Vaccines Provides Substantial<br />
Antitumor Immunity Against Poorly Immunogenic<br />
Mouse Lung Cancer <strong>Cell</strong>s<br />
Megumi Narusawa, Hiroyuki Inoue, Chika Sakamoto, Tomoko<br />
Inoue, Shohei Miyamoto, Makoto Inoue, Koichi Takayama, Mamoru<br />
Hasegawa, Yoichi Nakanishi, Kenzaburo Tani.<br />
443<br />
Elimination <strong>of</strong> Contaminating Capsid-Expressing <strong>Gene</strong>s<br />
in AAV Vectors Reduces Immune Responses to Vector<br />
Administration in a Canine <strong>Gene</strong> <strong>Therapy</strong> Model<br />
Zejing Wang, Christine L. Halbert, Donghoon Lee, Tiffany Butts,<br />
Rainer Storb, Stephen H. Tapscott, Dusty Miller.<br />
444<br />
Immune Activation and Transgene Stability Following<br />
AAV9 <strong>Gene</strong> Transfer to an Acute Myocardial Infarct<br />
Mouse Model<br />
Susan Faust, Ehre Gao, Rebecca Tenney, Jason Ohlstein, Peter Bell,<br />
Walter Koch, James Wilson, Joseph Rabinowitz.<br />
445<br />
Blood Proteins Affecting rAAV Eficiency<br />
Jerome Denard, Hans Jörg Fehling, Philippe Moullier, Tomas Voit,<br />
Fedor Svinartchouk.<br />
446<br />
B & T <strong>Cell</strong> Immunomodulation Attenuates Anaphylaxis<br />
in a Novel Mouse Model <strong>of</strong> Pompe Disease<br />
Phillip A. Doerler, Sushrusha Nayak, Roland W. Herzog, Barry J.<br />
Byrne.<br />
447<br />
Quantiication <strong>of</strong> Alpha-L-Iduronidase Expression<br />
Outcomes in Mice Following Sleeping Beauty<br />
Transposon-Mediated <strong>Gene</strong> <strong>Therapy</strong><br />
Elena L. Aronovich, Bryan C. Hall, Jason B. Bell, R. Scott McIvor,<br />
Perry B. Hackett.<br />
449<br />
Over-Expression <strong>of</strong> Interleukin-6 Alone Induces<br />
Dexamethasone-Relieved Multiple-Organ Lesion in<br />
Mice<br />
Tao Bian, Zhenhua Yuan, Chunxia Zhao, Ting Zhang, Juan Li, Xiao<br />
Xiao.<br />
Oligonucleotide & RNAi Therapeutics<br />
450<br />
AAV6-Mediated Systemic Administration <strong>of</strong> miR30-<br />
Based RNAi Hairpins Impacts Muscle Disease in the<br />
HSALR Myotonic Dystrophy Mouse Model<br />
Darren R. Bisset, Maja Zavaljevski, Carrie Stoltzman, Joel R.<br />
Chamberlain.<br />
451<br />
Peptide-Conjugated Morpholino Rescues Utrophin-<br />
Dystrophin-Deicient Mice by Restoring Dystrophin<br />
Expression and Improvement in Muscle Pathology and<br />
Function<br />
Bo Wu, Caryn Cloer, Stephanie Milazi, Mona Shaban, Sapana N.<br />
Shah, Peijuan Lu, Hong M. Moulton, Qi Long Lu.<br />
452<br />
Co-Delivery <strong>of</strong> VEGF siRNA and IL-4 into Chitosan<br />
Nanoparticles in Breast Tumor Model <strong>of</strong> Rat<br />
Emine Salva, Suna Özbas-Turan, Levent Kabasakal, Fatih Eren,<br />
Saadet Alan, Naziye Özkan, Jülide Akbuga.<br />
453<br />
Systemic Delivery <strong>of</strong> a Peptide-Linked Morpholino<br />
Oligonucleotide Neutralizes Mutant RNA Toxicity in a<br />
Mouse Model <strong>of</strong> Myotonic Dystrophy<br />
Andrew J. Leger, Leocadia M. Mosquea, Nicholas P. Clayton,<br />
I.-Huan Wu, Timothy Weeden, Carol A. Nelson, Lucy Phillips,<br />
Errin Roberts, Peter A. Piepenhagen, Seng H. Cheng, Bruce M.<br />
Wentworth.<br />
454<br />
<strong>Gene</strong> Expression from Pseudouridine and<br />
5-Methylcytidine Modiied Messenger RNAs<br />
Jiehua Zhou, Maggie L. Bobbin, Julie Escamilla-Powers, Eric<br />
Arnold, Anthony Lauer, Laura Juckem, Karl-Dimiter Bissig, Anton<br />
P. McCaffrey, John J. Rossi.<br />
455<br />
Integrin-Targeted Nanoparticle Delivery <strong>of</strong> miRNA for<br />
Anti-Tumor <strong>Therapy</strong><br />
Puthupparampil Scaria, Yijia Liu, Betty Zhou, Martin Woodle.<br />
456<br />
Multi-Subtype Targeting, Escape-Reducing (MuSTER)<br />
Anti-HIV shRNA <strong>Gene</strong> <strong>Therapy</strong><br />
Ulrike Jung, John J. Rossi.<br />
448<br />
Hepatocyte Heparan Sulfate Is Dispensable for<br />
Adenovirus Liver Transduction In Vivo<br />
Anne K. Zaiss, Erin Foley, Ding Xu, Roger Lawrence, Jeffrey D. Esko,<br />
Harvey R. Herschman.<br />
120<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013
Abstract Directory, Friday, May 17, 2013<br />
Poster Session II, continued<br />
5:30 PM - 7:30 PM<br />
Room: Exhibit Hall C/D<br />
Oligonucleotide & RNAi Therapeutics<br />
457<br />
RNA Interference-Based <strong>Gene</strong> <strong>Therapy</strong> for Parkinson’s<br />
Disease Using shRNA That Speciically Targeting the<br />
LRRK2 G2019S Allele<br />
Liang Huang, Xiaomin Su, Juan Wang, Kathleen Maguire-Zeiss,<br />
Howard J. Feder<strong>of</strong>f.<br />
458<br />
An RNAi Screening Platform To Identify Modulators <strong>of</strong><br />
Glioma Stem <strong>Cell</strong>s Viability and Differentiation<br />
Christian E. Badr, Romain Amante, Stephanie Van Hoppe, Bakhos<br />
A. Tannous.<br />
459<br />
Short Hairpin RNAs (shRNA) Against Steroid Receptor<br />
Coactivator-3/Ampliied in Breast Cancer 1 (SRC-3)<br />
and Steroid Receptor Coactivator-1 (SRC-1) as Breast<br />
Cancer Therapeutic Agents<br />
David M. Lonard, Donald Rao, John J. Nemunaitis, Neil N. Senzer,<br />
Bert W. O’Malley.<br />
460<br />
Lentiviral-Mediated ENaCa Knockdown, as a Treatment<br />
for Cystic Fibrosis Lung Disease<br />
Rebekka E. Harding-Smith, Deborah R. Gill, Stephen C. Hyde.<br />
461<br />
Engineering Mammalian <strong>Cell</strong>s To Secrete RNA to the<br />
Extracellular Space<br />
Kevin J. Polach, Leslie Wilkinson, Elaine Brunhoeber, Amy<br />
Pettigrew, Kirby Wallace, Jeff Sparks, Angela Rea-Ramsey, Casey<br />
Pence, Majed Matar, Diane McClure, Jennifer Rice, Khursheed<br />
Anwer, Jason G. Fewell.<br />
462<br />
Lipid-Polymer Mediated siRNA <strong>Therapy</strong> for Silencing<br />
CXCR4 in Acute Myeloid Leukemia<br />
Breanne Landry, Hilal Gül-Uludag, Hasan Uludag.<br />
463<br />
Messenger RNA as a Novel Therapeutic Approach<br />
Antonin de Fougerolles.<br />
464<br />
Development <strong>of</strong> Post-Column Process <strong>of</strong> Immobilized<br />
Colanic Acid Degrading Enzyme Reactor<br />
Chromatography in SuperClean Therapeutic Plasmid<br />
DNA Production<br />
Li Chen, Christopher M. Jay, Nancy S. Templeton, John Nemunaitis,<br />
Nicolas Taquet, Phillip B. Maples.<br />
Pluripotent Stem <strong>Cell</strong>s<br />
465<br />
<strong>Cell</strong> Replacement <strong>Therapy</strong> for Parkinson’s Disease with<br />
Neuronal <strong>Cell</strong>s Derived from Human Parthernogenetic<br />
Stem <strong>Cell</strong><br />
Ibon Garitaonandia, Rodolfo Gonzalez, Alina Ostrowska, Tatiana<br />
Abramihina, Gerald Wambua, Alexander Noskov, Francesca<br />
Boscolo, Louise Laurent, Eugene Redmond, Evan Snyder, Ruslan<br />
Semechkin.<br />
466<br />
<strong>Gene</strong>ration <strong>of</strong> Isogenic Pairs <strong>of</strong> Mutant and<br />
Complemented Fanconi Anemia Human Induced<br />
Pluripotent Stem <strong>Cell</strong>s<br />
Laura I. Marquez, Kathryn Schubert, Feorillo Galivo, Milton<br />
Finegold, Susan Olson, Markus Grompe.<br />
467<br />
Engineering <strong>of</strong> Safety Control Switches for Human<br />
Induced Pluripotent Stem <strong>Cell</strong>s<br />
Wu Ou, Pingjuan Li, Jakob Reiser.<br />
468<br />
Eficient <strong>Gene</strong>ration <strong>of</strong> Integration-Free iPS <strong>Cell</strong>s from<br />
Human Adult Peripheral Blood<br />
Rui-Jun Su, David J. Baylink, Amanda Nieses, Xianmei Meng, Jason<br />
B. Kiroyan, Kimberly J. Payne, Yuyou Duan, Benjamin Tschudy-<br />
Seney, Mary Kearns-Jonker, K.-H William Lau, Xiao-Bing Zhang.<br />
469<br />
iPSC-Based Strategy To Correct the Bleeding Phenotype<br />
in Hemophilia A Targeting FVIII Expression to<br />
Endothelial <strong>Cell</strong>s<br />
Maria Talmon, Gabriella Ranaldo, Chantal Grosso, Angelo<br />
Lombardo, Simone Merlin, Stefania E. Cannizzo, Angel Raya, Luigi<br />
Naldini, Piercarla Schinco, Antonia Follenzi.<br />
470 Abstract Moved to Oral Abstract Session 233<br />
471<br />
Correction <strong>of</strong> DF508 CFTR in Patient-Derived iPS <strong>Cell</strong>s<br />
Using Small Frgement Homologous Replacement<br />
(SFHR) and Transcription Activator-Like Effector<br />
Nucleases (TALENs)<br />
Shingo Suzuki, Thomas J. Cradick, Roy G. Sargent, Lee Albert,<br />
Matthew Porteus, Gang Bao, Dieter C. Gruenert.<br />
472<br />
Directed Differentiation <strong>of</strong> Human Induced Pluripotent<br />
Stem (hiPS) <strong>Cell</strong>s into Functional Parathyroid Tissue<br />
for <strong>Cell</strong> <strong>Therapy</strong><br />
Alaleh Esmaeili Shandiz, Albert Lee, Shingo Suzuki, R. G. Sargent,<br />
Michael H. Yezzi, Lisa A. Orl<strong>of</strong>f, Dieter C. Gruenert.<br />
473<br />
iPSC-Derived Cardiomyocytes from the Urine <strong>of</strong> a<br />
Patient with Duchenne Muscular Dystrophy<br />
Xuan Guan, David Mack, Yingai Shi, Chad Markert, Guihua Liu,<br />
Michael Lawlor, Emily Mooreield, Tara Jones, Mark Furth,<br />
Yuanyuan Zhang, Martin K. Childers.<br />
Friday, May 17, 2013<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013<br />
121
Abstract Directory, Friday, May 17, 2013<br />
Friday, May 17, 2013<br />
Poster Session II, continued<br />
5:30 PM - 7:30 PM<br />
Room: Exhibit Hall C/D<br />
Pluripotent Stem <strong>Cell</strong>s<br />
474<br />
Process Development for Scalable Manufacturing <strong>of</strong><br />
hESC-Derived Cardiomyocytes<br />
Vincent C. Chen, Jingjing Ye, Giau Hua, Ziguang Lin, Dalin Chen,<br />
Aparna Roy, Patricia Huang, Wei Dang, Josh Tompkins, Arthur<br />
Riggs, Wenyi Chen, Joseph Wu, David Hsu, Larry Couture.<br />
475 Abstract Withdrawn from Presentation<br />
476<br />
Development and Qualiication <strong>of</strong> Assays To<br />
Characterize Human Embryonic Stem <strong>Cell</strong>s (hESC) and<br />
hESC-Derived Products at the Center <strong>of</strong> Biomedicine<br />
and <strong>Gene</strong>tics (CBG)<br />
Patricia Huang, Aparna Krishnan, Wei Dang, Vincent Chen, Jing<br />
Jing Ye, Aparna Roy, Patricia Lopez, Jing Chai, Ruilin Wu, Tania<br />
Aguilar, David Hsu, Larry Couture.<br />
Late Breaking Abstracts II<br />
701 Abstract Withdrawn from Presentation<br />
702<br />
Preclinical study <strong>of</strong> a scAAV2/8-Lp1-huPPCA vector in<br />
the treatment <strong>of</strong> the galactosialidosis mouse model<br />
Huimin Hu, Elida Gomero, Erik Bonten, John T Gray, Arthur<br />
Nienhuis, Alessandra d’Azzo<br />
703<br />
Improved Retroviral Replicating Vectors for Prodrug-<br />
Activator <strong>Gene</strong> <strong>Therapy</strong> <strong>of</strong> Cancer<br />
Noriyuki Kasahara, Christopher Logg, Omar Perez, Oscar Diago,<br />
Ryan Burnett, Aki Inagaki, Amy Lin, Cindy Burrascano, Mitchel<br />
Berger, Kryst<strong>of</strong> Bankiewicz, Weijun Wang, Hyun Kim, Walter Wolf,<br />
Thomas Chen, Kenneth Cornetta, Carlos Ibañez, Joan Robbins,<br />
Harry Gruber, Douglas J. Jolly<br />
704<br />
The recombinant tight junction opener JO-1* decreases<br />
hypoxia in tumors<br />
Kamola Saydaminova, Roma Yumul, Christine Wang, Akseli<br />
Hemminki, Andre Lieber<br />
705<br />
Development <strong>of</strong> a Clinical AAV-Based Vector Encoding<br />
a Microdystrophin Transgene for the Treatment <strong>of</strong><br />
Duchenne Muscular Dystrophy<br />
Yoshiaki Miura, Joohee Han, Julia Davydova, Masato Yamamoto<br />
706<br />
Novel therapeutic nanoparticles for in vivo delivery <strong>of</strong><br />
low dose siRNA in liver cells and for the treatment <strong>of</strong><br />
liver ibrosis associated nonalcoholic steatohepatitis<br />
Xavier de Mollerat du Jeu, Akiko Eguchi, Andronikou Nektaria,<br />
Ariel E. Feldsteinand, Peter Welch<br />
707<br />
Directed evolution <strong>of</strong> artiicial viruses<br />
Ir. Erik Teunissen, Dr. Enrico Mastrobattista Pr<strong>of</strong>. Dr. Peter Rottier,<br />
Pr<strong>of</strong>. Dr. Daan Crommelin<br />
708<br />
The NanoAssemblr Platform: Microluidics-based<br />
Manufacture <strong>of</strong> Limit Size Lipid Nanoparticles for<br />
Nucleic Acid Delivery<br />
J. Taylor, C. Walsh, N. Belliveau, P. Lin, R. Rungta, H. Choi, J. Lee, E.<br />
Ramsay, T. Leaver, A. Wild, K. Ou, A. Leung, Y. Tam, I. Hafez, S. Chen,<br />
B. MacVicar, C. Hansen, P. Cullis<br />
709<br />
Preclinical development <strong>of</strong> an AAV vector for the<br />
treatment <strong>of</strong> X-linked retinitis pigmentosa due to<br />
RPGRorf15 mutation<br />
Zhijian Wu, Suja Hiriyanna, Haohua Qian, Suddhasil Mookherjee,<br />
Kayleigh Kaneshiro, Maria Campos, Chun Gao, Robert Fariss,<br />
Anand Swaroop, Tiansen Li, Peter Colosi<br />
710<br />
Cytotoxic T-cell Surface Engineering with Chemically<br />
Self-Assembled Antibody Nanorings (CSANS)<br />
C. R. Wagner, Kari Gabrielse, Dr. Jae Chul Lee<br />
711<br />
Speciic siRNA delivery to primary activated T cells for<br />
anti-inlammatory therapy<br />
Na Hyung Kim, Yuran Xie, Archana Thakur, Lawrence Lum, Olivia<br />
Merkel<br />
712<br />
MicroRNA-155 confers encephalogenic potential to<br />
Th17 cells by promoting effector gene expression<br />
Ruozhen Hu, Thomas B. Huffaker, Dominique A. Kagele, Marah C.<br />
Runtsch, Erin Bake, Aadel A. Chaudhuri, June L. Round, Ryan M.<br />
O’Connell<br />
713<br />
DNA Mini Strings: The Gold Standard for Transgene<br />
Delivery<br />
Naiseh Naissi, Roderick Slavcev<br />
714<br />
Restoring a minimal motor unit potential and<br />
peripheral motor conductivity in lower extremities in<br />
a 15 years old girl with complete loss <strong>of</strong> spinal cord<br />
continuity at Th2-Th3 after combine autologous bone<br />
marrow derived cells and mesenchymal stem cells tr<br />
Jarocha Danuta, Milczarek Olga, Kwiatkowski Stanislaw, Majka<br />
Marcin<br />
715<br />
Genomic correction <strong>of</strong> Duchenne Muscular Dystrophy<br />
patient-derived iPSCs using TALENs<br />
Hongmei Li, Naoko Fujimoto, Noriko Sasakawa, Takashi<br />
Yamamoto, Knut Woltjen, Hidetoshi Sakurai, Shinya Yamanaka,<br />
Akitsu Hotta<br />
122<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013
Abstract Directory, Friday, May 17, 2013<br />
Poster Session II, continued<br />
5:30 PM - 7:30 PM<br />
Room: Exhibit Hall C/D<br />
Late Breaking Abstracts II<br />
716<br />
A versatile method for titration <strong>of</strong> chimeric antigen<br />
receptor-expressing retroviral vectors based on<br />
translocation <strong>of</strong> CD3-epsilon<br />
Daniel Abate-Daga, Steve A. Feldman, Rachel E. Beard, Steven A.<br />
Rosenberg, Richard A. Morgan<br />
717<br />
Dual Speciic Suicide <strong>Gene</strong> Expression plasmid Delivery<br />
Using Bio-reducible Polymer for Hepatocellular<br />
Carcinoma <strong>Gene</strong> <strong>Therapy</strong><br />
Hyun Ah Kim, Kihoon Nam, Minhyung Lee, Sung Wan Kim<br />
723<br />
Attenuation <strong>of</strong> TGF-β induced liver ibrosis by bone<br />
morphogenetic protein 2.<br />
Mu-Rou Tsai, Pao-Rong Chen, Yu-Lin Yang,, Ming-Chuan Hung,<br />
Tao-Chen Lee, Tsuing-Jeu Hung, Chien-Ya Hung, Wen-Teng Chang,<br />
Pei-fang Hsieh Shu-Fen Liu<br />
724<br />
Subpopulation <strong>of</strong> T-lymphocytes in leukoconcentrates<br />
obtained from bone marrow donors in the setting <strong>of</strong><br />
stable hematopoiesis<br />
R. Bogdanov, L. Mendeleeva, I. Galtseva, L. Kuzmina, T. Gaponova,<br />
N. Kalinin, E. Parovichnikova, V. Savchenko<br />
718<br />
Expression <strong>of</strong> the immunomodulatory cytokines<br />
interleukin-10 and interleukin-6 are increased in<br />
equine MSCs cultured in an inlammatory environment<br />
in vitro<br />
KO Ortved, J Witten, AJ Nixon<br />
719<br />
<strong>Gene</strong> Delivery Using Therapeutic Ultrasound To<br />
Mesenchymal Stem <strong>Cell</strong>s: Targeted Antiangiogenic<br />
Tumor <strong>Therapy</strong><br />
Tom Haber, Marcelle Machluf<br />
720<br />
The role <strong>of</strong> S100A4 in glioblastoma and cancer stem<br />
cell<br />
Shih-Hwa Chiou, Jun-Ming Han, Hsin-I Ma<br />
721<br />
Biodistribution and toxicology studies <strong>of</strong> AAV2/rh8-<br />
CBA-β-hexosaminidase after intracranial delivery in<br />
C57/Bl6 mice<br />
Kirsten Erger, Ronald Mandel, William Castleman, Martha<br />
Campbell-Thompson, Corinne Abernathy, Matthew Getz, Travis<br />
Cossette, Lynda Schneider, Ryan Brown, Miguel Sena-Esteves,<br />
Thomas Conlon<br />
Friday, May 17, 2013<br />
722<br />
Qualiication for human umbilical cord derived<br />
mesenchymal stem cells (hUC-MSCs) for regenerative<br />
medicine<br />
Hyun Woo Lee, Su-Youne Han, Hyeon Kyeong Lee, Seung Hoon Pak,<br />
Jin Tac Kim, Ji Hyang Kim, Byung-Rok Do<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013<br />
123
Abstract Directory, Saturday, May 18, 2013<br />
Oral Abstract Session 410<br />
Late Breaking Abstracts<br />
10:15 am - 12:15 pm<br />
Room: 150 DEFG<br />
Oral Abstract Session 411<br />
Adenovirus and Other DNA Virus Vectors<br />
10:15 AM - 12:15 PM<br />
Room: 150 ABC<br />
Saturday, May 18, 2013<br />
725 10:15 AM<br />
AAV/RNAi-mediated improvement <strong>of</strong> anti-malaria<br />
vaccination strategies<br />
Christiane Hammerschmidt-Kamper, Franziska Hentzschel,<br />
Kirsten Heiss, Kathleen Boerner, Ann-Kristin Mueller, Dirk Grimm<br />
726 10:30 AM<br />
Post-entry Restriction <strong>of</strong> AAV Vectors by A PHD Finger<br />
Protein<br />
Claire A. Shreiber, Toshie Sakuma, Yasuhiro Ikeda<br />
727 10:45 AM<br />
AAV-mediated Bevacizumab Maintenance <strong>Therapy</strong><br />
Inhibits Ovarian Cancer In Vivo<br />
Yi Xie, PhD, Martin J. Hicks, PhD, Stephen M. Kaminsky, PhD,<br />
Malcolm A.S. Moore, PhD, Ronald G. Crystal, MD, Arash Raii, MD,<br />
PhD<br />
728 11:00 AM<br />
Human iPS-derived antigen-targeted T cells are<br />
functional innate-like cytotoxic effectors and lyse<br />
CD19-positive tumor cells<br />
Maria Themeli, Christopher C. Kloss, Giovanni Ciriello, Michel<br />
Sadelain<br />
729 11:15 AM<br />
Penton-dodecahedral particles trigger opening <strong>of</strong><br />
intercellular junctions and facilitate viral spread<br />
during adenovirus serotype 3 infection <strong>of</strong> epithelial<br />
cells.<br />
Zhuo-Zhuang Lu, Hongjie Wang, YiYi Zhang, Zongyi Li, Pascal<br />
Fender, Andre Lieber<br />
730 11:30 AM<br />
An oncolytic adenovirus able to express a full-length<br />
functional anti-Her2 monoclonal antibody from tumor<br />
cells<br />
Paula Savola, Ilkka Liikanen, Theresia Gutmann, Kilian Guse and<br />
Akseli Hemminki<br />
731 11:45 AM<br />
Non-toxic and highly eficient targeting <strong>of</strong> a human<br />
chromosomal locus with a lentiviral vector-associated<br />
meganuclease<br />
Chenxia He, Agnès Gouble, Alix Bourdel, Laurent Poirot, Frédéric<br />
Pâques, Philippe Duchateau, Aleksander Edelman, Olivier Danos<br />
732 12:00 PM<br />
Non-viral gene delivery to human embryonic stem cellderived<br />
retinal pigment epithelium<br />
A Subrizi, T Ilmarinen, M Yliperttula, H Tenhu, H Skottman, A Urtti<br />
477 10:15 AM<br />
Molecular Modeling <strong>of</strong> Coagulation FVII Binding to<br />
Adenovirus Hexon Revealed a Binding Pocket for<br />
Coagulation Factor GLA Domain That Deines the<br />
Infectivity <strong>of</strong> Adenovirus-Coagulation Factor Complexes<br />
Eric E. Irons, Justin W. Flatt, Konstantin Doronin, Tara L. Fox,<br />
Mauro Acchione, Phoebe L. Stewart, Dmitry M. Shayakhmetov.<br />
478 10:30 AM<br />
Identiication <strong>of</strong> a Negative Regulator <strong>of</strong> TRPV1 Via an<br />
HSV Based cDNA Library Screen<br />
Bonnie L. Reinhart, Asaff Harel, MingDi Zhang, James R. Goss,<br />
William F. Goins, Justus B. Cohen, Joseph C. Glorioso.<br />
479 10:45 AM<br />
Long Term Follow Up <strong>of</strong> Phase II Clinical Trial <strong>of</strong> a<br />
Granulocyte-Macrophage Colony-Stimulating Factor-<br />
Encoding, Second-<strong>Gene</strong>ration Oncolytic Herpes Virus<br />
(Talimogene Laherparepvec, T-VEC) in Patients with<br />
Unresectable Metastatic Melanoma<br />
John Nemunaitis, Cynthia Bedell, Beena O. Pappen, Staci Horvath,<br />
Neil Senzer.<br />
480 11:00 AM<br />
Combined Therapies with Oncolytic Adenovirus<br />
Suppress Tumor Growth in Hamsters<br />
Brittany A. Young, Karoly Toth, Jacqueline F. Spencer, William S. M.<br />
Wold.<br />
481 11:15 AM<br />
Analysis <strong>of</strong> Host <strong>Cell</strong> and Viral Transcriptomes during<br />
Infection by Wild-Type Species C Ad6 and Species D<br />
Ad26<br />
Mallory A. Turner, Sean E. Hoherr, Michael A. Barry.<br />
482 11:30 AM<br />
Suppression <strong>of</strong> the Leaky Expression <strong>of</strong> Adenovirus<br />
<strong>Gene</strong>s Following Transduction with a Replication-<br />
Incompetent Adenovirus Vector by Incorporating<br />
microRNA-Targeted Sequences into the 3’-Untranslated<br />
Region <strong>of</strong> the E2A, E4, or pIX <strong>Gene</strong><br />
Kahori Shimizu, Fuminori Sakurai, Shin-ichiro Nakamura, Yasuhito<br />
Nagamoto, Kyoko Tomita, Masashi Tachibana, Hiroyuki Mizuguchi.<br />
483 11:45 AM<br />
Biodistribution <strong>of</strong> Fiber-Chimeric Ad5/3 Vectors in<br />
DSG2-Transgenic Mice<br />
Maximillian Richter, Roma Yumul, Hongjie Wang, Dirk Nettelbeck,<br />
Andre Lieber.<br />
124<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013
Abstract Directory, Saturday, May 18, 2013<br />
Oral Abstract Session 411, continued<br />
Adenovirus and Other DNA Virus Vectors<br />
10:15 AM - 12:15 PM<br />
Room: 150 ABC<br />
484 12:00 PM<br />
Ad5-Based Vectors Can Transduce Hepatocytes with<br />
High Eficiency Independent <strong>of</strong> FX<br />
Jan-Michael Prill, Lea Krutzke, Tatjana Engler, Stefan Kochanek,<br />
Florian Kreppel.<br />
Oral Abstract Session 412<br />
Nuclease Mediated Genome Editing<br />
10:15 AM - 12:15 PM<br />
Room: Ballroom A<br />
485 10:15 AM<br />
Cross Your Fingers: A Comprehensive Survey <strong>of</strong> the<br />
Cys2His2 Zinc Finger Domain Hints toward Codes <strong>of</strong><br />
Speciicity and Assembly<br />
Elizabeth F. Rowland, Anton V. Persikov, Benjamin L. Oakes, Mona<br />
Singh, Marcus B. Noyes.<br />
486 10:30 AM<br />
Eficient Site-Speciic Integration and In Situ <strong>Gene</strong><br />
Correction <strong>of</strong> Human Long-Term Repopulating<br />
Hematopoietic Stem <strong>Cell</strong>s by Zinc Finger Nucleases<br />
Pietro Genovese, Angelo Lombardo, Giulia Schiroli, Giulia Escobar,<br />
Tiziano Di Tomaso, Claudia Firrito, Davide Moi, Lucia Sergi Sergi,<br />
Philip D. Gregory, Michael C. Holmes, Luigi Naldini.<br />
487 10:45 AM<br />
Optimizing Plasmid DNA Templates for Homologous<br />
Recombination-Mediated <strong>Gene</strong> Modiication Following<br />
Zinc Finger Nuclease Treatment <strong>of</strong> Hematopoietic Stem<br />
<strong>Cell</strong>s<br />
Colin M. Exline, Orla L. Mulhern, Nick Llewellyn, Jill Henley, Jianbin<br />
Wang, Kenneth Kim, Jenny Yan, Philip D. Gregory, Michael C.<br />
Holmes, Paula M. Cannon.<br />
488 11:00 AM<br />
In Vivo ZFN Mediated Targeting <strong>of</strong> Albumin as a<br />
Platform for Expression <strong>of</strong> Multiple Therapeutic <strong>Gene</strong>s<br />
Rajiv Sharma, Xavier M. Anguela, Yannick Doyon, Sunnie Y. Wong,<br />
David E. Paschon, Hojun Li, Virginia Haurigot, Robert J. Davidson,<br />
Shangzhen Zhou, Philip D. Gregory, Michael C. Holmes, Edward J.<br />
Rebar, Katherine A. High.<br />
489 11:15 AM<br />
Quantifying Rates <strong>of</strong> <strong>Gene</strong> Targeting and Off-Target<br />
Cleavage <strong>of</strong> Engineered Nucleases Using SMRT<br />
Sequencing<br />
Eli J. Fine, Eric Kildebeck, Yanni Lin, Matthew H. Porteus, Thomas<br />
J. Cradick, Gang Bao.<br />
490 11:30 AM<br />
Precise Modiication <strong>of</strong> Human <strong>Gene</strong>s Directed by<br />
Single-Stranded DNA Oligonucleotides and TALENs<br />
Shengdar Q. Tsai, Deepak Reyon, Cyd Khayter, J. Keith Joung.<br />
491 11:45 AM<br />
Eficient Template-Free <strong>Gene</strong>tic Correction with TALE<br />
Nucleases<br />
David G. Ousterout, Pablo Perez-Pinera, Pratiksha I. Thakore,<br />
Ami M. Kabadi, Matthew T. Brown, Kamel Mamchaoui, Jacques P.<br />
Tremblay, Charles A. Gersbach.<br />
492 12:00 PM<br />
Enhancing the Eficiency <strong>of</strong> Zinc Finger Nuclease<br />
Delivery by Integrase-Defective Lentiviral Vectors Using<br />
Valproic Acid<br />
Alok V. Joglekar, Michelle Ho, Libby Stein, Sara Sanadiki, Roger P.<br />
Hollis, Donald B. Kohn.<br />
Oral Abstract Session 413<br />
<strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> <strong>of</strong> Diabetes, Metabolic<br />
and <strong>Gene</strong>tic Diseases II<br />
10:15 AM - 12:15 PM<br />
Room: Ballroom B<br />
494 10:15 AM<br />
Hematopoietic Stem <strong>Cell</strong> <strong>Gene</strong> <strong>Therapy</strong> for Cystinosis<br />
Sarah Ur, Celine J. Rocca, Jay Sharma, Donald B. Kohn, Stephanie<br />
Cherqui.<br />
493 10:30 AM<br />
Mechanism <strong>of</strong> Hematopoietic Stem <strong>Cell</strong>-Mediated<br />
<strong>Therapy</strong> in Cystinosis<br />
Brian A. Yeagy, Jay Sharma, Celine J. Rocca, Sarah Ur, Thomas<br />
Whisenant, Daniel R. Salomon, Stephanie Cherqui.<br />
495 10:45 AM<br />
Prevention <strong>of</strong> Neurocognitive Deicit by Ex Vivo<br />
Lentiviral Transduction <strong>of</strong> Hematopoietic Stem <strong>Cell</strong>s in<br />
a Murine Model <strong>of</strong> Mucopolysaccharidosis Type II<br />
Kelly M. Podetz-Pedersen, Andrea D. Karlen, Megan P. Buckvold,<br />
Zenhong Nan, Vladimir Slepushkin, Andre Roy, Galina Yakovief,<br />
Zi Gong, Joseph Muenzer, Boro Dropulic, Walter C. Low, R. Scott<br />
McIvor.<br />
496 11:00 AM<br />
Hyper-Functional Synthetic Transgenes Improve the<br />
Eficiency <strong>of</strong> Lentiviral and AAV-Based Liver-Directed<br />
<strong>Gene</strong> <strong>Therapy</strong> and Induce Immune Tolerance<br />
Nisha Nair, Alessio Cantore, Mario Di Matteo, Patrizia Della Valle,<br />
Jessica Willems, Hanneke Evens, Armando D’Angelo, Janka Matrai,<br />
Luigi Naldini, Marinee Chuah, Thierry VandenDriessche.<br />
Saturday, May 18, 2013<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013<br />
125
Saturday, May 18, 2013<br />
Abstract Directory, Saturday, May 18, 2013<br />
Oral Abstract Session 413, continued<br />
<strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> <strong>of</strong> Diabetes, Metabolic<br />
and <strong>Gene</strong>tic Diseases II<br />
10:15 AM - 12:15 PM<br />
Room: Ballroom B<br />
497 11:15 AM<br />
Prevention <strong>of</strong> Neuropathology and Normal Cognitive<br />
Development in the Hyperargininemic Mouse with AAV<br />
<strong>Gene</strong> Transfer<br />
Ellen K. Lee, Chuhong Hu, Ragini Bhargava, Ravi Ponnusamy, Hana<br />
Park, Sarah Novic<strong>of</strong>f, Nora Rozengurt, Bart Marescau, Peter De<br />
Deyn, David Stout, Lisa Schlichting, Wayne W. Grody, Stephen D.<br />
Cederbaum, Gerald S. Lipshutz.<br />
498 11:30 AM<br />
Assessing the Safety <strong>of</strong> AAV Liver <strong>Gene</strong> Transfer <strong>of</strong><br />
Hyperfunctional FIX Padua in Inhibitor-Prone Dogs and<br />
Mice<br />
Julie M. Crudele, Jonathan D. Finn, Joshua I. Siner, Yifeng Chen,<br />
Shangzhen Zhou, Glenn P. Niemeyer, Clinton D. Lothrop Jr, Federico<br />
Mingozzi, Katherine A. High, Valder R. Arruda.<br />
499 11:45 AM<br />
Development <strong>of</strong> AAV2/8-Mediated <strong>Gene</strong> <strong>Therapy</strong><br />
Clinical Trial for Crigler-Najjar Syndrome Type I:<br />
Optimization <strong>of</strong> Liver-Speciic Expression Cassette<br />
Nunzia Pastore, Alberto Auricchio, Nicola Brunetti-Pierri.<br />
500 12:00 PM<br />
Stromal <strong>Cell</strong>-Derived Factor-1 Non-Viral DNA <strong>Therapy</strong><br />
Accelerates Wound Healing and Decreases Scar<br />
Formation in Porcine Surgical Incisions<br />
Timothy J. Miller, Karissa Henson, Rahul Aras, Evan Facher, Marc<br />
S. Penn.<br />
Oral Abstract Session 414<br />
Cancer Immunotherapy II<br />
10:15 AM - 12:15 PM<br />
Room: 151 ABCG<br />
501 10:15 AM<br />
DAI-Armed Double Deleted Oncolytic Vaccinia Virus<br />
Displays Enhanced Anti-Tumor Activity by Eliciting a<br />
More Robust Anti-Tumor Immune Response<br />
Mari Hirvinen, Kilian Guse, Marko Ahonen, Eerika Karli, Dario<br />
Greco, Markus Vähä-Koskela, Akseli Hemminki, Vincenzo Cerullo.<br />
502 10:30 AM<br />
Clinical Implications <strong>of</strong> ROR1-Speciic T <strong>Cell</strong>s That<br />
Target B-<strong>Cell</strong> Leukemia<br />
Drew C. Deniger, Thomas H. Kipps, Simon Olivares, Harjeet Singh,<br />
Sourindra Maiti, Lenka V. Hurton, Kirsten C. Switzer, Tiejuan Mi,<br />
Radhika Thokala, Helen Huls, William G. Wierda, Laurence J. N.<br />
Cooper.<br />
503 10:45 AM<br />
Randomized Phase II Trial <strong>of</strong> Adjuvant Autologous<br />
Tumor <strong>Cell</strong> Vaccine (FANG) for High Risk Stage III/IV<br />
Ovarian Cancer: Preliminary Results<br />
Neil Senzer, Minal Barve, Cynthia Bedell, Jonathan Oh, Joseph<br />
Kuhn, Bruce Fine, Thomas P. Heffernan, Carolyn M. Matthews, C.<br />
Allen Stringer, E. Colin Koon, Samuel Lifshitz, Beena O. Pappen,<br />
Padmasini Kumar, Phillip B. Maples, John Nemunaitis.<br />
504 11:00 AM<br />
TCR <strong>Gene</strong> Editing To Treat Acute Leukemia and<br />
Multiple Myeloma<br />
Sara Mastaglio, Pietro Genovese, Zulma Magnani, Elena Provasi,<br />
Angelo Lombardo, Andreas Reik, Nicoletta Cieri, Aurora Brambilla,<br />
Fabio Ciceri, Claudio Bordignon, Michael C. Holmes, Philip D.<br />
Gregory, Luigi Naldini, Chiara Bonini.<br />
505 11:15 AM<br />
Novel VSV-Based Immunotherapy for Brain Tumors<br />
Rosa Diaz, Diana Rommelfanger-Konkol, Timothy Kottke, Jill<br />
Thompson, Oliver Donnelly, Addie Embry, Alan Melcher, Richard<br />
Vile.<br />
506 11:30 AM<br />
CD19-Targeted T <strong>Cell</strong>s Rapidly Induce Molecular<br />
Remissions in Adults with Chemotherapy Refractory<br />
Acute Leukemia<br />
Renier Brentjens, Marco L. Davila, Isabelle Riviere, Jae Park,<br />
Xiuyan Wang, Shirley Bartido, Jolanta Stefankski, Oriana Borquez-<br />
Ojeda, Yvette Bernal, Michel Sadelain.<br />
507 11:45 AM<br />
A Vaccinia Virus Armed with Interleukin-10 That<br />
Dampens Host Immunity to the Vctor but Not to Tumor<br />
Is a Promising Therapeutic Agent for Pancreatic Cancer<br />
Louisa S. Chard, Eleni Maniati, Pengju Wang, Jonathan Hughes,<br />
Jiwei Wang, Bela Denes, Istvan Fodor, Thorsten Hagemann,<br />
Nicholas R. Lemoine, Yaohe Wang.<br />
508 12:00 PM<br />
Identiication <strong>of</strong> an Alternative Speciicity for<br />
Engineered T <strong>Cell</strong>s Expressing an Enhanced Afinity<br />
MAGE A3 TCR<br />
Namir J. Hassan, Brian Cameron, Andrew Gerry, Joseph Dukes,<br />
Jane Harper, Vivekanandan Kannan, Frayne Bianchi, Francis<br />
Grand, Giovanna Bossi, Joanna Brewer, Debbie Sutton, Annelise<br />
Vuidepot, Alex Powlesland, Alison Legg, Katherine Adams, Alan<br />
Bennett, Nick Pumphrey, Gwen Binder-Scholl, Irina Kulikovskaya,<br />
Minnal Gupta, Gabriela Plesa, Michael Kalos, Bruce L. Levine,<br />
James L. Riley, Angel Varela-Rohena, Edward A. Stadtmauer, Aaron<br />
P. Rapoport, Gerald P. Linette, Carl H. June, Bent Jakobsen.<br />
126<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013
Abstract Directory, Saturday, May 18, 2013<br />
Oral Abstract Session 415<br />
Oligonucleotide & RNAi Therapeutics<br />
10:15 AM - 12:15 PM<br />
Room: Ballroom C<br />
509 10:15 AM<br />
Targeted, Multifunctional RNA Therapies for Advanced<br />
Cancers <strong>of</strong> the Prostate<br />
Luiza I. Hernandez, William M. Rockey, Craig A. Howell, Justin P.<br />
Dassie, Frank J. Hernandez, Xiu Y. Liu, Paloma H. Giangrande.<br />
510 10:30 AM<br />
Circulating Plasmid Detection in a Phase I Clinical Trial<br />
<strong>of</strong> Intratumoral Injection <strong>of</strong> pbi-shRNA Nanoplex<br />
Christopher M. Jay, Donald D. Rao, Zhaohui Wang, Neil Senzer,<br />
Phillip B. Maples, John Nemunaitis.<br />
511 10:45 AM<br />
Smooth Muscle <strong>Cell</strong> Targeted RNA Aptamers for the<br />
Treatment <strong>of</strong> Vascular Disease<br />
William H. Thiel, Maysam Takapoo, Shuxia Jiang, Jennifer<br />
Streeter, Bojana Stanic, Xiuying Liu, Francis J. Miller, Jr, Paloma H.<br />
Giangrande.<br />
512 11:00 AM<br />
Long-Term Inhibition <strong>of</strong> miR-122 Signiicantly<br />
Increases Hepatocellular Carcinoma in Multiple Adult<br />
Mouse Models<br />
Jun Xie, Qin Mao, Jia Li, Xia<strong>of</strong>ang Yang, Qin Su, Ran He, Zhong Jiang,<br />
Xia<strong>of</strong>ei Wang, Phillip D. Zamore, Guangping Gao.<br />
513 11:15 AM<br />
In Vitro and In Vivo Effects <strong>of</strong> Composite Calcium<br />
Phosphate and Poly(Lactic-Co-Glycolic Acid) (PLGA)<br />
Nanoparticles Engineered for Enhanced Cancer <strong>Cell</strong><br />
Internalization and Sustained siRNA Release<br />
Lisa A. Tobin, Yili Xie, Su I. Chung, King F. Kwong.<br />
514 11:30 AM<br />
Robust RNAi Enhancement Via Human Argonaute-2<br />
Overexpression from Plasmids, Viral Vectors or <strong>Cell</strong><br />
Lines<br />
Kathleen Boerner, Dominik Niopek, Daniel Gilbert, Konrad Streetz,<br />
Hans-Georg Kraeusslich, Dirk Grimm.<br />
515 11:45 AM<br />
Ultra-Stable Trivalent RNA Nanoparticles for Targeting<br />
to Cancers and HIV-1<br />
Kato Shum, Peixuan Guo, John J. Rossi.<br />
516 12:00 PM<br />
Directed Evolution-Based Dissection <strong>of</strong> the Four Human<br />
Argonaute Proteins<br />
Nina Schuermann, Leonardo Trabucco, Christian Bender, Rob<br />
Russell, Dirk Grimm.<br />
Poster Session III<br />
3:45 PM - 5:45 PM<br />
Room: Exhibit Hall C/D<br />
RNA Virus Vectors<br />
517<br />
Data Mining Methods for Integration Site Analysis in<br />
Hematopoietic Stem <strong>Cell</strong> <strong>Gene</strong> <strong>Therapy</strong> Trials<br />
Andrea Calabria, Luca Biasco, Fabrizio Benedicenti, Simone Leo,<br />
Martina Cesani, Gianluigi Zanetti, Elia Stupka, Alessandra Bifi,<br />
Alessandro Aiuti, Luigi Naldini, Eugenio Montini.<br />
518<br />
Mutually Dependent Barcode Strategy for Accurate<br />
Deep Sequencing <strong>of</strong> Vector Integration Sites<br />
Richard Gabriel, Cynthia C. Bartholomae, Frank Giordano, Raffaele<br />
Fronza, Stephanie Laufs, Christ<strong>of</strong> von Kalle, Manfred Schmidt.<br />
519<br />
Sequencing <strong>of</strong> a <strong>Gene</strong>tic Barcode Reveals Altered<br />
Population Dynamics in a Mouse Model <strong>of</strong> Transplanted<br />
Hematopoietic Stem <strong>Cell</strong>s Transduced with a Lentivirus<br />
Encoding LMO2<br />
Bryan E. Strauss, Daniela B. Zanatta, Rodrigo Aguiar, Maristela<br />
Tsujita, Primavera Borelli.<br />
520<br />
A New Strategy for Retroviral Vector Puriication Based<br />
on LNGR-Ab Immunomagnetic Selection<br />
Stornaiuolo Anna, Corna Stefano, Bossi Sergio, Bordignon Claudio,<br />
Rizzardi Gian Paolo, Bovolenta Chiara.<br />
521<br />
Lentiviral <strong>Gene</strong> <strong>Therapy</strong> in a SCID-X1 Mouse Model<br />
after Treatment with Granulocyte Colony Stimulating<br />
Factor<br />
Marshall W. Huston, Niek P. van Til, Rana Yadak, Yvette van<br />
Helsdingen, Gerard Wagemaker.<br />
522<br />
Development <strong>of</strong> Human Mesenchymal Stem <strong>Cell</strong>-Based<br />
Tumor-Homing Producer <strong>Cell</strong>s for Eficient Delivery <strong>of</strong><br />
Retroviral Replicating Vectors to Brain Tumors<br />
Shuichi Kamijima, Akihito Inagaki, Kei Hiraoka, Masamichi<br />
Takahashi, Quincy Tam, Emmanuelle Faure-Kumar, Janet Treger,<br />
Brooke Bogan, Christopher R. Logg, Noriyuki Kasahara.<br />
523<br />
Reducing In Vivo Clearance <strong>of</strong> Lentiviral Vectors by<br />
Immune <strong>Cell</strong>s in order To Improve Eficacy and Safety<br />
<strong>of</strong> <strong>Gene</strong> <strong>Therapy</strong><br />
Nisha G. Sosale, Irena I. Ivanovska, Richard K. Tsai, Philip K.<br />
Zoltick, Yale Goldman, Dennis E. Discher.<br />
524<br />
A Novel Retroviral Envelope as a Candidate for<br />
Pseudotyping Retroviral and Lentiviral Vectors<br />
Wenqin Xu, Jill Russ, Kristen Gorman, Kyle Delaney, Maribeth V.<br />
Eiden.<br />
Saturday, May 18, 2013<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013<br />
127
Abstract Directory, Saturday, May 18, 2013<br />
Poster Session III, continued<br />
3:45 PM - 5:45 PM<br />
Room: Exhibit Hall C/D<br />
RNA Virus Vectors<br />
525<br />
A Novel Shuttle Vector Approach To Identify <strong>Gene</strong>s<br />
Involved in Hematopoietic Stem <strong>Cell</strong> Disorders Using<br />
Vector-Mediated Genotoxicity<br />
Dustin T. Rae, Ryan Maynard, H. Joachim Deeg, Grant D. Trobridge.<br />
526<br />
A Systematic Approach to the Establishment and<br />
Characterisation <strong>of</strong> Endothelial Progenitor <strong>Cell</strong>s for<br />
<strong>Gene</strong> <strong>Therapy</strong><br />
Yuan Zhao, Natalie Werling, Robin Thorpe.<br />
527<br />
Lentiviral Vector Transduction <strong>of</strong> Human CD34+ <strong>Cell</strong>s<br />
Requires a Wash/Rest Phase between Vector Exposures<br />
To Produce Additional Beneit but Does Not Require<br />
High Dose Cytokines<br />
Michael R. Greene, Brian P. Sorrentino.<br />
528<br />
Phorbol 12-Myristate 13-Acetate Enhances Lentiviral<br />
Transduction <strong>of</strong> Human Hematopoietic <strong>Cell</strong>s through<br />
Activation PKC<br />
Jennifer M. Johnston, Gabriela Denning, Christopher B. Doering, H.<br />
Trent Spencer.<br />
529<br />
Production <strong>of</strong> Recombinant Lentiviral Vectors<br />
in Serum-Free Suspension Culture and Column<br />
Chromatography Puriication<br />
Yonghai Li, Bernd Hauck, Tiffany Kincaid, Fraser J. Wright,<br />
Katherine A. High, Guang Qu.<br />
533<br />
Lentiviral Vectors-Mediated Long-Term and High<br />
Eficiency Expression <strong>of</strong> Foreign <strong>Gene</strong> in HEK 293T <strong>Cell</strong>s<br />
Renhe Yan, Yingying Mao, Zhibing Liang, Yi Feng, Tianbai Li,<br />
Maomin Lu, Weiwang Gu, Jingang Zhang, Hongwei Li.<br />
534<br />
Tumorigenic Effect <strong>of</strong> Transplanted Myoblasts<br />
Overexpressing Bcl-2 Based on Retroviral Vector<br />
Francisco Martinez-F, Maria Ustoa, Araceli Barrera-L, Hugo<br />
E. Sandoval-Zamora, Bruno Guevara, Pablo Vizcaino-Dorado,<br />
Alejandro Zentella-Dehesa, Catalina Machuca-R.<br />
535<br />
Transduction <strong>of</strong> Ferret Airways with Avian Inluenza<br />
Virus Hemagglutinin Pseudotyped Equine Infectious<br />
Anemia Virus Vector<br />
Ziying Yan, Manij Patel, Xingshen Sun, Diana CM - Lei-butters,<br />
Hongshu Sui, John C. Olsen, John F. Engelhardt.<br />
AAV Vectors III<br />
536<br />
Multiple Molecular Alterations in Phosphodegrons 1-3<br />
within AAV2 Capsid Demonstrates Higher Hepatic <strong>Gene</strong><br />
Transfer Eficiency<br />
Dwaipayan Sen, V. Kalaivani, Rupali Gadkari, G. Sudha, N.<br />
Srinivasan, Alok Srivastava, Giridhara R Jayandharan.<br />
537<br />
Improved Adeno-Associated Virus Serotype (AAV)5<br />
Vectors for Hepatic <strong>Gene</strong> <strong>Therapy</strong><br />
Dwaipayan Sen, Prachi Agarwal, V. Kalaivani, Alok Srivastava,<br />
Giridhara R Jayandharan.<br />
538<br />
Adeno Associated Virus Puriication Independent <strong>of</strong><br />
Gradient Centrifugation and Chromatography<br />
Erik Arden, Joseph M. Metzger.<br />
Saturday, May 18, 2013<br />
530<br />
Eficient Selection <strong>of</strong> <strong>Gene</strong>tically Engineered HIV<br />
Resistant <strong>Cell</strong>s by Short Hairpin RNA Mediated HPRT<br />
and CCR5 Knockdown<br />
Munetoshi Narukawa, Saki Shimizu, Katrin Hacke, Janet Treger,<br />
Ruth Cortado, Erica Egger, Noriyuki Kasahara, Dong Sung An.<br />
531<br />
Designing Trans-Regulated Lentiviral Vectors for <strong>Gene</strong><br />
Transfer in Functional Genomics and <strong>Gene</strong> <strong>Therapy</strong><br />
Agnes Holczbauer, Adriana Zingone, Suresh Arya.<br />
532<br />
Application <strong>of</strong> VSV-G Induced Nanovesicles (Gesicles)<br />
for the Delivery <strong>of</strong> Active Protein To Target <strong>Cell</strong>s<br />
Thomas P. Quinn, Lily Lee, Mei Fong, Michael Haugwitz, Hiroaki<br />
Sagawa, Andrew Farmer.<br />
539<br />
Transcriptional Repression with Zinc-Finger and Tale<br />
Protein Scaffold<br />
Salvatore Botta, Elena Marrocco, Nicola de Prisco, Toni Cathomen,<br />
Claudio Mussolino, Enrico Maria Surace.<br />
540<br />
Mouse Gender Inluences Brain Expression <strong>of</strong> a<br />
Reporter <strong>Gene</strong> by Intravascularly-Administered AAV9<br />
Casey A. Maguire, Matheus H. W. Crommentuijn, Dakai Mu, Eloise<br />
Hudry, Alberto Serrano-Pozo, Bradley Hyman, Bakhos A. Tannous.<br />
541<br />
Site-Directed Mutagenesis <strong>of</strong> Surface-Exposed<br />
Lysine Residues Leads to Improved Transduction<br />
by Recombinant AAV2 and AAV8 Vectors in Murine<br />
Hepatocytes In Vivo<br />
Baozheng Li, Wenqin Ma, George Aslanidi, Chen Ling, Kim Van<br />
Vliet, Mavis Agbandje-McKenna, Arun Srivastava.<br />
128<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013
Abstract Directory, Saturday, May 18, 2013<br />
Poster Session III, continued<br />
3:45 PM - 5:45 PM<br />
Room: Exhibit Hall C/D<br />
AAV Vectors III<br />
542<br />
Transduction <strong>of</strong> Murine, Nonhuman Primate,<br />
and Human Hematopoietic Stem <strong>Cell</strong>s (HSCs) by<br />
Recombinant AAV Serotype Vectors: Identiication <strong>of</strong><br />
AAV6 as the Most Eficient Serotype for Human HSCs,<br />
and Further Augmentation in Transduction Eficiency<br />
In Vitro and In Vivo with Point-Mutations <strong>of</strong> Tyrosine<br />
Residues in the Viral Capsid<br />
Liujiang Song, M. Ariel Kauss, Etana Kopin, Manasa Chandra,<br />
Tahira Ul-Hasan, Erin Miller, Giridhara R. Jayandharan, Angela<br />
E. Rivers, George V. Aslanidi, Chen Ling, Baozheng Li, Wenqin<br />
Ma, Xiaomiao Li, Lourdes M. Andino, Li Zhong, Alice F. Tarantal,<br />
Mervin C. Yoder, Kamehameha K. Wong, Jr, Menqun Tan, Saswati<br />
Chatterjee, Arun Srivastava.<br />
543<br />
Eficient Transduction <strong>of</strong> Canine Muscle Using rAAV6<br />
Jane T. Seto, Julian N. Ramos, James M. Allen, Eric Finn, Guy L.<br />
Odom, Jeffrey S. Chamberlain.<br />
544<br />
Integrated Transcriptional and Post Transcriptional<br />
Targeting Results in Cardiac Biased Transgene<br />
Expression<br />
Jinhui Wang, Joshua Gorsky, Jonathan Peterson, Joseph<br />
Rabinowitz.<br />
545<br />
Reprogramming Adipose Tissue Derived Mesenchymal<br />
Stem <strong>Cell</strong>s (AT-MSC) into Pluripotent Stem <strong>Cell</strong>s by a<br />
Mutant AAV Vector<br />
Mong-Jen Chen, Yuanqing Lu, Takashi Hamazaki, Hsin-Yin Tsai,<br />
Kirsten Erger, Thomas Conlon, Arun Srivastava, Mark Brantly,<br />
Vince Chiodo, William Huaswirth, Naohiro Terada, Sihong Song.<br />
546<br />
Optimized Kidney-Targeted <strong>Gene</strong> Delivery Using AAV in<br />
a Mouse Model <strong>of</strong> Cystinosis<br />
Celine J. Rocca, Sarah M. Ur, Corinne Antignac, Jude R. Samulski,<br />
Stephanie Cherqui.<br />
547<br />
Concentration <strong>of</strong> AAV8 and 9 Vector to Titers Ranging<br />
from 1-6 x1014 vg/mL: Feasibility Assessment for<br />
Volume-Limited Dosing<br />
J. Fraser Wright, Bernd Hauck, Shang-Zhen Zhou, Olga Zelenaia,<br />
Marina Sumaroka, Katherine A. High.<br />
548<br />
Enhanced Fracture Healing Utilizing Directed Evolution<br />
<strong>of</strong> Adeno-Associated Virus<br />
Seth G. Levy, Damien Marsic, Shawn Gilbert, Sergei Zolotukhin,<br />
Selvarangan Ponnazhagan.<br />
549<br />
Targeted Bio-Engineering <strong>of</strong> Adeno Associated Virus<br />
Serotype rh.10 Capsid Improves Its <strong>Gene</strong> Delivery In<br />
Vitro and In Vivo<br />
Ruchita Selot, Nishanth Gabriel, Monika Kumari, Giridhara R<br />
Jayandharan.<br />
550<br />
Adeno-Associated Virus Vectors Containing Either<br />
Elongation Factor 1 alpha or Human Synapsin 1<br />
Promoters Eficiently Drive Expression in Rodent<br />
Primary Cortical Neurons<br />
Thomas G. Wolfe, Yun Lin, Holly Carlisle, Danny Ortuno, Huiren<br />
Zhao, Yun Hu, Yu Sun, Cherylene Plewa, Paul Kassner, Ki Jeong Lee.<br />
551<br />
Highly Flexible, Precise and Eficient <strong>Gene</strong> Editing <strong>of</strong><br />
Diverse Human Tissue Types Using rAAV-Mediated<br />
Homologous Recombination<br />
Ramu Mangena, Tom Henley, Christine Schoield, Chris Torrance,<br />
Eric Rhodes.<br />
Adenovirus and Other DNA Virus Vectors II<br />
552<br />
Structural Studies on the Interaction <strong>of</strong> Adenovirus<br />
Serotype 3 Fiber Knob and Desmoglein 2<br />
Hongjie Wang, Roma Yumul, Maximillian Richter, Pascal Fender,<br />
Andre Lieber.<br />
553<br />
Blocking <strong>of</strong> Scavenger-Receptor A Results in Increased<br />
HDAd-Mediated Hepatocyte Transduction through<br />
Inhibition <strong>of</strong> Vector Particle Uptake by Kupffer <strong>Cell</strong>s<br />
and Liver Sinusoidal Endothelial <strong>Cell</strong>s<br />
Pasquale Piccolo, Pratibha Mithbaokar, Nicola Brunetti-Pierri.<br />
554<br />
Artiicial Riboswitches for External Regulation <strong>of</strong><br />
Oncolytic Viruses<br />
Patrick Ketzer, Johanna K. Kaufmann, Sarah Engelhardt, Sascha<br />
Bossow, Jörg S. Hartig, Guy Ungerechts, Dirk M. Nettelbeck.<br />
555<br />
Liver-Detargeted Ad5/48 Chimaeric Hexon Based<br />
Oncolytic Adenoviruses Targeting TGFb Signaling:<br />
A Safe and Effective Approach for the Treatment <strong>of</strong><br />
Prostate Cancer Bone Metastases<br />
Weidong Xu, Zhenwei Zhang, Zebin Hu, Stuart Stock, Charles<br />
Brendler, Prem Seth.<br />
Saturday, May 18, 2013<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013<br />
129
Abstract Directory, Saturday, May 18, 2013<br />
Saturday, May 18, 2013<br />
Poster Session III, continued<br />
3:45 PM - 5:45 PM<br />
Room: Exhibit Hall C/D<br />
Adenovirus and Other DNA Virus Vectors II<br />
556<br />
Enhanced Anti-Tumor Eficacy by Oncolytic Adenovirus<br />
Using a Stimuli Responsive Block Copolymer<br />
mPEG-b-Poly(His)<br />
Joung-Woo Choi, You Han Bae, Chae-Ok Yun.<br />
557<br />
Distinct Integration Proiles after Sleeping Beauty<br />
Transposase-Mediated Somatic Integration from AAV<br />
and Adenoviral Vectors<br />
Wenli Zhang, Christina Rauschhuber, Martin Haeusl, Nadine<br />
Muether, Anja Ehrhardt.<br />
558<br />
Screening and Characterization <strong>of</strong> Alternative<br />
Adenovirus Serotypes with Enhanced Virus-<br />
Lateralization Ability In Vitro for Improved Antitumor<br />
Eficacy<br />
Junji Uchino, David T. Curiel, Hideyo Ugai.<br />
559<br />
Improved Animal Models for Testing <strong>Gene</strong> or <strong>Cell</strong><br />
Therapies for Atherosclerosis<br />
Liang Du, Jingwan Zhang, David Dichek.<br />
560<br />
Liver Retargeting <strong>of</strong> Adenovirus Using Robo4 Promoter<br />
and Hexon Substitution<br />
Sergey A. Kaliberov, Lyudmila N. Kaliberova, Zhi Hong Lu,<br />
Meredith A. Preuss, Justin A. Barnes, Cecil R. Stockard, William E.<br />
Grizzle, Jeffrey M. Arbeit, David T. Curiel.<br />
561 (Moved to Poster Session I after Abstract 138)<br />
Lister Strain Vaccinia Virus Demonstrates Favourable<br />
Anti-Tumor Eficacy and Tumor-Selectivity Compared<br />
to Western Reserve Strain<br />
Jonathan Hughes, Pengju Wang, Ghassan Alusi, Vipul Bhakta,<br />
Yongchao Chu, Hailiang Shi, Iain McNeish, Andrea McCart, Istvan<br />
Fodor, Nicholas Lemoine, Yaohe Wang.<br />
562<br />
Development <strong>of</strong> a Novel Vaccine Vector Based on<br />
Synthesized Adenovirus Genome<br />
Yong Yang, Chao Zhang, Xiang Wang, Yudan Chi, Dongming Zhou.<br />
563<br />
Construction <strong>of</strong> a Novel Bicistronic Adenoviral Vector<br />
Feilong Jie, Shengyao Wang, Jie Luo, Na Zhang, Jingang Zhang,<br />
Hongwei Li.<br />
564<br />
Ciproloxacin Regulates Negatively Egr-1<br />
Transcriptional Activity in Human Primary Tenocytes<br />
Transduced with Ad-Egr-1/Luc<br />
Francisco Martinez-F, Araceli Barrera-Lopez, Hugo Sandoval-<br />
Zamora, Karina Guzman-M, Alejandro Jimenez-Orozco, Christian Y.<br />
Lomeli-R, David T. Curiel, Rebecca E. Franco-Bourland.<br />
DNA Vectorology & <strong>Gene</strong> Targeting II<br />
565<br />
A Placebo Controlled Phase IIb Clinical Study<strong>of</strong><br />
Tissue<strong>Gene</strong>-C (TG-C) in Patients with Osteoarthritis<br />
Jung Jong Cho, Tae Won Kim, Yeo Myeong Park, Eu <strong>Gene</strong> Jeong,<br />
Moon Jong Noh, Kwan Hee Lee, Bum Sup Lee.<br />
566<br />
A Novel Oncolytic HSV Design Based on the Common<br />
Over-Expression <strong>of</strong> miR-21 in Tumors<br />
Marco Marzulli, Lucia Mazzacurati, Bonnie Reinhart, Mark E.<br />
Hatley, Joseph C. Glorioso, Paola Grandi, Justus B. Cohen.<br />
567<br />
Comparing the Activity <strong>of</strong> TALENs Constructed with<br />
Different Guanine-Targeting RVDs<br />
Yanni Lin, Thomas J. Cradick, Eli J. Fine, Gang Bao.<br />
568<br />
In Vivo Cleavage <strong>of</strong> Transgene Donors Promotes<br />
Nuclease-Mediated Targeted Integration<br />
Sandra Cristea, Yevgeniy Freyvert, Yolanda Santiago, Michael C.<br />
Holmes, Fyodor D. Urnov, Philip D. Gregory, Gregory J. Cost.<br />
569<br />
Helper Independent Self-Inactivating Chimeric<br />
PiggyBac Transposases for Genomic Targeting in<br />
Human <strong>Cell</strong>s<br />
Jesse B. Owens, Johann Urschitz, Ilko Stoytchev, Stefan Moisyadi.<br />
570<br />
Eficacy with Extremely Low Doses <strong>of</strong> Plasmid DNA<br />
Using Small Molecule Targeted Delivery <strong>of</strong> Bilamellar<br />
Invaginated Vesicles (BIVs) to Melanoma Tumor<br />
Xenografts<br />
Nancy S. Templeton, Li Chen, Connor Phalon, Phillip B. Maples,<br />
Neil Senzer, John Nemunaitis.<br />
571<br />
Zinc Finger Nuclease-Based <strong>Gene</strong> <strong>Therapy</strong> for Arginase<br />
I Deiciency as a Model for Curing Single-Enzyme Urea<br />
Cycle Disorders<br />
Ragini Bhargava, Hana Park, Sarah Novic<strong>of</strong>f, Alok V. Joglekar,<br />
Donald B. Kohn, Gerald S. Lipshutz.<br />
572<br />
Poly(2-Dimethylaminoethyl Methacrylate)-<br />
Functionalized Graphene Oxide Nanolakes for Eficient<br />
siRNA Delivery<br />
Jung Eun Lee, Min Sang Lee, Jeong A. Nam, Nak Won Kim, Sung<br />
Young Park, Ji Hoon Jeong.<br />
130<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013
Abstract Directory, Saturday, May 18, 2013<br />
Poster Session III, continued<br />
3:45 PM - 5:45 PM<br />
Room: Exhibit Hall C/D<br />
DNA Vectorology & <strong>Gene</strong> Targeting II<br />
573<br />
Eficient Delivery DNA Vaccine for Alzheimer’s Disease<br />
by Triggered Release <strong>of</strong> Polyplexes from Microneedles<br />
Kim Nak Won, Lee Kyuri, Lee Min Sang, Jeong Ji Hoon.<br />
574<br />
Improved Lentiviral Formats for Genome Editing Using<br />
Zinc Finger Nucleases<br />
Monika Gjoka, Kevin Kang, Yuping Huang, Gregory D. Davis.<br />
575<br />
CFTR Reporter Construct for Optimization <strong>of</strong> Airway<br />
Epithelium Differentiation<br />
Myra E. Gordon, Alaleh Esmaeili-Shandiz, Shingo Suzuki, Roy G.<br />
Sargent, Dieter C. Gruenert.<br />
576<br />
Non-Viral <strong>Gene</strong> Delivery to Oral Squamous <strong>Cell</strong><br />
Carcinoma and Cervical Carcinoma <strong>Cell</strong>s and Suicide<br />
<strong>Gene</strong> <strong>Therapy</strong><br />
Nejat Düzgünes, Aruna Singh, Senait Gebremedhin, Krystyna<br />
Konopka.<br />
577<br />
Orphan Product Development for DNA Therapeutics<br />
Gladice Wallraven, Phillip B. Maples, John Nemunaitis.<br />
<strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> <strong>of</strong> Diabetes, Metabolic<br />
and <strong>Gene</strong>tic Diseases II<br />
578<br />
Development <strong>of</strong> Lentiviral <strong>Gene</strong> <strong>Therapy</strong> for Type VII<br />
Collagen Deicient Epidermolysis Bullosa<br />
Christos Georgiadis, Ekaterina Semenova, Andy P. South, Adrian<br />
Thrasher, John A. McGrath, Wei-Li Di, Waseem Qasim.<br />
579<br />
Reprogramming <strong>of</strong> Murine Gallbladder <strong>Cell</strong>s towards<br />
the Pancreatic b-<strong>Cell</strong> Fate In Vitro<br />
Yuhan Wang, Ray D. Hickey, Markus Grompe.<br />
580<br />
Fetal <strong>Gene</strong> <strong>Therapy</strong> at Early and Late Gestation:<br />
Analysis <strong>of</strong> Outcomes and Postnatal Vector Challenge in<br />
a Macaque Model<br />
Citra Mattar, Cecilia Rosales, Rowell Fragata, Nuryanti Johana,<br />
Simon Waddington, Arijit Biswas, Mahesh Choolani, Amit<br />
Nathwani, Jerry Chan.<br />
581<br />
Factors Inluencing Direct Reprogramming <strong>of</strong> Primary<br />
Human Extrahepatic Biliary <strong>Cell</strong>s into Pancreatic beta<br />
<strong>Cell</strong>s<br />
Feorillo Galivo, Eric Benedetti, Craig Dorrell, Olga Smirnova,<br />
Jonathan Schug, Klaus Kaestner, Markus Grompe.<br />
582<br />
Mesenchymal Stem <strong>Cell</strong>s Do Not Prevent Alloantibody<br />
Response Against IDUA Used To Treat MPSI<br />
Priscila K. Matsumoto, Roberta S. Stilhano, Flavia H. da Silva,<br />
Vivian Y. Samoto, Giovani B. Perez, Sang W. Han.<br />
583<br />
Comparison <strong>of</strong> <strong>Gene</strong> <strong>Therapy</strong> Vectors in a New<br />
Hypomorphic Model <strong>of</strong> Propionic Acidemia<br />
Adam J. Guenzel, Sean E. Hoherr, Matthew Hillestad, Mary Barry,<br />
Eric Weaver, Jan P. Kraus, Dietrich Matern, Michael A. Barry.<br />
584<br />
Transcription Activator-Like Effector Nuclease-Based<br />
Genome Editing for Mucopolysaccharidosis Type I<br />
Mark J. Osborn, Colby G. Starker, Anthony P. DeFeo, Daniel F.<br />
Voytas, Bruce R. Blazar, Jakub Tolar.<br />
585<br />
<strong>Gene</strong> <strong>Therapy</strong> for Mouse Model <strong>of</strong> Krabbe Disease<br />
Hiroshi Kobayashi, Masamichi Ariga, Takahiro Fukuda, Takeo<br />
Iwamoto, Sayoko Izuka, Yota Shimada, Yoshikatsu Eto, Toya<br />
Ohashi.<br />
586<br />
Choroid Plexus-Directed Viral <strong>Gene</strong> <strong>Therapy</strong> as a<br />
Source <strong>of</strong> alpha-N-Acetyl-Glucosaminidase Enzyme<br />
Replacement in Cerebrospinal Fluid for Sanilippo B<br />
Syndrome<br />
Patricia I. Dickson, Shih-hsin Kan, Steven Q. Le, Maria Reina<br />
Haddad, Eun-Young Choi, Anthony Donsante, Stephen G. Kaler.<br />
587<br />
Mitigation <strong>of</strong> DNA Damage Responses and Oxidative<br />
Stress in <strong>Cell</strong>s with Impaired Telomerase Function<br />
Larisa Pereboeva, Erik Westin, Mary Gannon, Lawrence Lamb,<br />
Aloysius Klingelhutz, Frederick Goldman.<br />
588 Abstract Withdrawn<br />
Neurologic <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> II<br />
589<br />
CNS Administration <strong>of</strong> an AAVrh.10 <strong>Gene</strong> Transfer<br />
Vector Expressing the Human ARSA cDNA to Nonhuman<br />
Primates Provides Widespread Activity across the CNS<br />
Jonathan B. Rosenberg, Dolan Sondhi, David G. Rubin, Sébastien<br />
Monette, Alvin Chen, Sara A. Cram, Benjamin Van de Graaf, Bishnu<br />
P. De, Stephen M. Kaminsky, Caroline Sevin, Patrick Aubourg,<br />
Ronald G. Crystal.<br />
Saturday, May 18, 2013<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013<br />
131
Abstract Directory, Saturday, May 18, 2013<br />
Saturday, May 18, 2013<br />
Poster Session III, continued<br />
3:45 PM - 5:45 PM<br />
Room: Exhibit Hall C/D<br />
Neurologic <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> II<br />
590 (Moved to Poster Session II after Abstract 391)<br />
Intracranial AAV <strong>Gene</strong> <strong>Therapy</strong> Extends the Life Span<br />
<strong>of</strong> GM2 Gangliosidosis Cats >Four-Fold with No Clinical<br />
Evidence <strong>of</strong> Vector Toxicity<br />
Allison M. Bradbury, Victoria J. McCurdy, Aime K. Johnson, Heather<br />
Gray-Edwards, Brandon L. Brunson, Ashley N. Randle, Nancy R.<br />
Cox, Miguel Sena-Esteves, Douglas R. Martin.<br />
591<br />
Prophylactic and Therapeutic Treatment <strong>of</strong> Pain<br />
in a Varicella- Zoster Virus (VZV) Rodent Model <strong>of</strong><br />
Post-Herpetic Neuralgia (PHN) Using an HSV Vector<br />
Expressing Enkephalin<br />
William F. Goins, Jean-Marc Guedon, Mingdi Zhang, Joseph C.<br />
Glorioso, Paul R. Kinchington.<br />
592<br />
AAV <strong>Gene</strong> <strong>Therapy</strong> Normalizes Disease Phenotype<br />
and Extends Life Span >Four-Fold in Feline GM1<br />
Gangliosidosis<br />
Aime K. Johnson, Victoria J. McCurdy, Allison M. Bradbury, Heather<br />
Gray-Edwards, Ashley N. Randle, Misako Hwang, Nancy R. Cox,<br />
Stanley G. Leroy, Miguel Sena-Esteves, Douglas R. Martin.<br />
593<br />
MECP2e1 Is<strong>of</strong>orm-Speciic Lentivirus Rescues Rett<br />
Syndrome Neuronal Soma Size Defects in a <strong>Cell</strong><br />
Autonomous Manner<br />
Ugljesa Djuric, Aaron Cheung, Alina Piekna, Jason Hendry, Joel<br />
Ross, Peter Pasceri, Dae-Sung Kim, James Ellis.<br />
594<br />
Immune Response Activation and Neurotoxicity after<br />
Intrathecal Infusion <strong>of</strong> AAV9 Vector Encoding a Non-Self<br />
Reporter Protein in the Non-Human Primate<br />
Lluis Samaranch, Waldy San Sebastian, Adrian P. Kells, John<br />
Bringas, Phillip Pivirotto, John Forsayeth, Kryst<strong>of</strong> S. Bankiewicz.<br />
595<br />
Intracranial Injection <strong>of</strong> AAVrh8-mbgal in a GM1<br />
Gangliosidosis Mouse Model Delays Disease Onset and<br />
Signiicantly Extends Life Span<br />
Cara M. Weismann, Jennifer Ferreira, Stacy Maitland, Miguel<br />
Sena-Esteves.<br />
596<br />
Enhanced CNS Transduction and Transgene Expression<br />
by rAAV Capsid Tyrosine Mutants<br />
Nicholas M. Kanaan, Caryl E. Sortwell, Sara E. Gombash, Nathan<br />
M. Kuhn, William W. Hauswirth, Sanford E. Boye, Vince A. Chiodo,<br />
Tessa Grabinski, Fredric P. Manfredsson.<br />
597<br />
Peripheral Blood White <strong>Cell</strong>s Present Molecular<br />
Signatures <strong>of</strong> Neurodegeneration and Respond to a<br />
Single Intravenous rAAV9-hNAGLU <strong>Gene</strong> Delivery in<br />
MPS IIIB Mice<br />
Bartholomew J. Naughton, F. Jason Duncan, Tierra Ware, Darren<br />
Murrey, Aaron Meadows, Douglas M. McCarty, Haiyan Fu.<br />
598<br />
The Therapeutic Potential <strong>of</strong> MicroRNA Regulation in a<br />
Rat Intracerebral Hemorrhage Model<br />
Jangsup Moon, Jeong-Min Kim, Jung Ick Byun Byun, Kon Chu,<br />
Keun-Hwa Jung, Soon-Tae Lee, Jin-Hee Kim, Dong-Kyu Park, So-<br />
Yoon Kim, Jae-Kyu Roh.<br />
599<br />
Adeno-Associated Virus Type 6 Is Retrogradely<br />
Transported in the Non-Human Primate Brain,<br />
Implications for <strong>Gene</strong> Delivery in Huntington’s Disease<br />
Waldy San Sebastian, Lluis Samaranch, Adrian P. Kells, John<br />
Bringas, Phillip Pivirotto, John Forsayeth, Kryst<strong>of</strong> S. Bankiewicz.<br />
600<br />
Herpes Simplex Virus 2 <strong>Gene</strong>, ICP10PK, Protects<br />
Against Neuronal <strong>Cell</strong> Death Associated with Valproic<br />
Acid-Induced Autism Spectrum Disorder<br />
Dominique Bollino, Laure Aurelian.<br />
601<br />
Targeted <strong>Gene</strong> Transfer into the Ependymal <strong>Cell</strong>s<br />
and Long-Term Enzyme Replacement in the CSF by<br />
Intraventricular Injection <strong>of</strong> AAV Vector Serotype 1<br />
Yoshiyuki Yamazaki, Kohei Hironaka, Yukihiko Hirai, Koichi<br />
Miyake, Takashi Shimada.<br />
602<br />
Neuroprotective Effect <strong>of</strong> a <strong>Cell</strong>-Free Extract Derived<br />
from Human Adipose Stem <strong>Cell</strong>s in Experimental Stroke<br />
Models<br />
Jung Ick Byun, Daejong Jeon, Kon Chu, Soon-Tae Lee, Keun-Hwa<br />
Jung, Jae-Jun Ban, Hye-Jin Yoon, Seungmoon Jung, Hyunwoo Yang,<br />
Byung Sun Kim, Ji Ye Choi, Jeong-Min Kim, Chong-Hyun Won,<br />
Manho Kim, Sang Kun Lee, Jae-Kyu Roh.<br />
603<br />
Artiicial MicroRNAs Against Spliced Variants <strong>of</strong><br />
the <strong>Gene</strong> C9ORF72, the Major Cause for Familial<br />
Amyotrophic Lateral Sclerosis<br />
Gabriela Toro, Florie Borel, Peter Sapp, Lina Song, Robert Brown,<br />
Christian Mueller.<br />
604<br />
Reduction <strong>of</strong> Hyperammonemia In Vitro and In Vivo by<br />
a Baculovirus-Delivered Glutamine Synthetase as a New<br />
Approach for the Treatment <strong>of</strong> Hepatic Encephalopathy<br />
Miguel A. Torres-Vega, Lizbeth M. Vázquez-Carcaño, Roxana<br />
Y. Vargas-Jerónimo, Ana G. Montiel-Martínez, Rosa M. Muñoz-<br />
Fuentes, Misael Uribe, Absalom Zamorano-Carrillo, Ana R. Pastor-<br />
Flores, Laura A. Palomares.<br />
132<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013
Abstract Directory, Saturday, May 18, 2013<br />
Poster Session III, continued<br />
3:45 PM - 5:45 PM<br />
Room: Exhibit Hall C/D<br />
Musculo-skeletal <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> II<br />
605<br />
MTMR2 Ameliorates the Phenotype <strong>of</strong> Myotubular<br />
Myopathy in Mice<br />
Nathalie Danièle, Thibaud Jamet, Christelle Moal, Romain Joubert,<br />
Nadia Messaddeq, Michael W. Lawlor, Alban Vignaud, Anna<br />
Buj-Bello.<br />
606<br />
Restricting MTM1 Transgene Expression to Skeletal<br />
Muscle in AAV-Mediated <strong>Gene</strong> <strong>Therapy</strong> for Myotubular<br />
Myopathy<br />
Romain Joubert, Christelle Moal, Alban Vignaud, Samia Martin,<br />
Isabelle Richard, Philippe Moullier, Alan H. Beggs, Martin K.<br />
Childers, Anna Buj-Bello.<br />
607<br />
Long Term Intra-Articular Expression <strong>of</strong> PRG4 Prevents<br />
Post-Traumatic Osteoarthritis<br />
Zhechao Ruan, Vincenzo Cerullo, Kilian Guse, Ayelet Erez, Brian<br />
Dawson, Frank Gannon, Brendan Lee.<br />
608<br />
Immune Tolerance Induction in Canine X-Linked<br />
Muscular Dystrophy with Trans-Placental rAAV9-<br />
Microdystrophin Transduction<br />
Hiromi Hayashita-Kinoh, Hironori Okada, Yuko Nitahara-Kasahara,<br />
Tomoko Chiyo, Naoko Yugeta, Takashi Okada, Shin’ichi Takeda.<br />
609<br />
Low Dystrophin Levels Increase Survival and Improve<br />
Muscle Pathology and Function in Dystrophin/Utrophin<br />
Double Knockout Mice<br />
Maaike van Putten, Margriet Hulsker, Courtney Young, Peter A. ‘t<br />
Hoen, Gert-Jan B. van Ommen, Annemieke M. Aartsma-Rus.<br />
610<br />
Limb Girdle Muscular Dystrophy 2I: <strong>Gene</strong>ration <strong>of</strong> a<br />
New Mouse Model and Test <strong>of</strong> Therapeutic Approaches<br />
by rAAV2/9 <strong>Gene</strong> Transfer<br />
Evelyne Gicquel, Karine Charton, Nathalie Daniele, Isabelle<br />
Richard.<br />
611<br />
Hepatocyte Growth Factor Contributes to the Improved<br />
Pathology <strong>of</strong> mdx:p65+/- Mice<br />
Jonathan D. Proto, Ying Tang, Aiping Lu, Minakshi Poddar, Sarah<br />
Beckman, Kayla Imbrogno, Timothy Hannigan, Wendy Mars, Bing<br />
Wang, Johnny Huard.<br />
612<br />
Urine Biomarkers for Duchenne Muscular Dystrophy<br />
and Its Animal Models<br />
Jérémy Rouillon, Thibaut Léger, Jean-Michel Camadro, Tomas Voit,<br />
Fedor Svinartchouk.<br />
613<br />
Systemic <strong>Gene</strong> Transfer in Neonatal Dogs Using<br />
Tyrosine Mutant AAV-1,6 and9<br />
Chady H. Hakim, Yongping Yue, Jin-Hong Shin, Regina R. Williams,<br />
Keqing Zhang, Arun Srivastava, Bruce F. Smith, Dongsheng Duan.<br />
614<br />
Effect <strong>of</strong> Angiogenesis on the Regenerative Capacity <strong>of</strong><br />
ACL-Derived CD34+ <strong>Cell</strong>s in ACL Reconstruction<br />
Koji Takayama, Tang Ying, Wang Bing, Freddie Fu, Johnny Huard.<br />
615<br />
The Phenotype <strong>of</strong> Dysferlin-Deicient Mice Is Not<br />
Rescued by AAV-Mediated Transfer <strong>of</strong> Anoctamin 5<br />
Karine Charton, Laurence Suel-Petat, François Monjaret, Nathalie<br />
Bourg-Alibert, Carinne Roudaut, Evelyne Gicquel, Bjarne Udd,<br />
Isabelle Richard.<br />
616<br />
Transfection <strong>of</strong> Murine Dermis Via Topical DNA<br />
Plasmid Delivery<br />
Chunqing Dou, Guy P. Marti, Lixin Liu, Frank Lay, Donald J. Rees,<br />
Aaron T. Tabor, Junkai Du, Sayed Mohammd Hosseini, Amir Mehdi<br />
Ansari, Ali Karim Ahmed, James A. Williams, John W. Harmon.<br />
617<br />
Clinical Trial Readiness for <strong>Gene</strong> Replacement to the<br />
Diaphragm: Translational Respiratory Endpoints in<br />
Ventilator-Dependent Neuromuscular Diseases<br />
Barbara K. Smith, Melissa A. Goddard, Martin K. Childers, Barry J.<br />
Byrne.<br />
618<br />
Adjunctive b2-Agonist <strong>Therapy</strong> Enhances Traficking <strong>of</strong><br />
Acid a-Glucosidase to Lysosomes in the Skeletal Muscle<br />
<strong>of</strong> Mice with Pompe Disease<br />
Dwight D. Koeberl, Songtao Li, Benjamin Farah, Andrew Bird, Paul<br />
M. Yen, Sarah P. Young.<br />
619<br />
Comparative In Vitro Potency <strong>of</strong> a Novel Disc-Derived<br />
<strong>Cell</strong> Population (Discospheres) and Mesenchymal Stem<br />
<strong>Cell</strong>s for the Treatment <strong>of</strong> Degenerative Disc Disease<br />
Lara I. Silverman, Galina Dulatova, Terry Tandeski, Kevin T. Foley.<br />
620<br />
Functional Analysis <strong>of</strong> GNE Expression Plasmids in<br />
Immortalized Human Myoblasts<br />
Yadira Valles-Ayoub, Rosangela Carbajo, Lucia Sandoval, Jorge<br />
Garcia-Figueroa, Sarah Stein, Zeshan Khokher, Daniel No, Babak<br />
Darvish, Jacques P. Tremblay, Daniel Darvish.<br />
Cancer Immunotherapy III<br />
621<br />
Tethered IL-15 Mutein on CD19-Speciic T <strong>Cell</strong>s<br />
Sustains Persistence When Tumor Antigen Is Low and<br />
Can Treat Minimal Residual Disease<br />
Lenka V. Hurton, Kirsten Switzer, Harjeet Singh, Marie A. Forget,<br />
Tiejuan Mi, Simon Olivares, Richard E. Champlin, Dean A. Lee,<br />
Laurence J. N. Cooper.<br />
Saturday, May 18, 2013<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013<br />
133
Abstract Directory, Saturday, May 18, 2013<br />
Saturday, May 18, 2013<br />
Poster Session III, continued<br />
3:45 PM - 5:45 PM<br />
Room: Exhibit Hall C/D<br />
Cancer Immunotherapy III<br />
622<br />
Rapid Assessment <strong>of</strong> T-<strong>Cell</strong> Function in an Animal-Free<br />
Model – The Artiicial-Mouse Model<br />
Usanarat Anurathapan, Pradip Bajgain, Norihiro Watanabe,<br />
Malcolm K. Brenner, Cliona M. Rooney, John R. Wilson, Juan F. Vera.<br />
623<br />
Distinct Signaling from Chimeric Antigen (CAR) and T<br />
<strong>Cell</strong> Receptors in T <strong>Cell</strong>s<br />
Omkar U. Kawalekar, Avery D. Posey Jr, Sonia Guedan, Jihyun Lee,<br />
Matthew J. Frigault, Maria C. Basil, John Scholler, Yangbing Zhao,<br />
Carl H. June.<br />
624<br />
Long Term F/U – Phase I Study <strong>of</strong> the “Triad”<br />
Autologous (FANG) Vaccine, Incorporating<br />
Bifunctional shRNAfurin and GMCSF Transgene<br />
Expression, in Advanced Cancer Patients<br />
John Nemunaitis, Neil Senzer, Minal Barve, Cynthia Bedell, Joseph<br />
Kuhn, Peter Beitsch, Robert Steckler, Jeffrey Lamont, Robert<br />
Hebeler, Mitchell J. Magee, Walton Taylor, Brian M. Gogel, Maurizio<br />
Ghisoli, F. Charles Brunicardi, Beena O. Pappen, Padmasini Kumar,<br />
Phillip B. Maples.<br />
625<br />
Phase I Study Results <strong>of</strong> pbi-shRNA STMN1 Nanoplex<br />
Via Intratumoral Injection in Advanced Cancer<br />
John Nemunaitis, F. Charles Brunicardi, Donald D. Rao, Nancy S.<br />
Templeton, Neil Senzer, Zhaohui Wang, Minal Barve, Padmasini<br />
Kumar, Gladice Wallraven, Christopher M. Jay, Joseph Kuhn, Peter<br />
Beitsch, Phillip B. Maples.<br />
626<br />
T-<strong>Cell</strong> Immunotherapy for Multiple Myeloma<br />
Sonny O. Ang, Sourindra N. Maiti, Ippei Sakamaki, Hong Qin, Larry<br />
W. Kwak, Richard E. Champlin, Laurence J. N. Cooper.<br />
627<br />
<strong>Gene</strong> Transfer <strong>of</strong> Chondroitin Sulfate-Coated<br />
Granulocyte Macrophage-Colony-Stimulating Factor in<br />
Mouse Ovarian Tumor Model<br />
Katsuyuki Hamada, Kazuko Takagi, Chieko Yoshihara, Tomoko Ito,<br />
Yoshiyuki Koyama, Hiroshi Ito, Akihiro Nawa.<br />
628<br />
EphrinA1-EphA2 Interaction-Mediated Apoptosis and<br />
Flt3L-Induced Immunotherapy in Inhibiting Tumor<br />
Progression in an Immunocompetent Mouse Model <strong>of</strong><br />
Breast Cancer<br />
Sai vikram V. vemula, Manish Tandon, Suresh K. Mittal, Anurag<br />
Sharma, Dinesh Bangari.<br />
629<br />
Optimization <strong>of</strong> Chimeric Antigen Receptors Design:<br />
Novel Retroviral Vectors Expressing CAR with<br />
Intracellular Signaling Domain <strong>of</strong> Glucocorticoid-<br />
Induced TNF-Receptor<br />
Yasunori Amaishi, Sachiko Okamoto, Yoshikazu Kurosawa, Hiroshi<br />
Shiku, Junichi Mineno, Kazuto Takesako.<br />
630<br />
Combined p19Arf and Interferon-beta <strong>Gene</strong> <strong>Therapy</strong>:<br />
Evidence <strong>of</strong> Immune Response in Murine Models <strong>of</strong><br />
Melanoma and Lung Carcinoma<br />
Bryan E. Strauss, Ruan Felipe V. Medrano, Aline H. Ribeiro, Joao<br />
Paulo P. Catani, Christian A. Merkel.<br />
631<br />
Histidine-Rich Glycoprotein (HRG): A Novel <strong>Gene</strong>-<br />
<strong>Therapy</strong> Effector for the Treatment <strong>of</strong> Cancer<br />
Francis P. Roche, K. Elisabet Ohlin, Magnus Essand, Lena<br />
Claesson-Welsh.<br />
632<br />
Effect <strong>of</strong> Interferon-Omega <strong>Gene</strong> Transfer on Feline<br />
Mammary Carcinoma and Melanoma <strong>Cell</strong>s<br />
Marcela S. Villaverde, Alexandra M. Targovnik, Daniel R. Bafico,<br />
Maria V. Miranda, Gerardo C. Glikin, Liliana M. E. Finocchiaro.<br />
633<br />
HLA Typing Identity Test for Patients and Their FANG<br />
Autologous Cancer Vaccines – Update<br />
Yang Yu, Padmasini Kumar, Fabienne Norvell, Connor Phalon,<br />
Nicolas Taquet, Phillip B. Maples.<br />
Cancer-Oncolytic Viruses II<br />
634<br />
Therapeutic Targeting <strong>of</strong> Chitosan-PEG-Folate-<br />
Complexed Oncolytic Adenovirus for Active and<br />
Systemic Cancer <strong>Gene</strong> <strong>Therapy</strong><br />
Oh-Joon Kwon, Eunah Kang, Sung Wan Kim, Chae-Ok Yun.<br />
635<br />
Human Ovarian Cancer-Derived Ascitic Fluid Has Mixed<br />
Effects on CRAds Eficacy<br />
Veronica M. Lopez, Nicasio Cuneo, Mariela A. Gangemi, Leonardo<br />
Sganga, Marina Demonte, Alejandro Soderini, Osvaldo L.<br />
Podhajcer.<br />
636<br />
The Oncolytic Virus DPK Lyses <strong>Cell</strong>s with Cancer<br />
Stem <strong>Cell</strong> (CSC) Properties through Calpain-Mediated<br />
Clearance <strong>of</strong> the Selective Autophagy Protein p62/<br />
SQSTM1<br />
Aric Colunga, Dominique Bollino, Amanda Schech, Laure Aurelian.<br />
637<br />
Demonstration <strong>of</strong> Anti-Metastasis Activity <strong>of</strong> Oncolytic<br />
Measles Virus Retarging Urokinase Receptor in<br />
Experimental Breast Cancer Metastasis Model<br />
Yuqi Jing, Krisztina Kovacs, Jaime Merchan.<br />
134<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013
Abstract Directory, Saturday, May 18, 2013<br />
Poster Session III, continued<br />
3:45 PM - 5:45 PM<br />
Room: Exhibit Hall C/D<br />
Cancer-Oncolytic Viruses II<br />
638<br />
Development <strong>of</strong> Dual Targeted Oncolytic Adenovirus<br />
for Human Malignant Mesothelioma<br />
Shuji Kubo, Misato Takagi-Kimura, Atsuko Tamamoto, Tomoko<br />
Hashimoto-Tamaoki, Noriyuki Kasahara, Masatoshi Tagawa.<br />
639<br />
MicroRNA-Mediated Multi-Tissue-Detargeting <strong>of</strong><br />
Oncolytic Measles Virus<br />
Marc-Andrea Bärtsch, Mathias F. Leber, Sascha Bossow,<br />
Martin Singh, Jessica Albert, Dirk Jäger, Christ<strong>of</strong> von Kalle, Guy<br />
Ungerechts.<br />
646 Abstract Withdrawn from Presentation<br />
647<br />
Iraqi Newcastle Disease Virus Virulent Strain as Cancer<br />
Antiangiogenic Agent<br />
Ahmed M. Al-Shammari, Zaid A. Al-Hili, Nahi Y. Yaseen.<br />
Cancer-Targeted <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>: <strong>Cell</strong><br />
<strong>Therapy</strong> / Immunotherapy / <strong>Cell</strong>ular Targets<br />
648<br />
Neural Stem <strong>Cell</strong>s Expressing a Modiied Human<br />
Carboxylesterase Potentiate Anti-Cancer Activity <strong>of</strong><br />
Irinotecan in Murine Glioma Model<br />
Karen S. Aboody, Lucy Y. Ghoda, Margarita Gutova, Megan<br />
Gilchrist, Christine Brown, Vazgen Kankaldyyan, Elizabth Garcia,<br />
Lauren H<strong>of</strong>fman, Rex A. Moats, Phil Potter, Timothy W. Synold,<br />
Michael Barish.<br />
640<br />
In Vivo Therapeutic Eficacy <strong>of</strong> Oncolytic Adenovirus<br />
Delivered by New Biodegradable Polymers through<br />
Systemic Injection<br />
Jaesung Kim, Yi Li, Sung Wan Kim, Doo Sung Lee, Chae-Ok Yun.<br />
641<br />
Eficacy <strong>of</strong> Measles Virotherapy in Osteosarcoma<br />
Evidio Domingo Musibay, Cory Allen, Ianko D. Iankov, Jayson J.<br />
Hardcastle, Evanthia Galanis.<br />
642<br />
A Cancer-Targeting Ligand Strongly Enhances Oncolytic<br />
Activity <strong>of</strong> a Conditionally Replicative Adenovirus<br />
Yuki Yamamoto, Naoko Goto, Shumpei Ohnami, Masatoshi Tagawa,<br />
Kazunori Aoki.<br />
643<br />
Cyclophosphamide Enhances Antitumor<br />
Eficacy <strong>of</strong> Oncolytic Adenovirus Expressing<br />
Uracil Phosphoribosyltransferase (UPRT) in<br />
Immunocompetent Syrian Hamsters<br />
Naoyuki Hasegawa, Masato Abei, Kazunari K. Yokoyama, Kuniaki<br />
Fukuda, Emiko Seo, Rei Kawashima, Yuri Nakano, Takeshi Yamada,<br />
Koji Nakade, Hir<strong>of</strong>umi Hamada, Yuichi Obata, Ichinosuke Hyodo.<br />
644<br />
Therapeutic Evaluation <strong>of</strong> Prostate Cancer Speciic<br />
Oncolytic Adenovirus Armed with Fusion Protein <strong>Gene</strong><br />
PSA-IZ-CD40L<br />
Yue-feng Yang, Shu-Ya Xue, Hua Wang, Feng-jun Xiao, Yue Yin,<br />
Zhuo-zhuang Lu, Qun-wei Zhang, Chu-tse Wu, Li-sheng Wang.<br />
645<br />
Bioreducible Polymer-Conjugated Oncolytic<br />
Adenovirus for Hepatoma-Speciic <strong>Therapy</strong> Via<br />
Systemic Administration<br />
Pyung-Hwan Kim, Jaesung Kim, Tae-il Kim, Hye Yeong Nam, James<br />
W. Yockman, Minjung Kim, Sung Wan Kim, Chae-Ok Yun.<br />
649<br />
Re-Directing T <strong>Cell</strong>s with Chimeric Antigen Receptors<br />
To Target CD123+ Leukemia<br />
Radhika Thokala, Kirsten Switzer, Mi Ti juan, Simon Olivares,<br />
Harjeet Singh, Helen Huls, Richard E. Champlin, Laurence J. N.<br />
Cooper.<br />
650<br />
The Speciicity <strong>of</strong> Rhesus T <strong>Cell</strong>s Can Be Redirected<br />
to CD20 Using the Sleeping Beauty Transposon/<br />
Transposase System To Express a Chimeric Antigen<br />
Receptor<br />
Simon Olivares, Colleen O’Connor, Sonny Ang, Leslie Kean, Perry<br />
Hackett, Amer Najjar, Harjeet Singh, Helen Huls, Elizabeth Shpall,<br />
Laurence Cooper.<br />
651<br />
Tumor Targeting Using CD19-Speciic CAR (Chimeric<br />
Antigen Receptor) for B-<strong>Cell</strong> Lymphoma<br />
Tomonori Tsukahara, Ken Ohmine, Ryosuke Uchibori, Hiroyuki<br />
Ido, Masashi Urabe, Hiroaki Mizukami, Akihiro Kume, Masataka<br />
Nakamura, Junichi Mineno, Kazutoh Takesako, Isabelle Riviere,<br />
Michel Sadelain, Renier Brentjens, Keiya Ozawa.<br />
652<br />
Tumor Relapse Is Associated with an Innate Immune<br />
Resistant Phenotype across Tumor and Treatment<br />
Types Which Can Be Targeted with Rational Second<br />
Line Therapies<br />
Timothy Kottke, Oliver Donnelly, Nicolas Boisgerault, Rosa<br />
Diaz, Diana Rommelfanger-Konkol, Jose Pulido, Jill Thompson,<br />
Debabrata Mukhopadhyay, Keith Knutson, Marshall Behrens,<br />
Roger Kaspar, Matt C<strong>of</strong>fey, Peter Selby, Kevin Harrington, Alan<br />
Melcher, Hardev Pandha, Richard Vile.<br />
653<br />
Chimeric Antigen Receptor T-<strong>Cell</strong> <strong>Therapy</strong> for<br />
Companion Canines with Spontaneous B-<strong>Cell</strong> Non-<br />
Hodgkin Lymphoma<br />
Colleen M. O’Connor, Simon Olivares, Sonny Ang, Richard E.<br />
Champlin, Heather M. Wilson-Robles, Laurence J. N. Cooper.<br />
Saturday, May 18, 2013<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013<br />
135
Abstract Directory, Saturday, May 18, 2013<br />
Saturday, May 18, 2013<br />
Poster Session III, continued<br />
3:45 PM - 5:45 PM<br />
Room: Exhibit Hall C/D<br />
Cancer-Targeted <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>: <strong>Cell</strong><br />
<strong>Therapy</strong> / Immunotherapy / <strong>Cell</strong>ular Targets<br />
654<br />
Combination <strong>of</strong> CD3+CD56+ (NKT) <strong>Cell</strong>s<br />
Immunotherapy and Adenovirus-p53 <strong>Gene</strong> <strong>Therapy</strong> for<br />
Head and Neck Squamous <strong>Cell</strong> Carcinoma<br />
Hiroki Saito, Naoya Morishita, Kyung-Mi Lee, Dante Dator, Masato<br />
Kawabata, Masato Fujisawa, Toshiro Shirakawa.<br />
655<br />
Interfering Peptides Targeting Transcription Factor<br />
AP1 for Pancreatic Cancer <strong>Gene</strong> <strong>Therapy</strong><br />
Soukaina Rejiba, Megan Frelan, Alex Hermann, Katja Arndt, Amor<br />
Hajri.<br />
656<br />
JMJD3 Regulates Senescence-Associated Secretory<br />
Phenotype Factors Responsible for Robust Neural Stem<br />
<strong>Cell</strong> Tropism toward Glioma<br />
Michael E. Silva, Patrick M. Perrigue, Daniel Mota, Lauren Joseph,<br />
Nathan Feng, Michael Reid, Joseph Najbauer, Karen S. Aboody.<br />
657<br />
Bioengineering ACE Synthetic Chromosomes for Anti-<br />
Cancer <strong>Gene</strong> <strong>Therapy</strong><br />
Edward Perkins, Jon Holy, Teresa Rose-Hellkant, Amy Greene.<br />
658<br />
DNA Vaccine with scFv6.C4 Prevent the Growth <strong>of</strong><br />
Tumor Expressing CEA<br />
Priscila M. A. Denapoli, Roberta S. Stilhano, Priscila K. Matsumoto,<br />
Carolina B. Parise, Jane Z. de Moraes, Wolfgang Zimmermann, Sang<br />
W. Han.<br />
659<br />
Presumptive Oncogenic Transcriptional Factor PDX1<br />
Regulates Pancreatic Cancer Via an Ampliication Loop<br />
Involving the RAS, PI3K and p53 Signaling Pathways<br />
Juehua Yu, Guisheng Zhou, Shi-he Liu, James Wu, John Nemunaitis,<br />
Neil Senzer, Donald Rao, Enrique Rosengurt, F. C. Brunicardi.<br />
660<br />
Targeting Lysosome Integrity as a Method To Restore<br />
Sensitivity to Ceramide-Induced <strong>Cell</strong> Death<br />
Lorianne S. Turner, Krissy Smith, Lenton Holley, Christopher<br />
Johnson, Timothy Prince, Heather Yancey.<br />
Hematologic and Immunologic <strong>Gene</strong> & <strong>Cell</strong><br />
<strong>Therapy</strong> II<br />
661<br />
Lentiviral <strong>Gene</strong> <strong>Therapy</strong> <strong>of</strong> RAG Severe Combined<br />
Immunodeiciency<br />
Niek P. van Til, Guus van der Velden, Helen de Boer, Pietro L.<br />
Poliani, Michael N. Antoniou, Anna Villa, Chantal Lagresle-Peyrou,<br />
Marina Cavazzana-Calvo, Fang Zhang, Adrian J. Thrasher, Gerard<br />
Wagemaker.<br />
662<br />
Stem <strong>Cell</strong> and T-<strong>Cell</strong> <strong>Gene</strong> <strong>Therapy</strong> Using SIN-<br />
Lentiviral Vector in Type 3 Familial Hematophagocytic<br />
Lymphohistocytosis: A Comparative Approach<br />
Julie Rivière, Tayebeh Soheili, Els Verhoeyen, Anne Galy, <strong>Gene</strong>viève<br />
De Saint Basile, Marina Cavazzana-Calvo.<br />
663<br />
High Expression Bioengineered Factor VIII Enables<br />
AAV-Based <strong>Gene</strong> <strong>Therapy</strong> <strong>of</strong> Hemophilia A<br />
H. Trent Spencer, Harrison C. Brown, Jordan E. Shields, J. Fraser<br />
Wright, Christopher B. Doering.<br />
664<br />
Identiication <strong>of</strong> Novel Combination <strong>of</strong> Elements in<br />
cHS4 with Optimal Insulator Activity and Improved<br />
Titers in g-Globin Carrying SIN Lentiviral Vectors<br />
Paritha Arumugam, Michael Wourms, Joseph Starnes, Catherine<br />
Fox, Xiaoming Li, Punam Malik.<br />
665<br />
Intraosseous Delivery <strong>of</strong> Lentiviral Vectors Expressing<br />
Factor VIII under the Control <strong>of</strong> the Platelet-Speciic<br />
Glycoprotein 1ba Promoter Leads to Long-Term<br />
Correction <strong>of</strong> Bleeding in Hemophilia A Mice<br />
Xuefeng Wang, Andy Chiang, Simon C. Shin, Liping Chen, Dao Pan,<br />
David J. Rawlings, Carol H. Miao.<br />
666<br />
Lentiviral Vectors Tailored for Liver-Directed <strong>Gene</strong><br />
<strong>Therapy</strong> Do Not Show Any Detectable Genotoxicity in<br />
Sensitive In Vivo Biosafety Assays<br />
Marco Ranzani, Alessio Cantore, Monica Volpin, Pierangela Gallina,<br />
Stefano Annuziato, Francesca Sanvito, Fabrizio Benedicenti,<br />
Cynthia Bartholomae, Lucia Sergi Sergi, Christ<strong>of</strong> von Kalle,<br />
Manfred Schmidt, Claudio Doglioni, Luigi Naldini, Eugenio<br />
Montini.<br />
667<br />
Successful Liver <strong>Gene</strong> <strong>Therapy</strong> <strong>of</strong> Hemophilia B<br />
Dogs Using Lentiviral Vectors and Hyperfunctional<br />
Transgene<br />
Alessio Cantore, Patrizia Della Valle, Sara Bartolaccini, Lucia Sergi<br />
Sergi, Thierry VandenDriessche, Marinee Chuah, Dwight Bellinger,<br />
Armando D’Angelo, Timothy Nichols, Luigi Naldini.<br />
136<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013
Abstract Directory, Saturday, May 18, 2013<br />
Poster Session III, continued<br />
3:45 PM - 5:45 PM<br />
Room: Exhibit Hall C/D<br />
Hematologic and Immunologic <strong>Gene</strong> & <strong>Cell</strong><br />
<strong>Therapy</strong> II<br />
Late Breaking Abstracts III<br />
733<br />
Correction <strong>of</strong> canine Factor VII (FVII) deiciency by<br />
AAV-mediated expression <strong>of</strong> canine FVII zymogen<br />
Paris Margaritis, Dwight A. Bellinger, Shangzhen Zhou, Armida<br />
Faella, Timothy C. Nichols, Katherine A. High<br />
668<br />
Engineering <strong>of</strong> a HIV-Resistant Immune System with<br />
Hematopoietic Stem <strong>Cell</strong>s – A Polycistronic Approach<br />
Janet Chung, Chy-Anh Tran, Angel Gu, Lisa Scherer, Haitang Li,<br />
David L. DiGiusto, John J. Rossi.<br />
669<br />
Direct and Indirect Immune Suppressive Effects <strong>of</strong> High<br />
Hepatic Expression Direct Effective Reversal <strong>of</strong> Factor<br />
IX Inhibitors by AAV <strong>Gene</strong> Transfer<br />
David Markusic, Roland Herzog.<br />
670<br />
Combination <strong>Therapy</strong> <strong>of</strong> Anti-CD20, AMD3100, G-CSF<br />
and Factor VIII Plasmid-Mediated <strong>Gene</strong> <strong>Therapy</strong><br />
Can Successfully Promote Tolerance to Factor VIII in<br />
Hemophilia A Mice with Pre-Existing Inhibitors<br />
Chao Lien Liu, Meghan Lyle, Simon C. Shin, Carol H. Miao.<br />
671<br />
Identiication <strong>of</strong> Novel Chromatin Insulator<br />
Elements for Use in Lentiviral <strong>Gene</strong> <strong>Therapy</strong> <strong>of</strong> T <strong>Cell</strong><br />
Immunodeiciencies<br />
Sheng Zhou, Taihe Lu, Zhijun Ma, John T. Gray, Brian P. Sorrentino.<br />
672<br />
Transduction <strong>of</strong> Human CD34+ <strong>Cell</strong>s in the NOD SCIDg<br />
(NSG) Mouse Model Using Lenti-hWASP-Vectors Derived<br />
from Stable Producer Clones<br />
Matthew M. Wielgosz, Yoon S. Kim, Robert E. Throm, Gael G.<br />
Carney, John T. Gray, Arthur W. Nienhuis.<br />
673<br />
Pre-Clinical Modeling <strong>of</strong> Foamy Virus Vector Based<br />
<strong>Gene</strong> <strong>Therapy</strong> for SCID-X1<br />
Gabrielle M. Curinga, Iram Khan, Swati Singh, Joey Pangallo, Brian<br />
Beard, Troy Torgerson, Andrew Scharenberg, Grant Trobridge,<br />
Hans-Peter Kiem, David Rawlings.<br />
674<br />
Long Term Safety <strong>of</strong> Clinical Grade LentiGlobin Vectors<br />
in a b-Thalassemic and Normal Mice<br />
Olivier Negre, Cynthia Bartholoma, Robert Kutner, Beatrix Gillet-<br />
Legrand, Celine Courne, Anais Paulard, Byoung Ryu, Maria Denaro,<br />
Christ<strong>of</strong> von Kalle, Emmanual Payan, Michell Finer, Gabor Veres.<br />
675 Abstract Withdrawn from Presentation<br />
734 Abstract Withdrawn from Presentation<br />
735<br />
Optimization <strong>of</strong> dual-function vectors for Alpha-1<br />
Antitrypsin Deiciency<br />
Qiushi Tang, Christian Mueller, Lina Song, Terence Flotte<br />
736<br />
Targeting dendritic cells with lentiviral vectors induces<br />
tolerance <strong>of</strong> effector T cells and protects mice from<br />
experimental autoimmune encephalomyelitis<br />
Bruna de Andrade Pereira, Mathias Ackermann, Christiane Dresch,<br />
Cornel Fraefel<br />
737<br />
Novel adenoviral vectors induce robust T cell responses<br />
to HSV2 and signiicantly boost responses after repeat<br />
homologous administration<br />
Christopher A. Lazarski, Douglas E. Brough, Lisa L. Wei<br />
738<br />
TALEN-based Individualized <strong>Gene</strong> Medicine for<br />
Epidermolysis Bullosa<br />
Mark J. Osborn, Colby G. Starker, Amber N. McElroy, Beau R.<br />
Webber, Anthony P. DeFeo, Richard Gabriel, Manfred Schmidt,<br />
Christ<strong>of</strong> von Kalle, Daniel F. Carlson, Morgan L. Maeder, J. Keith<br />
Joung, John E. Wagner, Daniel F. Voytas, Bruce R. Blazar, Jakub<br />
Tolar<br />
739<br />
Prevention <strong>of</strong> Anti-Capsid Immune Response Following<br />
Systemic AAV9 Dosing in Infant Rhesus Monkeys<br />
Darin Falk<br />
740<br />
Inhibition <strong>of</strong> ocular neovascularization by<br />
subconjunctival gene delivery <strong>of</strong> calreticulin-derived<br />
peptide, vasostatin 112<br />
Guei-Sheung Liu, Youn-Shen Bee, Gregory J. Dusting, Ming-Hong<br />
Tai<br />
741<br />
Transient Epigenetic <strong>Gene</strong> <strong>Therapy</strong> as a New<br />
Therapeutic/Prophylactic Avenue for Aging/<br />
Degenerative Diseases and Neurodevelopmental<br />
Disorders<br />
Roger Bertolotti<br />
742<br />
Successful Systemic Cancer Targeting by Adenovirus<br />
Vector with AB-loop Redesigned Fiber via Reducing<br />
Liver Sequestration<br />
Yoshiaki Miura, Joohee Han, Julia Davydova, Masato Yamamoto<br />
Saturday, May 18, 2013<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013<br />
137
Abstract Directory, Saturday, May 18, 2013<br />
Poster Session III, continued<br />
3:45 PM - 5:45 PM<br />
Room: Exhibit Hall C/D<br />
Late Breaking Abstracts III<br />
743<br />
Prevention <strong>of</strong> radiation-induced salivary hyp<strong>of</strong>unction<br />
by delivering hSFRP2 into submandibular glands<br />
Changyu Zheng, Ana P. Cotrim, Corinne Goldsmith, Stefanie<br />
Aquilina, Anastasia Sowers, Indu S. Ambudkar, James B. Mitchell,<br />
Bruce J. Baum<br />
744<br />
The non-signaling extracellular spacer domain <strong>of</strong><br />
CD19-speciic chimeric antigen receptors is decisive for<br />
in vivo antitumor activity<br />
Michael Hudecek, Lingfeng Liu, Anne Silva, Paula Kosasih, Yvonne<br />
Y. Chen, Cameron J. Turtle, Michael C. Jensen, Stanley R. Riddell<br />
745 Abstract Withdrawn from Presentation<br />
746<br />
Determining the physiologic effect <strong>of</strong> B-Type<br />
Natriuretic Peptide on cardio-renal homeostasis<br />
Sara J Holditch, Alessandro Cataliotti, Aron Geurts, Yasuhiro Ikeda<br />
747<br />
Bortezomib induced unfolded protein response<br />
synergizes with oHSV to induce necroptic cell death in<br />
solid tumors<br />
Ji Young Yoo, Brian Hurwitz, Chelsea Bolyard, Jun-Ge Yu, Jeffrey<br />
Wojton, Jianying Zhang, Matthew Old, Balveen Kaur<br />
753<br />
Non-myeloablative preconditioning with Ack-2 for cell<br />
therapy for Hunter disease<br />
Takayuki Yokoi, Kentaro Yokoi, Kazumasa Akiyama, Takashi<br />
Higuchi, Yohta Shimada, Hiroshi Kobayashi, Yoshikatsu Eto, Taku<br />
Sato, Makoto Otsu, Hiromitsu Nakauchi, Shinichi Nishikawa,<br />
Hiroyuki Ida, Toya Ohashi<br />
754<br />
Human placenta-derived mesenchymal stem cells<br />
augments spinal cord functional recovery in scid mice<br />
model<br />
Vikram Sabapathy, Dorai Murugan, George Tharion, Sanjay Kumar<br />
755<br />
Correlation <strong>of</strong> molecular parameters with lipoplex<br />
structure and transfection eficacy in pyridinium-based<br />
cationic lipids<br />
Paria Parvizi, Emile Jubeli, Liji Raju, Nada Abdul Khalique, Ahmed<br />
Almeer, Hebatalla Allam, Maryem Al Manaa, Helge Larsen, David<br />
Nicholson, Michael D. Pungente, Thomas M. Fyles<br />
756<br />
Transplantation <strong>of</strong> human adipose tissue-derived stem<br />
cells provides behavioral improvement, clinical onset<br />
delay and life extension in mouse<br />
Hong J. Lee,Jin An, Kwang S. Kim, In J. Lim, Seung U. Kim<br />
748<br />
Assessing Small Vector Pharmacology Using the Cre-<br />
LoxP System in Mice<br />
Matthew L. Hillestad, Adam Guenzel, Michael A. Barry<br />
749 Abstract Withdrawn from Presentation<br />
750<br />
Hydrodynamic Delivery <strong>of</strong> mIL10 <strong>Gene</strong> Protects Mice<br />
from High-Fat Diet-induced Obesity and Glucose<br />
Intolerance<br />
Mingming Gao, Chunbo Zhang, Yongjie Ma, Le Bu, Linna Yan, Dexi<br />
Liu<br />
Saturday, May 18, 2013<br />
751<br />
Heparin afinity puriication <strong>of</strong> extracellular vesicles<br />
Leonora Balaj, Nadia A. Atai, Weilin Chen, Dakai Mu, Bakhos A.<br />
Tannous, Johan Skog, Xandra O. Breakeield, Casey A. Maguire<br />
752<br />
Preclinical study <strong>of</strong> AAV-sTRAIL: pharmaceutical<br />
eficacy, tissue distribution, and animal safety<br />
Dexian Zheng, Xin Wang, Wei Li, Yanxin Liu<br />
138<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013
Abstract Author Index<br />
Aartsma-Rus, Annemieke M. ........................................................609<br />
Abate-Daga, Daniel ...........................................................................716<br />
Abdalla, Renne J. ................................................................................265<br />
Abdul-Razak, Hayder H ..................................................................678<br />
Abei, Masato ........................................................................................643<br />
Abels, Erik R. .......................................................................................304<br />
Abernathy, Corinne .................................................................. 55, 721<br />
Abhyankar, Harshal ..........................................................................305<br />
Aboody, Karen S. ......................................................................648, 656<br />
Abramihina, Tatiana .........................................................................465<br />
Abuin, Allison M....................................................................................31<br />
Acchione, Mauro ................................................................................477<br />
Ackermann, Mania ...............................................................................84<br />
Ackermann, Mathias ........................................................................736<br />
Adachi, Kei ........................................................72, 78, 119, 124, 259<br />
Adair, Jennifer E. ........................................................................ 25, 421<br />
Adamo, Richard F. ...................................................................366, 372<br />
Adams, Katherine ..............................................................................508<br />
Aderca, Ileana ........................................................................................17<br />
Adjali, Oumeya ...................................................................................111<br />
Agapova, Larisa ........................................................................137, 470<br />
Agarwal, Prachi ..................................................................................537<br />
Agbandje-McKenna, Mavis ................. 4, 71, 333, 334, 335, 541<br />
Aghi, Manish K. ...................................................................................676<br />
Aguiar, Rodrigo ..................................................................................519<br />
Aguilar, Tania ......................................................................................476<br />
Ahmed, Ali Karim ..............................................................................616<br />
Ahmed, Seemin S. .................................................................................35<br />
Ahonen, Marko ...................................................................................501<br />
Aiuti, Alessandro ......................................................28, 63, 309, 517<br />
Ai-Young, Lee .......................................................................................699<br />
Akbarpour, Mahzad ................................................................323, 441<br />
Akbuga, Jülide .....................................................................................452<br />
Akinfenwa, Patricia Y. ......................................................................131<br />
Akiyama, Kazumasa ...............................................................365, 753<br />
Akpek, Gorgon ....................................................................................298<br />
Al Manaa, Maryem ............................................................................755<br />
Alan, Saadet .........................................................................................452<br />
Albert, Jessica .....................................................................................639<br />
Albert, Lee ............................................................................................471<br />
Alberti, Michael O. .............................................................................438<br />
Alexander, Ian E. ................................................................................356<br />
Alexandrov, Krill ................................................................................224<br />
Alferiev, Ivan........................................................................................372<br />
Al-Hili, Zaid A. .....................................................................................647<br />
Allam, Hebatalla.................................................................................755<br />
Allen, Cory .......................................................................... 23, 417, 641<br />
Allen, James M. ...................................................................................543<br />
Allen, Margaret D. ................................................................................96<br />
Almeer, Ahmed ...................................................................................755<br />
Almeida-Porada, Graca ...................................................................253<br />
Alonso-Ferrero, María Eugenia ...................................................678<br />
Al-Shammari, Ahmed M. ................................................................647<br />
Altaleb, Naderah ................................................................................379<br />
Alusi, Ghassan .....................................................................................561<br />
Amaishi, Yasunori ..................................................................... 16, 629<br />
Amano, Shinya U. ..................................................................................96<br />
Amante, Dinah ....................................................................................153<br />
Amante, Romain ................................................................................458<br />
Amarante, Trish .................................................................................339<br />
Ambudkar, Indu S. .............................................................................743<br />
Ammayappan, Arun .........................................................................413<br />
An, Dong Sung ....................................................................................530<br />
An, Jin .....................................................................................................756<br />
An, Songtao ..........................................................................................369<br />
Anagnostopoulos, Achilles .................................................... 27, 427<br />
Andino, Lourdes M. ..........................................................................542<br />
Ando, Dale ...............................................................................................58<br />
Ando, Miki ............................................................................................684<br />
Andre, Franck M. ...............................................................................148<br />
Ang, Sonny ............................................................ 198, 626, 650, 653<br />
Angelino, Mark D. ....................................................................260, 262<br />
Anguela, Xavier M. ..................................................................110, 488<br />
Ankoudinova, Irina ...........................................................................184<br />
Anna, Stornaiuolo .............................................................................520<br />
Annan, Anand C..................................................................................438<br />
Annoni, Andrea ........................................................................323, 441<br />
Annunziata, Patrizia .........................................................................362<br />
Annunziato, Stefano ...............................................................233, 666<br />
Ansari, Amir Mehdi ..........................................................................616<br />
Anstaett, Olivia ...................................................................................185<br />
Antico, Christopher J........................................................................324<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013<br />
139
Abstract Author Index<br />
Antignac, Corinne..............................................................................546<br />
Antoniou, Michael N. ..............................................................430, 661<br />
Anurathapan, Usanarat ........................................................398, 622<br />
Anwer, Khursheed ............................................................................461<br />
Aoki, Kazunori ..........................................................................414, 642<br />
Aouadi, Myriam.....................................................................................96<br />
Aprile, Annamaria .............................................................................420<br />
Aquilina, Stefanie ..............................................................................743<br />
Aramaki, Yukihiko .............................................................................142<br />
Arambula, Claudia ............................................................................394<br />
Aras, Rahul ...........................................................................................500<br />
Arbeit, Jeffrey M. ................................................................................560<br />
Arbel-Ornath, Michal .......................................................................283<br />
Arber, Caroline ...................................................................................305<br />
Arden, Erik ...........................................................................................538<br />
Ardinger, Jeffery .................................................................................338<br />
Arens, Anne .........................................................................................204<br />
Ariga, Masamichi ...............................................................................585<br />
Armentano, Donna ...........................................................................338<br />
Arndt, Katja .........................................................................................655<br />
Arndt, Michaela A. E. ...........................................................................21<br />
Arnold, Eric ..........................................................................................454<br />
Arnould, Sylvain .................................................................................401<br />
Aronovich, Elena L. .................................................................102, 447<br />
Aronowitz, Eric .....................................................................................11<br />
Arpke, Robert W.................................................................................168<br />
Arruda, Valder R. ...............................................................................498<br />
Artus, Alexandre ...................................................................................64<br />
Arumugam, Paritha ..........................................................................664<br />
Arumugam, Ramamani ...................................................................276<br />
Arya, Suresh ..............................................................................531, 696<br />
Asaad, Wael..........................................................................................387<br />
Aschbacher, Kirstin A. .....................................................................369<br />
Ashley, Scott ........................................................................................108<br />
Ashtari, M. ...............................................................................................53<br />
Aslanidi, George ............................................................ 333, 334, 541<br />
Aslanidi, George V. ......................................75, 118, 314, 328, 542<br />
Asokan, Aravind ....................................................................... 2, 73, 77<br />
Aswani, Vijay H. .................................................................................280<br />
Atai, Nadia A. .......................................................................................751<br />
Atala, Anthony ....................................................................................253<br />
Athanasopoulos, Takis ....................................................................734<br />
Atherton, Pamela ..................................................................................17<br />
Aubourg, Patrick ....................................................62, 278, 279, 589<br />
Aucher, Anne ..........................................................................................24<br />
Aurelian, Laure .........................................................................600, 636<br />
Auricchio, Alberto .............................................. 340, 359, 360, 499<br />
Badr, Christian E. ...............................................................................458<br />
Badros, Ashraf Z. ...............................................................................298<br />
Bae, You Han ........................................................................................556<br />
Baek, Sun-Young ................................................................................375<br />
Baert, Miranda R. M. ........................................................................313<br />
Bafico, Daniel R. ................................................................................632<br />
Bagel, Jessica .......................................................................................289<br />
Baird, Michelle....................................................................................255<br />
Bajgain, Pradip ...................................................................................622<br />
Bake, Erin .............................................................................................712<br />
Baker, David ...............................................................................188, 318<br />
Balaj, Leonora .....................................................................................751<br />
Balakrishnan, Amritha ............................................................ 45, 397<br />
Balakrishnan, Balaji .........................................................................337<br />
Baliakas, Panayotis ...........................................................................427<br />
Ballon, Douglas J. ..................................................................................11<br />
Balzer, Frederick ...................................................................................92<br />
Ban, Jae-Jun ..........................................................................................602<br />
Bangari, Dinesh ..................................................................................628<br />
Bankiewicz, Kryst<strong>of</strong> S. ...................................... 288, 594, 599, 703<br />
Bao, Gang ......................................................................... 471, 489, 567<br />
Bao, Jialing ...........................................................................................312<br />
Barber, Glen N. ....................................................................................413<br />
Barber, Jane .............................................................................................33<br />
Baricordi, Cristina ........................................................................ 28, 63<br />
Barish, Michael ...................................................................................648<br />
Barnard, Alun R. ................................................................................229<br />
Barnes, Josephine L. .........................................................................434<br />
Barnes, Justin A. .................................................................................560<br />
Barrera-L, Araceli ..............................................................................534<br />
Barrera-Lopez, Araceli ..........................................................246, 564<br />
Barrett, J. ..................................................................................................15<br />
Barry, Mary ..........................................................................................583<br />
Barry, Michael A. ................................................. 132, 481, 583, 748<br />
Bartel, Melissa A. .................................................................................... 3<br />
140<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013
Abstract Author Index<br />
Bartel, Ronnda L. ...............................................................................371<br />
Bartholomae, Cynthia C. .......................................37, 62, 210, 233,<br />
.......................................................................... 279, 518, 666, 674, 678<br />
Bartido, Shirley ...................................................................50, 61, 506<br />
Bartlett, Jeffrey S. .................................................................................48<br />
Bartolaccini, Sara ................................................................... 441, 667<br />
Bärtsch, Marc-Andrea .....................................................................639<br />
Baruchel, André ....................................................................................62<br />
Barve, Minal ......................................199, 273, 402, 503, 624, 625<br />
Barzel, Adi ............................................................................................343<br />
Bashkirova, Lisa .................................................................................277<br />
Basil, Maria C. .....................................................................................623<br />
Basner-Tschakarjan, Etiena ................................................110, 224<br />
Batshaw, Mark L. ...............................................................................128<br />
Baum, Bruce J. .....................................................................................743<br />
Baum, Christopher ........................................37, 66, 313, 320, 425<br />
Baumgart, J. .........................................................................................694<br />
Baumhover, Nick J. ...............................................................................41<br />
Baylink, David J. .................................................................................468<br />
Beadlescomb, Patricia M. ....................................................... 36, 377<br />
Bear, Adham S. ....................................................................................269<br />
Beard, Brian .........................................................................................673<br />
Beard, Brian C. .................................................................. 25, 311, 421<br />
Beard, Rachel E. .................................................................................716<br />
Beaton, Brendan ...................................................................................24<br />
Beattie, Stuart .....................................................................................274<br />
Beckman, Sarah .................................................................................611<br />
Bedell, Cynthia .......................................... 273, 351, 479, 503, 624<br />
Bee, Youn-Shen ...................................................................................740<br />
Beggs, Alan H. ............................................................................. 33, 606<br />
Beggs, Alan ..............................................................................................90<br />
Begum, Laila ................................................................................ 89, 257<br />
Behrens, Marshall .............................................................................652<br />
Beitsch, Peter ................................................................. 199, 624, 625<br />
Belay, Eyayu ...............................................................................419, 423<br />
Belbellaa, Brahim ..............................................................................278<br />
Bell, Jason B. ..............................................................................102, 447<br />
Bell, John C. ..................................................................................... 19, 22<br />
Bell, Peter ...................................................................................108, 444<br />
Bellinger, Dwight A. ..........................................................................733<br />
Bellinger, Dwight ...............................................................................667<br />
Bellio, Laura.........................................................................................420<br />
Belliveau, N. .........................................................................................708<br />
Bello, Alexander .................................................................................379<br />
Belur, Lalitha .......................................................................................364<br />
Bemelmans, Alexis-Pierre .............................................................225<br />
Bender, Christian ...............................................................................516<br />
Benedetti, Eric ....................................................................................581<br />
Benedicenti, Fabrizio .................................64, 233, 309, 517, 666<br />
Bennet, Alan ...........................................................................................59<br />
Bennett, Alan ......................................................................................508<br />
Bennett, Alan D. .................................................................................298<br />
Bennett, Jean .......................................53, 222, 224, 227, 231, 330<br />
Bennicelli, Jeannette L. .........................................................224, 227<br />
Benson, Janet ......................................................................................106<br />
Berger, Adeline ...................................................................................225<br />
Berger, Mitchel ...................................................................................703<br />
Bergeron, E. .........................................................................................694<br />
Berkley, Brown ...................................................................................307<br />
Bernal, Yvette ......................................................................................506<br />
Bernt, William ....................................................................................264<br />
Berry, Charles .....................................................................................310<br />
Bertolotti, Roger ......................................................................700, 741<br />
Betensky, Rebecca A.........................................................................283<br />
Beyer, Ines .................................................................................... 13, 201<br />
Beyers, R. J. ..............................................................................................40<br />
Bhakta, Vipul .......................................................................................561<br />
Bhargava, Ragini ......................................................................497, 571<br />
Bian, Tao ................................................................................................449<br />
Bianchi, Frayne...................................................................................508<br />
Biasco, Luca ................................................................28, 63, 309, 517<br />
Bienemann, Alison S. .......................................................................158<br />
Bifi, Alessandra .......................................................................309, 517<br />
Binder-Scholl, Gwen .........................................................................508<br />
Binder-Scholl, Gwendolyn K. ................................................ 59, 298<br />
Bing, Wang ...........................................................................................614<br />
Binley, Katie ................................................................................. 56, 232<br />
Bird, Andrew .......................................................................................618<br />
Birkett, Ashley .......................................................................................45<br />
Bishop, Nina ........................................................................................387<br />
Bisset, Darren R. ................................................................................450<br />
Bissig, Karl-Dimiter ..........................................................................454<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013<br />
141
Abstract Author Index<br />
Biswas, Arijit .............................................................................363, 580<br />
Bjornson, Christopher R. ...............................................................174<br />
Black, Aaron D. ...................................................................................227<br />
Black, Aaron H. ...................................................................................224<br />
Blake, Derek J. .....................................................................................296<br />
Blanche, Stéphane ................................................................................62<br />
Blanche, Stephane .............................................................................306<br />
Blau, C. Anthony.......................................................................419, 423<br />
Blau, Helen M. .....................................................................................255<br />
Blazar, Bruce R. ............................................................. 270, 584, 738<br />
Block, Matthew .....................................................................................17<br />
Blondeau, Johanna ............................................................................306<br />
Bloom, Matthew D. ...........................................................................332<br />
Blumenkrnz, Mark S. ..........................................................................54<br />
Blundell, Michael ...............................................................................678<br />
Bobbin, Maggie L. ..............................................................................454<br />
Bochner, Bernard H. ............................................................................70<br />
Boerner, Kathleen ...................................................................514, 725<br />
Bogan, Brooke ....................................................................................522<br />
Bogan, Dan ..............................................................................................92<br />
Bogan, Janet ................................................................................. 92, 391<br />
Bogdanov, R. ........................................................................................724<br />
Boisgerault, Nicolas ...............................................................395, 652<br />
Boissel, Sandrine ...............................................................................318<br />
Bole-Richard, Elodie ........................................................................399<br />
Bollino, Dominique .................................................................600, 636<br />
Bolyard, Chelsea M. ................................................................409, 747<br />
Bonato, Riccardo ...............................................................................433<br />
Bond, Wesley S. ..................................................................................131<br />
Bondanza, Attilio ...............................................................................197<br />
Bonifacino, Aylin ........................................................................ 30, 428<br />
Bonini, Chiara ................................................................... 28, 197, 504<br />
Bonnin, Delphine ..................................................................................52<br />
Bonten, Erik .........................................................................................702<br />
Bordignon, Claudio .................................................................197, 504<br />
Borel, Florie ...............................................................................211, 603<br />
Borelli, Primavera .............................................................................519<br />
Borg, Christophe ................................................................................399<br />
Börner, Kathleen ................................................................................123<br />
Boros, Gábor ........................................................................................147<br />
Borquez-Ojeda, Oriana ...................................................................506<br />
Borretta, Lisa ......................................................................................115<br />
Bortolotti, Francesca ............................................................... 95, 368<br />
Borzone, Roberta ..............................................................................362<br />
Boscolo, Francesca ...........................................................................465<br />
Bosnakovski, Darko ................................................................168, 169<br />
Bosque, Doyle ........................................................................................10<br />
Bossi, Giovanna ..................................................................................508<br />
Bossow, Sascha .............................................................. 405, 554, 639<br />
Botta, Salvatore ..................................................................................539<br />
Bouchier-Hayes, Lisa .......................................................................684<br />
Bougnères, Pierre ................................................................................62<br />
Bouinta, Asimina ..................................................................................27<br />
Boulad, Farid ..........................................................................................61<br />
Boulis, Nicholas M. ...........................................................................287<br />
Bourdel, Alix ........................................................................................731<br />
Bourg-Alibert, Nathalie ..................................................................615<br />
Boutin Fontaine, Marjorie ................................................................52<br />
Boyd, Maureen P. ..................................................................................48<br />
Boye, Sanford E. .................................................................................596<br />
Boye, Sanford L. ............................................................... 55, 115, 223<br />
Boye, Shannon E. ....................................................................... 55, 223<br />
Bradbury, A. M. ......................................................................................40<br />
Bradbury, Allison M. .............................................36, 377, 590, 592<br />
Braga, Luca ..............................................................................................95<br />
Brambilla, Aurora ..............................................................................504<br />
Brantly, Mark .......................................................................................545<br />
Braun, Stephen E. ..............................................................................195<br />
Breakeield, Xandra O. ...................................... 103, 286, 304, 751<br />
Bredius, Robbert G. M. ....................................................................313<br />
Breitbach, Caroline H. ........................................................................22<br />
Breitbach, Caroline J. ..........................................................................19<br />
Bremer, William G. ...............................................................................91<br />
Brendel, Christian ................................................................................66<br />
Brendler, Charles ...............................................................................555<br />
Brenner, Gary J. ..................................................................................286<br />
Brenner, Malcolm K. .........................14, 299, 300, 398, 622, 684<br />
Brennig, Sebastian ..................................................................320, 429<br />
Brentjens, Renier.....................................................................506, 651<br />
Breton, Louis R. .....................................................................................48<br />
Brewer, Joanna ........................................................................... 59, 508<br />
Brewer, Joanna E. ..............................................................................298<br />
142<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013
Abstract Author Index<br />
Bridges, Charles R. ............................................................................374<br />
Bringas, John .................................................................. 288, 594, 599<br />
Broderick, Kate E. ...................................................................153, 341<br />
Brooks, Elizabeth J. ...........................................................................276<br />
Brooks, Susan .....................................................................................295<br />
Broome, Ted ...........................................................................................88<br />
Brough, Douglas E. ............................................................................737<br />
Brown, Ami S..........................................................................................45<br />
Brown, Christine ...............................................................................648<br />
Brown, Harrison C. ...........................................................................663<br />
Brown, Jillian R. .................................................................................365<br />
Brown, Jr, Robert H. .............................................................................93<br />
Brown, Kevin ..........................................................................................51<br />
Brown, Matthew T. ..................................................................292, 491<br />
Brown, Patricia...................................................................................351<br />
Brown, Rachel E. ................................................................................434<br />
Brown, Robert ..........................................................................388, 603<br />
Brown, Ryan ........................................................................................721<br />
Brownlie, Joe .......................................................................................185<br />
Brugman, Martijn H. ..............................................................319, 425<br />
Brunetti-Pierri, Nicola ..................................... 353, 362, 499, 553<br />
Brunger, Jonathan M. .......................................................................241<br />
Brunhoeber, Elaine ...........................................................................461<br />
Brunicardi, F. C. ..................................................................................659<br />
Brunicardi, F. Charles ................................................. 272, 624, 625<br />
Brunson, Brandon L. ........................................................................590<br />
Brusko, Todd M. .................................................................................109<br />
Bu, Le ......................................................................................................750<br />
Buckvold, Megan P. ...........................................................................495<br />
Bueren, Juan Antonio ......................................................................678<br />
Buggio, Maurizio ...............................................................................438<br />
Bui, Jacquelin H. .................................................................................107<br />
Buj-Bello, Anna ...............................................33, 90, 240, 605, 606<br />
Bundschuh, Ralf .................................................................................412<br />
Bunnell, Bruce A. ...............................................................................195<br />
Burcus, Niculina ....................................................................................99<br />
Burke, Bryan P. ......................................................................................48<br />
Burke, James ...........................................................................................19<br />
Burkin, Dean J. ....................................................................................175<br />
Burnett, Ryan ......................................................................................703<br />
Burrascano, Cindy .............................................................................703<br />
Burtner, Christopher R. ........................................................311, 421<br />
Bushman, Frederic .................................................................306, 310<br />
Butts, Tiffany .......................................................................................443<br />
Byrne, Barry J............................................ 57, 60, 90,376, 446, 617<br />
Byrne, Leah C. .......................................................................................... 5<br />
Byrnes, Andrew P. .............................................................................133<br />
Byun, Jung-Ick ............................................................... 143, 598, 602<br />
Byung-Rok, Do ....................................................................................699<br />
Caccavelli, Laure ......................................................................306, 310<br />
Cagnina, Shannon..............................................................................351<br />
Cai, Ying .................................................................................................263<br />
Calabria, Andrea .......................................................28, 63, 309, 517<br />
Calcedo, Roberto ...............................................................................360<br />
Calos, Michele P. .................................................................................174<br />
Camadro, Jean-Michel .....................................................................612<br />
Camba-Colon, Joanna .........................................................................48<br />
Cameron, Brian ..................................................................................508<br />
Camisa, Barbara .................................................................................197<br />
Campell-Thompson, Martha .........................................................721<br />
Campo, John ........................................................................................184<br />
Campos, Maria ....................................................................................709<br />
Cancelas, Jose ......................................................................................426<br />
Candotti, Fabio .........................................................................307, 437<br />
Cannizzo, Stefania E. ..............................................................315, 469<br />
Cannon, Paula M. .......................................................... 183, 184, 487<br />
Cantore, Alessio .................................................. 441, 496, 666, 667<br />
Cao, Chunxia ........................................................................................335<br />
Cao, Hua .............................................................................. 13, 201, 303<br />
Cao, Hua ................................................................................................679<br />
Cao, Huibi .............................................................................................433<br />
Cao, Ou ...................................................................................................190<br />
Cao, Phuong .........................................................................................248<br />
Cappellini, Maria Domenica .........................................................420<br />
Capracotta, Sonja ..............................................................................264<br />
Carbajo, Rosangela .................................................................152, 620<br />
Carbonaro, Denise A. .......................................................................307<br />
Carlier, Pierre ......................................................................................734<br />
Carlisle, Holly ............................................................................339, 550<br />
Carlson, Daniel F. ...............................................................................738<br />
Carnes, Aaron E. .................................................................................263<br />
Carnes, Aaron .....................................................................................261<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013<br />
143
Abstract Author Index<br />
Carney, Gael G. ....................................................................................672<br />
Carroll, Maria V......................................................................................48<br />
Carruthers, K. ......................................................................................695<br />
Cartier, Nathalie ......................................................................... 62, 279<br />
Caruana, Ignazio ................................................................................392<br />
Cassinerio, Elena ...............................................................................420<br />
Castaldo, Sigismondo ......................................................................353<br />
Castello, Raffaele ...............................................................................362<br />
Castle, Michael J. ................................................................................116<br />
Castleman, William ..........................................................................721<br />
Castleton, Anna Z. .............................................................................411<br />
Castleton, Anna .....................................................................................24<br />
Casucci, Monica ..................................................................................197<br />
Caswell, Debbie ..................................................................................436<br />
Cataldi, Marcela P. .............................................................................125<br />
Catalgol, Betul .....................................................................................697<br />
Cataliotti, Alessandro ......................................................................746<br />
Catani, Joao Paulo P. .........................................................................630<br />
Cathomen, Toni ..................................................................................539<br />
Cattaneo, Roberto .............................................................................415<br />
Cavazzana-Calvo, Marina .....62, 279, 306, 310, 313, 661, 662<br />
Cederbaum, Stephen D. ..................................................................497<br />
Celis, Esteban ......................................................................................395<br />
Certoux, Jean-Marie .........................................................................399<br />
Cerullo, Vincenzo ....................................................................501, 607<br />
Cesana, Daniela .......................................................................... 64, 233<br />
Cesani, Martina ........................................................................309, 517<br />
Chérel, Yan ...........................................................................................111<br />
Chai, Jing ...............................................................................................476<br />
Chai, Kuan .............................................................................................216<br />
Chalberg, Thomas W. ..........................................................................54<br />
Chamberlain, Jeffrey S. ....................................................................543<br />
Chamberlain, Joel R. .........................................................................450<br />
Chambers, Christopher B. ........................................... 26, 200, 235<br />
Champlin, Richard E. .................................10, 621, 626, 649, 653<br />
Chan, Jerry ....................................................................... 363, 430, 580<br />
Chan, Jerry K. Y. ..................................................................................254<br />
Chan, T. ......................................................................................................15<br />
Chandler, Randy J. .............................................................................113<br />
Chandra, Manasa ...............................................................................542<br />
Chandrasekaran, Devikha..............................................................421<br />
Chang, Esther H. ................................................................................273<br />
Chang, Susan M. .................................................................................676<br />
Chang, Wen-Teng ...............................................................................723<br />
Chan-Seng, Delphine ........................................................................157<br />
Chapdelaine, Pierre ..........................................................................389<br />
Chapman, Michael S. ........................................................................336<br />
Chard, Louisa S. ..................................................................................507<br />
Charrier, Sabine .................................................................................306<br />
Charton, Karine ........................................................................610, 615<br />
Chatterjee, Saswati ...........................................................................542<br />
Chaudhuri, Aadel A. ..........................................................................712<br />
Chaurasiya, Shyambabu .................................................................403<br />
Chavez, Christopher L. ....................................................................174<br />
Chen, Alvin ...........................................................................................589<br />
Chen, Dalin ...........................................................................................474<br />
Chen, Hsing-Yu ....................................................................................348<br />
Chen, Jeremy. J.W. ..............................................................................348<br />
Chen, Li ........................................................................................464, 570<br />
Chen, Liping .........................................................................................665<br />
Chen, Mong-Jen ..................................................................................545<br />
Chen, Pao-Rong ..................................................................................723<br />
Chen, Qiumei .......................................................................................369<br />
Chen, S....................................................................................................708<br />
Chen, Stacey .........................................................................................373<br />
Chen, Thomas .....................................................................................703<br />
Chen, Vincent C. .................................................................................474<br />
Chen, Vincent ......................................................................................476<br />
Chen, Weilin .........................................................................................751<br />
Chen, Wenyi .........................................................................................474<br />
Chen, Xinglu .........................................................................................221<br />
Chen, Yifeng ...............................................................................110, 498<br />
Chen, Yvonne Y. ..................................................................................744<br />
Chen, Zhi-Ying .....................................................................................270<br />
Cheng, Seng H. .................................................................. 34, 282, 453<br />
Chen-Wichmann, Linping .................................................................66<br />
Cherel, Yan ...........................................................................................734<br />
Cherqui, Stephanie ...................................................... 493, 494, 546<br />
Cheung, Aaron ....................................................................................593<br />
Chi, Yudan .............................................................................................562<br />
Chiang, Andy .......................................................................................665<br />
Chiara, Bovolenta ..............................................................................520<br />
144<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013
Abstract Author Index<br />
Chicoine, Louis G. .................................................................................91<br />
Chien, Yin-Hsiu ........................................................................... 60, 376<br />
Chikovani, T .........................................................................................682<br />
Childers, Martin ......................................................................... 90, 391<br />
Childers, Martin K. ................................................33, 473, 606, 617<br />
Chiocca, E. Antonio .................................................................676, 690<br />
Chiodo, Vince A. .................................................................................596<br />
Chiodo, Vince ......................................................................................545<br />
Chion-Sotinel, Isabelle ....................................................................401<br />
Chiou, Shih-Hwa ................................................................................720<br />
Chiuchiolo, Maria J. ................................................................... 11, 106<br />
Chiyo, Tomoko ............................................................... 120, 176, 608<br />
Cho, Jung Jong .....................................................................................565<br />
Cho, Mong ........................................................................................ 19, 22<br />
Cho, Soo Kyung ........................................................................... 42, 163<br />
Chodon, Thinle ...................................................................................302<br />
Choi, Boyoung .....................................................................................238<br />
Choi, E. ...................................................................................................695<br />
Choi, Eun-Young .......................................................................383, 586<br />
Choi, H. ...................................................................................................708<br />
Choi, Il-Kyu ...................................................................... 129, 679, 689<br />
Choi, Ji Ye ..............................................................................................602<br />
Choi, Joung-Woo ......................................................................556, 689<br />
Choi, Ju Hyun .......................................................................................375<br />
Choi, Kyoung Baek ............................................................................138<br />
Choi, Min Jeong ..................................................................................375<br />
Choi, Uimook ............................................................................... 82, 308<br />
Choolani, Mahesh ......................................................... 363, 430, 580<br />
Chorny, Michael............................................................. 144, 366, 372<br />
Chou, Alice Y. .......................................................................................115<br />
Chou, Szu-Ting .......................................................................................44<br />
Choudhury, Sourav ................................................................................ 1<br />
Christiansen, Lauryn ............................................................... 37, 251<br />
Christiansen, Lauryn M. .................................................................258<br />
Christiansen-Weber, Trudy .................................................137, 470<br />
Chu, Jun .................................................................................................255<br />
Chu, Kirk ................................................................................7, 191, 681<br />
Chu, Kon ........................................................................... 143, 598, 602<br />
Chu, Tiffany ................................................................................137, 470<br />
Chu, Yongchao ....................................................................................561<br />
Chuah, Marinee K. .................................................................................. 8<br />
Chuah, Marinee ............................................................. 355, 496, 667<br />
Chuah, Marinne ..................................................................................354<br />
Chuang, Chen-Hua ............................................................................436<br />
Chung, Daniel C. .......................................................................160, 227<br />
Chung, Daniel ......................................................................................224<br />
Chung, Janet .........................................................................................668<br />
Chung, Su I. ..........................................................................................513<br />
Ciceri, Fabio ...............................................................................420, 504<br />
Cicieri, Fabio ........................................................................................197<br />
Cieri, Nicoletta ............................................................................ 28, 504<br />
Cingoz, Ahmet .....................................................................................697<br />
Ciriello, Giovanni ...............................................................................728<br />
Claesson-Welsh, Lena ......................................................................631<br />
Clark, K. R. ...............................................................................................91<br />
Clark, K. Reed ................................................................. 171, 182, 291<br />
Clark, Katherine A. ............................................................................366<br />
Clark, Randall ......................................................................................284<br />
Claudiani, Pamela ....................................................................359, 360<br />
Claudio, Bordignon ...........................................................................520<br />
Clayton, Nicholas P. ...........................................................................453<br />
Cleaver, Brian .........................................................................................55<br />
Clemens, Paula R. .................................................................................94<br />
Clemente, Nathalie ..............................................................................55<br />
Clevers, Hans ..........................................................................................85<br />
Cliby, William .........................................................................................17<br />
Clifford, Brian T. .................................................................................369<br />
Cloer, Caryn ..........................................................................................451<br />
Cloughesy, Timothy F. ......................................................................676<br />
Coates, Allan L. ...................................................................................433<br />
Cocchiarella, Fabienne ....................................................................347<br />
C<strong>of</strong>fey, Matt .................................................................................. 18, 652<br />
Cohen, Justus B. ............................................................. 416, 478, 566<br />
Cohen, Justus .......................................................................................285<br />
Cohen-Haguenauer, Odile .................................................................64<br />
Colahan, Patrick ....................................................................................88<br />
Cole-Strauss, Allyson .......................................................................386<br />
Collier, T. H. ..........................................................................................381<br />
Collier, Timothy J. ..............................................................................386<br />
Collins, Christopher J. ......................................................................745<br />
Collins, Shelley ......................................................................................57<br />
Colosi, Peter .........................................................................................709<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013<br />
145
Abstract Author Index<br />
Colunga, Aric .......................................................................................636<br />
Conlon, Thomas J.......................................................................... 55, 57<br />
Conlon, Thomas .......................................................................545, 721<br />
Connolly, Jeanne M. ..........................................................................366<br />
Constable, Ian J. .....................................................................................54<br />
Constantinou, Varnavas .....................................................................27<br />
Contag, Christopher H. ....................................................................255<br />
Cook, P. ......................................................................................................53<br />
Cooper, Laurence J. N. .........................................10, 198, 202, 393,<br />
......................................................431, 502, 621, 626, 649, 650, 653<br />
Cooper, Mark J. ...................................................................................344<br />
Corbel, Stephane Y. ...........................................................................255<br />
Cornetta, Kenneth .............................................................................703<br />
Corrales, Ricardo A. ..........................................................................366<br />
Cortado, Ruth ......................................................................................530<br />
Cossette, Travis L. ................................................................................55<br />
Cossette, Travis ..................................................................................721<br />
Cost, Gregory J. ...................................................................................568<br />
Cotrim, Ana P. ......................................................................................743<br />
Cotugno, Gabriella ..................................................................359, 360<br />
Coulombe, Zoé ....................................................................................389<br />
Courne, Celine ....................................................................................674<br />
Courtney, Amy .......................................................................................12<br />
Couture, Larry ..........................................................................474, 476<br />
Cox, N. R....................................................................................................40<br />
Cox, Nancy R. .............................................................................590, 592<br />
Cradick, Thomas J. ............................................. 324, 471, 489, 567<br />
Cram, Sara A. .......................................................................................589<br />
Crane, Ana M. ......................................................................................107<br />
Cranill, Palula J. .................................................................................255<br />
Crawford, Brett E. ..............................................................................365<br />
Crawford, Gregory E. .......................................................................241<br />
Cripe, Timothy P. ...............................................................................412<br />
Cristea, Sandra ...................................................................................568<br />
Crommelin, Daan ...............................................................................707<br />
Crommentuijn, Matheus H. W. .....................................................540<br />
Crosby, Catherine M. ........................................................................132<br />
Cross, Guillem .....................................................................................306<br />
Crossland, Denise L. .........................................................................198<br />
Crudele, Julie M. .................................................................................498<br />
Crystal, Ronald G. ..............................11, 106, 278, 373, 589, 727<br />
Cullis, P. ..................................................................................................708<br />
Cuneo, Nicasio ....................................................................................635<br />
Curiel, David T. .................................127, 246, 438, 558, 560, 564<br />
Curinga, Gabrielle ................................................................................32<br />
Curinga, Gabrielle M. .......................................................................673<br />
Curren, Elizabeth...............................................................................387<br />
Curtis, Sarah ........................................................................................276<br />
Cutler, Benjamin ................................................................................108<br />
Cutrera, Jeffry .....................................................................................139<br />
Cutz, Ernest..........................................................................................433<br />
Cyckowski, L. ..........................................................................................53<br />
Czarnecki, Kevin ................................................................................429<br />
Czech, Michael P. ...................................................................................96<br />
da Silva, Flavia H. ...............................................................................582<br />
Daenthanasanmak, Anusara............................................................47<br />
Daguindau, Etienne ..........................................................................399<br />
Dahlmann, Julia ..................................................................................320<br />
Dai, Jihong ............................................................................................681<br />
Dai, Mei .......................................................................................... 38, 248<br />
Dai, Mushui .............................................................................................72<br />
Dai, Yifan ...............................................................................................281<br />
Dal Ferro, Matteo..................................................................................95<br />
Dal-Cortivo, Liliane ...........................................................................306<br />
Dalkara, Deniz ......................................................................................... 5<br />
Dalle, Jean-Hughes ...............................................................................62<br />
Dandapat, Abhijit ..............................................................................169<br />
Dane, Allison P. ...................................................................................356<br />
Daneshmand, Manijeh .......................................................................19<br />
Dang, Wei ....................................................................................474, 476<br />
D’Angelo, Armando .................................................................496, 667<br />
Daniele, Nathalie ......................................................33, 90, 605, 610<br />
D”Aria, Stefania ...................................................................................362<br />
Danos, Olivier .....................................................................................731<br />
Danuta, Jarocha ..................................................................................714<br />
Dar, Gulam H. ......................................................................................156<br />
Darabi, Radbod ........................................................................168, 169<br />
Darvish, Babak .........................................................................152, 620<br />
Darvish, Daniel .........................................................................152, 620<br />
Dashk<strong>of</strong>f, Jonathan ...........................................................................283<br />
Dassie, Justin P. ...................................................................................509<br />
Dator, Dante .........................................................................................654<br />
146<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013
Abstract Author Index<br />
Daukandt, Marc ..................................................................................261<br />
Davidson, Beverly L. .........................................................................283<br />
Davidson, Michael W. .......................................................................255<br />
Davidson, Robert H. .........................................................................110<br />
Davidson, Robert J. ...........................................................................488<br />
Davila, Alejandra ...............................................................................307<br />
Davila, Marco L. ..................................................................................506<br />
Davis, Brian R. ....................................................................................107<br />
Davis, Carol ..........................................................................................295<br />
Davis, Daniel ...........................................................................................24<br />
Davis, Gregory D. ...............................................................................574<br />
Davydova, Julia .................................................... 408, 414, 705, 742<br />
Dawson, Brian ....................................................................................607<br />
Dawson, Margaret ................................................................................10<br />
d’Azzo, Alessandra ............................................................................702<br />
de Alencastro, Gustavo ...................................................................356<br />
de Andreade Pereira, Bruna .........................................................736<br />
De Bleser, Pieter .................................................................................355<br />
de Boer, Helen ...........................................................................275, 661<br />
De Deyn, Peter ....................................................................................497<br />
de Fougerolles, Antonin .................................................................463<br />
de Leeuw, Charles N. ........................................................................115<br />
de Mollerat du Jeu, Xavier .............................................................706<br />
de Moraes, Jane Z. .............................................................................658<br />
de Oliveira, Satiro ..............................................................................307<br />
De Palma, Michele .............................................................................301<br />
de Prisco, Nicola ................................................................................539<br />
De Ravin, Suk See ..............................................................................308<br />
De Saint Basile, <strong>Gene</strong>viève ............................................................662<br />
De Silva Feelixge, Harshana ..........................................................187<br />
De, Bishnu P. ................................................................................ 11, 589<br />
Dean, David A. ........................................................................................43<br />
Deconinck, Eric ..................................................................................399<br />
Deeg, H. Joachim ................................................................................525<br />
DeFeo, Anthony P. ...................................................................584, 738<br />
DeFrees, Kyle .......................................................................................745<br />
Degele, Meghan ..................................................................................273<br />
Deichmann, Annette ........................................................................210<br />
Del Vecchio, Michele ........................................................................394<br />
Delaney, Colleen .................................................................................249<br />
Delaney, Kyle .......................................................................................524<br />
Delebecque, Frederic ..........................................................................48<br />
Delhove, Juliette M. ...........................................................................435<br />
Della Valle, Patrizia .................................................................496, 667<br />
DeMaster, Amanda ............................................................................276<br />
Demonte, Marina ...............................................................................635<br />
Denapoli, Priscila M. A. .........................................................265, 658<br />
Denard, Jerome ..................................................................................445<br />
Denaro, Maria ............................................................................. 37, 674<br />
Denes, Bela ...........................................................................................507<br />
Deng, Wen-Tao .......................................................................................55<br />
Deniger, Drew C. ................................................................................502<br />
Denney, T. S. ............................................................................................40<br />
Denning, Gabriela .............................................................................528<br />
Dennis, James E. ....................................................................................96<br />
DeRavin, Suk See ..................................................................................82<br />
Derniame, Sophie ..............................................................................401<br />
Deschamps, Jack-Yves ......................................................................111<br />
Deschamps, Marina ..........................................................................399<br />
Desrosiers, Mélissa ...........................................................................225<br />
Devaux, Marie .....................................................................................111<br />
Dey, Aditi ....................................................................................... 24, 411<br />
Deyle, David R. ....................................................................................266<br />
Dhamne, Chetan ................................................................................431<br />
Di Matteo, Mario ......................................................................355, 496<br />
Di Serio, Clelia ........................................................................................63<br />
Di Stasi, Antonio ................................................................................684<br />
Di Tomaso, Tiziano ...........................................................................486<br />
Di, Wei-Li ..............................................................................................578<br />
Diago, Oscar .........................................................................................703<br />
Diamond, Scott L. ..............................................................................374<br />
Diao, Yong .............................................................................................154<br />
Diaz, Rosa .................................................................18, 395, 505, 652<br />
Dichek, David ......................................................................................559<br />
Dickey, David D. ..................................................................................327<br />
Dickson, Georges ...............................................................................734<br />
Dickson, Patricia I. ............................................................................586<br />
Dietz, Wynette M. ..............................................................................270<br />
Dighe, Niraja ........................................................................................430<br />
DiGiusto, David L. ..............................................................................668<br />
Dillberger, Alexa.................................................................................137<br />
Ding, W. ..................................................................................................694<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013<br />
147
Abstract Author Index<br />
Dionisio, Francesca .............................................................................63<br />
Discher, Dennis E. ..............................................................................523<br />
Disis, Nora ...............................................................................................13<br />
Dismuke, David J. ..................................................................................54<br />
Dissen, Gregory A. ................................................................................78<br />
Djuric, Ugljesa .....................................................................................593<br />
Do, Byung-Rok ....................................................................................722<br />
Dobbins, George C. ............................................................................127<br />
Doerler, Phillip A. .............................................................................446<br />
Doering, Christopher B. ................................................ 31, 528, 663<br />
Doering, Jon ............................................................................................33<br />
Doglioni, Claudio .....................................................................233, 666<br />
Doherty, Joseph E. .............................................................................269<br />
Doki, Yuichiro .....................................................................................749<br />
Domingo Musibay, Evidio ...................................................... 23, 641<br />
Donahue, Robert .................................................................29, 30, 428<br />
Donahue, Robert E. ..............................................................................65<br />
Dondero, Richard ..............................................................................214<br />
Dong, Biao ............................................................................................681<br />
Donnelly, Eleanor M. ........................................................................287<br />
Donnelly, Oliver ......................................................18, 395, 505, 652<br />
Donsante, Anthony .................................................................383, 586<br />
Dore, Franca ........................................................................................368<br />
Dorer, Dominik E. .................................................................................21<br />
Doria, Monica ......................................................................................340<br />
Doronin, Konstantin ........................................................................477<br />
Dorrell, Craig ............................................................................... 85, 581<br />
Dorward, Heidi ...................................................................................437<br />
Dotti, Gianpietro ............................................. 12, 14, 49, 197, 299,<br />
.......................................................................... 300, 305, 392, 394, 684<br />
Dou, Chunqing ....................................................................................616<br />
Dow, David ...........................................................................................309<br />
Dowdy, Sean ...........................................................................................17<br />
Doyal, Mark F. ......................................................................................285<br />
Doyon, Yannick ...................................................................................488<br />
Dresch, Christiane .............................................................................736<br />
Driessen, Gertjan J. A. ......................................................................313<br />
Drivas, Theodore G. ................................................................222, 231<br />
Dropulic, Boro ....................................................................................495<br />
Du, Junkai .............................................................................................616<br />
Du, Kai ....................................................................................................433<br />
Du, Liang ...............................................................................................559<br />
Duan, Cathleen ...................................................................................433<br />
Duan, Dongsheng ..................................................98, 166, 175, 613<br />
Duan, Yuyou .........................................................................................468<br />
Dubois, Kara ...........................................................................................19<br />
Dubreil, Laurence ...................................................................... 76, 111<br />
Duchateau, Philippe .........................................................................731<br />
Dufour, Brett ........................................................................................284<br />
Dukes, Joseph ......................................................................................508<br />
Dulatova, Galina .................................................................................619<br />
Dumitriu, Bogden .................................................................................65<br />
Dunbar, Cynthia E. ....................................................................... 29, 65<br />
Duncan, F. Jason ......................................................................... 39, 597<br />
Duncker, Dirk J. ...................................................................................275<br />
Duong, Jennifer .....................................................................................46<br />
Dupont, Jean Baptiste .........................................................................76<br />
Duros, Caroline .....................................................................................64<br />
Duskey, Jason ......................................................................................155<br />
Dusting, Gregory J. ..................................................................230, 740<br />
Dutilleul, Maeva .................................................................................111<br />
Düzgünes, Nejat .................................................................................576<br />
Dyka, Frank M. ..........................................................................115, 223<br />
Dyke, Jonathan P. ..................................................................................11<br />
Earley, Lauriel F. ....................................................................................72<br />
Earley, Lauriel .....................................................................................259<br />
Eastlund, Erik R. ................................................................................243<br />
Ebihara, Yasuhiro .................................................................................86<br />
Edelman, Aleksander .......................................................................731<br />
Edelman, Gerald ................................................................................273<br />
Egerer, Lisa ...........................................................................................195<br />
Egger, Erica ..........................................................................................530<br />
Eguchi, Akiko ......................................................................................706<br />
Ehrhardt, Anja ....................................................................................557<br />
Eiden, Maribeth V. .............................................................................524<br />
El Khoury, Joseph ..............................................................................304<br />
Elahi, Seyyed Mehdy ........................................................................135<br />
Elamouri, Salim S. .............................................................................248<br />
El-Amouri, Salim S. ..............................................................................38<br />
El-Attar, Laila M..................................................................................185<br />
Elazar, Menashe .................................................................................191<br />
Elder, James .........................................................................................676<br />
148<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013
Abstract Author Index<br />
Eleftheriadou, Ioanna .........................................................................67<br />
Elizabeth, Garabedian .....................................................................307<br />
Elkouby, Liron .....................................................................................110<br />
Ellis, James .................................................................................322, 593<br />
Ellis, Scott ..................................................................................... 56, 232<br />
Ellison, Stuart M. ..................................................................................67<br />
Elnaggar, Atif .......................................................................................208<br />
Emara, M.M. .........................................................................................682<br />
Embry, Addie .......................................................................................505<br />
Emery, David W. .......................................................................419, 423<br />
Emery, Lyndsey A. ................................................................................59<br />
Emri, Eszter .........................................................................................147<br />
Emri, Gabriella ...................................................................................147<br />
Emrick, Todd .......................................................................................157<br />
Endo-Takahashi, Yoko......................................................................142<br />
Engeland, Christine E. .....................................................................405<br />
Engelhardt, John F. ............................................................................535<br />
Engelhardt, Sarah ...................................................................... 21, 554<br />
Engler, Tatjana ..........................................................................126, 484<br />
Epperly, Michael W. ..........................................................................239<br />
Eren, Fatih ............................................................................................452<br />
Erez, Ayelet ..........................................................................................607<br />
Erger, Kirsten E. ....................................................................................55<br />
Erger, Kirsten ......................................................................................545<br />
Erger, Kirsten ......................................................................................721<br />
Ericson, Mark E. ....................................................................................41<br />
Escamilla-Powers, Julie ..................................................................454<br />
Escobar, Giulia ..........................................................................301, 486<br />
Escolar, Maria ........................................................................................62<br />
Esko, Jeffrey D. ....................................................................................448<br />
Esmaeili Shandiz, Alaleh ................................................................472<br />
Esmaeili-Shandiz, Alaleh ................................................................575<br />
Espenlaub, Sigrid ..............................................................................134<br />
Espinoza, Luis A. ................................................................................217<br />
Essand, Magnus .................................................................................631<br />
Estévez, Molina ..................................................................................678<br />
Eto, Yoshikatsu .........................................................................365, 585<br />
Eto, Yoshikatsu ...................................................................................753<br />
Evans, Molly ................................................................................. 30, 428<br />
Evens, Hanneke ............................................................................. 8, 496<br />
Exc<strong>of</strong>fon, Katherine J. ......................................................................327<br />
Exc<strong>of</strong>fon, Katherine J. D. A. ............................................................267<br />
Exline, Colin .........................................................................................184<br />
Exline, Colin M. ...................................................................................487<br />
Eyquem, Justin ....................................................................................401<br />
Fabre, Peggy ........................................................................................225<br />
Facher, Evan .........................................................................................500<br />
Faella, Armida .....................................................................................110<br />
Faella, Arminda ..................................................................................733<br />
Fairbanks, Carolyn A. .......................................................................364<br />
Falcione, Antonella ..............................................................................95<br />
Falcone, Laura ....................................................................................197<br />
Falk, Darin ............................................................................................739<br />
Fan, Luping ................................................................................101, 140<br />
Fang, Jin ....................................................................................................23<br />
Farag, Jacqueline ..................................................................................92<br />
Farah, Benjamin .................................................................................618<br />
Fargnoli, Anthony S. .........................................................................374<br />
Farin, Alicia M. ....................................................................................241<br />
Fariss, Robert ......................................................................................709<br />
Farmer, Andrew .................................................................................532<br />
Farrugio, Alfonso P. ...........................................................................174<br />
Faure-Kumar, Emmanuelle ...........................................................522<br />
Faust, Susan ...............................................................................108, 444<br />
Federici, Thais ....................................................................................287<br />
Feder<strong>of</strong>f, Howard J. ...........................................................................457<br />
Federspiel, Mark J. ............................................................................413<br />
Federspiel, Mark ...................................................................................17<br />
Fedsteinand, Ariel E. ........................................................................706<br />
Fehling, Hans Jörg .............................................................................445<br />
Feldman, Steve A. ..............................................................................716<br />
Felsburg, Peter J. ................................................................................311<br />
Fender, Pascal .....................................................................................552<br />
Fender, Pascal .....................................................................................729<br />
Feng, Nathan .......................................................................................656<br />
Feng, Yi ..................................................................................................533<br />
Fergus, Jeffrey .....................................................................................244<br />
Ferla, Rita ....................................................................................359, 360<br />
Fernandez-Ulibarri, Ines ...................................................................21<br />
Ferrand, Christophe .........................................................................399<br />
Ferrara, Antonella .............................................................................340<br />
Ferrari, Giuliana .................................................................................420<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013<br />
149
Abstract Author Index<br />
Ferraro, Bernadette .................................................................. 45, 397<br />
Ferreira, Jennifer ................................................................1, 384, 595<br />
Ferro, Leda ..............................................................................................61<br />
Ferrone, Soldano ...............................................................................394<br />
Festag, Marvin .......................................................................................81<br />
Fewell, Jason G. ...................................................................................461<br />
Fibbe, Willem E. .................................................................................319<br />
Fielding, Adele K. ....................................................................... 24, 411<br />
Fife, Jeremy ..........................................................................................162<br />
Figliola, Matthew J. ..............................................................................10<br />
Figueiredo, Constanca ........................................................................47<br />
Filareto, Antonio ................................................................................168<br />
Findlay, Andrew R. ............................................................................180<br />
Fine, Bruce ...........................................................................................503<br />
Fine, Eli J. ......................................................................... 324, 489, 567<br />
Finegold, Milton .......................................................................316, 466<br />
Fineman, Jeffrey R. ............................................................................369<br />
Finer, Michell .......................................................................................674<br />
Finer, Mitch ................................................................................260, 262<br />
Finer, Mitchell ............................................................................. 37, 251<br />
Finkelstein, Jeffrey ............................................................................298<br />
Finlayson, Neil ....................................................................................263<br />
Finn, Eric ...............................................................................................543<br />
Finn, Jonathan D. ...............................................................................498<br />
Finocchiaro, Liliana M. E. ...............................................................632<br />
Firrito, Claudia ...................................................................................486<br />
Fischbeck, Kenneth H. .....................................................................378<br />
Fischer, Alain ...........................................................62, 279, 306, 310<br />
Fischer, Anne C. ..................................................................................361<br />
Fischer, Ashley L. ...............................................................................243<br />
Fischer, David L. .................................................................................386<br />
Fishbein, Ilia ..............................................................................366, 372<br />
Fitzgibbons, Timothy P. .....................................................................96<br />
Flake, Alan W. ............................................................................236, 418<br />
Flanigan, Kevin M. .............................................................................180<br />
Flannery, John G. ..................................................................................... 5<br />
Flatt, Justin W. .....................................................................................477<br />
Fleming, S. M. ......................................................................................381<br />
Fleminig, Sheila M. ............................................................................386<br />
Flotte, Terence R. .............................................4, 211, 326 361, 735<br />
Fodor, Istvan ..............................................................................507, 561<br />
Foley, Erin .............................................................................................448<br />
Foley, Kevin T.......................................................................................619<br />
Follenzi, Antonia ......................................................................315, 469<br />
Foltz Greg ..............................................................................................676<br />
Fong, Mei ...............................................................................................532<br />
Fontes, Andrew ..................................................................................244<br />
Forbes, Scott P. ..........................................................................366, 372<br />
Forget, Marie A. ..................................................................................621<br />
Forsayeth, John ............................................................. 288, 594, 599<br />
Fortuno, Lowella V. ...........................................................................382<br />
Foster, Aaron .......................................................................................269<br />
Fox, Catherine .....................................................................................664<br />
Fox, Tara L. ...........................................................................................477<br />
Fraefel, Cornel ....................................................................................736<br />
Fragata, Rowell .........................................................................363, 580<br />
Francis, Sarah......................................................................................214<br />
Franco-Bourland, Rebecca E. .............................................246, 564<br />
Frederick, Amy ...................................................................................338<br />
Freemark, Michael S.........................................................................276<br />
Frelan, Megan .....................................................................................655<br />
French, Deborah L.............................................................................224<br />
French, Martyn ......................................................................................54<br />
Frenk, Esther Z. .......................................................................... 11, 106<br />
Frenzke, Marie ....................................................................................415<br />
Frey, William H. ..................................................................................364<br />
Freyvert, Yevgeniy ............................................................................568<br />
Frigault, Matthew J. ..........................................................................623<br />
Frittoli, Marta ......................................................................................420<br />
Fritz, Ashley .........................................................................................692<br />
Fronza, Raffaele .................................................. 204, 210, 274, 518<br />
Fry, Terry J. ...........................................................................................325<br />
Fu, Freddie ...................................................................... 281, 290, 614<br />
Fu, Haiyan ........................................................................... 39, 125, 597<br />
Fu, Pei-yu ..............................................................................................307<br />
Fujimoto, Naoko ......................................................................357, 715<br />
Fujisawa, Masato .....................................................................192, 654<br />
Fujita, Ryo ................................................................................................80<br />
Fukuda, Kuniaki .................................................................................643<br />
Fukuda, Takahiro .....................................................................365, 585<br />
Fulci, Giulia ..........................................................................................286<br />
Funato, Kosuke ......................................................................................11<br />
150<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013
Abstract Author Index<br />
Furth, Mark E. ........................................................................................33<br />
Furth, Mark ..........................................................................................473<br />
Furukawa, Yoichi ..................................................................................86<br />
Fyles, Thomas M. ...............................................................................755<br />
Gabriel, Nishanth ....................................................................114, 549<br />
Gabriel, Richard ............................................................ 518, 678, 738<br />
Gabrielse, Kari ....................................................................................710<br />
Gaddameedi, Rajashekhar ...................................................224, 227<br />
Gadkari, Rupali ...................................................................................536<br />
Gadkari, Rupali A. ..............................................................................114<br />
Gadue, Paul ..........................................................................................224<br />
Gahl, William A. ..................................................................................437<br />
Galanis, Evanthia ......................................................17, 23, 417, 641<br />
Galetto, Roman ...................................................................................401<br />
Galetto, Roman ...................................................................................683<br />
Galibert, Lionel ......................................................................................52<br />
Galivo, Feorillo ..........................................................................466, 581<br />
Gallina, Pierangela ..................................................................233, 666<br />
Galtseva, I. ............................................................................................724<br />
Galy, Anne ...................................................................................306, 662<br />
Gan, Chih Hao V..................................................................................678<br />
Gangemi, Mariela A. .........................................................................635<br />
Gannon, Frank ....................................................................................607<br />
Gannon, Mary......................................................................................587<br />
Ganser, Arnold .......................................................................................47<br />
Gao, Chun ..............................................................................................709<br />
Gao, Ehre ...............................................................................................444<br />
Gao, Guangping .......................4, 35, 93,128, 326, 328, 384, 512<br />
Gao, Mingming....................................................................................750<br />
Gao, Shijuan .........................................................................................194<br />
Gao, Xueqin ..........................................................................................178<br />
Gaponova, T..........................................................................................724<br />
Garcia, Elizabth ..................................................................................648<br />
Garcia, Luis...........................................................................................225<br />
Garcia-Figueroa, Jorge ..........................................................152, 620<br />
Garín, Marina I....................................................................................678<br />
Garitaonandia, Ibon .........................................................................465<br />
Garwick-Coppens, Sara ...................................................................295<br />
Gaudet, Daniel ....................................................................................274<br />
Gebremedhin, Senait .......................................................................576<br />
Gee, Adrian P. .............................................................................. 14, 299<br />
Gee, J. .........................................................................................................53<br />
Geekiyanage, Hirosha .........................................................................23<br />
Geguchadze, Ramaz ...............................................................141, 149<br />
Geiger, Sabine.........................................................................................26<br />
Geisinger, Jonathan M. .....................................................................174<br />
Genovese, Pietro ........................................................... 197, 486, 504<br />
Gentner, Bernhard ..................................................................197, 420<br />
Georger, Christophe ............................................................................76<br />
Georgiadis, Christos .........................................................................578<br />
Gerber, Mark A....................................................................................243<br />
Gerdemann, Ulrike............................................................................396<br />
Gernoux, Gwladys .............................................................................111<br />
Gerry, Andrew ....................................................................................508<br />
Gerry, Andrew B. ....................................................................... 59, 298<br />
Gersbach, Charles A. ...........167, 170, 177, 241, 292, 293, 491<br />
Getz, Matthew .....................................................................................721<br />
Geurten, Olivier ..................................................................................261<br />
Geurts, Aron ........................................................................................746<br />
Ghandeharian, Omid ............................................................................. 8<br />
Ghashghaei, Troy .................................................................................... 2<br />
Ghatrora, Rupinder K. .....................................................................434<br />
Ghisoli, Maurizio......................................................................203, 624<br />
Ghivizzani, Steve ................................................................................335<br />
Ghivizzani, Steven ................................................................................88<br />
Ghoda, Lucy Y. .....................................................................................648<br />
Gholizadeh, Shervin .........................................................................390<br />
Giacca, Mauro ............................................................................. 95, 368<br />
Gian Paolo, Rizzardi .........................................................................520<br />
Giangrande, Paloma H. ..........................................................509, 511<br />
Giannini, Caterina .............................................................................417<br />
Giardina, Patricia ..................................................................................61<br />
Gicquel, Evelyne .......................................................................610, 615<br />
Giedlin, Marty ........................................................................................58<br />
Gilbert, Daniel .....................................................................................514<br />
Gilbert, Rénald ....................................................................................135<br />
Gilbert, Shawn ....................................................................................548<br />
Gilchrist, Megan .................................................................................648<br />
Giles, April ............................................................................................116<br />
Gilkes, Janine A. ..................................................................................332<br />
Gill, Deborah R....................................................................................460<br />
Gillet-Legrand, Beatrix ....................................................................674<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013<br />
151
Abstract Author Index<br />
Giordano, Frank .................................................................................518<br />
Giovani, Michelle ...............................................................................679<br />
Gjoka, Monika .....................................................................................574<br />
Glass, Katherine A. ............................................................................241<br />
Glenn, Jeffrey S. ..................................................................................191<br />
Glikin, Gerardo C. ..............................................................................632<br />
Glimm, Hanno ...........................................................................204, 274<br />
Glorioso, Joseph C. ............................................. 285, 478, 566, 591<br />
Gochuico, Bernadette ......................................................................437<br />
Goddard, Melissa .......................................................................... 33, 90<br />
Goddard, Melissa A. ..........................................................................617<br />
Goerdt, Sergij ......................................................................................204<br />
Goetzmann, Jason................................................................................... 4<br />
Gogel, Brian M. .........................................................................199, 624<br />
Goins, William F. ................................................. 285, 478, 591, 690<br />
Gold, Michael .......................................................................................285<br />
Goldman, Frederick ..........................................................................587<br />
Goldman, Yale .....................................................................................523<br />
Goldowitz, Daniel ..............................................................................115<br />
Goldsmith, Corinne...........................................................................743<br />
Golebiowski, Diane ...........................................................................387<br />
Gombash, S. E. .....................................................................................381<br />
Gombash, Sara E. .....................................................................386, 596<br />
Gomero, Elida .....................................................................................702<br />
Gonçalves, Cristine ...........................................................................342<br />
Gonçalves, Manuel ............................................................................347<br />
Gong, Wenhui ......................................................................................369<br />
Gong, Zi ..................................................................................................495<br />
Gonzalez, Rodolfo....................................................................137, 465<br />
Gonzalez-Garay, Manuel .................................................................107<br />
Gonzalez-Gonzalez, Emilio ............................................................255<br />
Goodyer, Paul ......................................................................................247<br />
Gopal, Vijaya ..............................................................................150, 156<br />
Gordon, Myra E. .................................................................................575<br />
Gorham, Taron C. .................................................................................... 9<br />
Gorman, Kristen ................................................................................524<br />
Gorman, Wesley .................................................................................260<br />
Gorman, Wesley C. ............................................................................262<br />
Gornalusse, German ............................................................................81<br />
Gorsky, Joshua ....................................................................................544<br />
Goss, James R. .....................................................................................478<br />
Gosset, David .......................................................................................342<br />
Goto, Naoko .........................................................................................642<br />
Gottschalk, Stephen................................................................396, 400<br />
Gottschalk, Stephen M. ...................................................................410<br />
Gouble, Agnes .....................................................................................401<br />
Gouble, Agnès .....................................................................................731<br />
Goudy, Kevin ........................................................................................441<br />
Goudy, Kevin S. ...................................................................................323<br />
Gounis, Matthew................................................................................387<br />
Govindarajan, Srinath .....................................................................150<br />
Govindasamy, Lakshmanan ................................................334, 335<br />
Grabinski, Tessa .................................................................................596<br />
Gralow, Julie .........................................................................................303<br />
Grand, Francis .....................................................................................508<br />
Grandi, Paola .............................................................................416, 566<br />
Grange, Robert ......................................................................................90<br />
Grange, Robert W. ................................................................................33<br />
Gray, John T. .......................................................... 308, 671, 672, 702<br />
Gray, Steven J. ......................................................................................112<br />
Gray-Edwards, H. L. .............................................................................40<br />
Gray-Edwards, Heather ........................................................590, 592<br />
Gray-Edwards, Heather L....................................................... 36, 377<br />
Greco, Dario .........................................................................................501<br />
Greenberger, Joel S. ..........................................................................239<br />
Greene, Amy ........................................................................................657<br />
Greene, Michael R. ............................................................................527<br />
Gregory, Philip D. ............................................................ 46, 107, 183,<br />
......................................................184, 486, 487, 488, 504, 568, 678<br />
Grez, Manuel ..........................................................................................66<br />
Grieger, Joshua C. ..................................................................................54<br />
Grifin, Danielle ..................................................................................171<br />
Grifin, Danielle A. ..................................................................... 91, 172<br />
Grifiths, Duncan ...............................................................................264<br />
Grilley, Bambi .............................................................................. 14, 299<br />
Grimm, Dirk ............................................... 122, 123, 514, 516, 725<br />
Grizzle, William E. .............................................................................560<br />
Grody, Wayne W. ................................................................................497<br />
Grompe, Markus ..........................................85, 316, 466, 579, 581<br />
Grose, William E. ...............................................................................172<br />
Grossardt, Christian .........................................................................405<br />
Grosse, Stephanie ..............................................................................401<br />
152<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013
Abstract Author Index<br />
Grosso, Chantal ..................................................................................469<br />
Gruber, Harry ......................................................................................703<br />
Gruber, Harry E. .................................................................................676<br />
Gruenert, Dieter C. ....................................................... 471, 472, 575<br />
Gruh, Ina ...............................................................................................320<br />
Grunseich, Christopher...................................................................378<br />
Gu, Angel ...............................................................................................668<br />
Gu, Weiwang ........................................................................................533<br />
Guan, Xuan ...........................................................................................473<br />
Guan, Yue ..............................................................................................200<br />
Gubrij, Igor B. ......................................................................................439<br />
Guckes, Susan .....................................................................................295<br />
Guedan, Sonia .....................................................................................623<br />
Guedon, Jean-Marc ...........................................................................591<br />
Güenechea, Guillermo .....................................................................678<br />
Guenzel, Adam J. ................................................................................583<br />
Guenzel, Adam ....................................................................................748<br />
Guevara, Bruno ..................................................................................534<br />
GuhaSarkar, Dwijit ............................................................................212<br />
Guilak, Farshid ...................................................................................241<br />
Guilbaud, Mickaël ..............................................................................111<br />
Guilbault, Claire .................................................................................135<br />
Gül-Uludag, Hilal ...............................................................................462<br />
Gunther, Roland .................................................................................102<br />
Guo, Junjing..........................................................................................344<br />
Guo, Peixuan ........................................................................................515<br />
Gupta, Minnal ............................................................................298, 508<br />
Guschin, Dmitry Y. .............................................................................107<br />
Guschin, Dmitry .................................................................................184<br />
Guse, Kilian ................................................................................501, 607<br />
Guse, Kilian ..........................................................................................730<br />
Gutmann, Theresia ...........................................................................730<br />
Gutova, Margarita .............................................................................648<br />
Guy, John ...............................................................................................385<br />
Guzman-M, Karina ............................................................................564<br />
Haber, Tom ...........................................................................................719<br />
Habib, N.A. ............................................................................................682<br />
Hacein-Bey-Abina, Salima .......................62, 279, 306, 310, 313<br />
Hacke, Katrin .......................................................................................530<br />
Hackett, Neil R. ................................................................. 11, 106, 373<br />
Hackett, Perry .....................................................................................650<br />
Hackett, Perry B. .............................................................. 10, 102, 447<br />
Hadac, Elizabeth M. ..........................................................................407<br />
Haddad, Daniel J. ...............................................................................369<br />
Haddad, Marie Reine .............................................................383, 586<br />
Haemmerle, Reinhard .....................................................................422<br />
Haeusl, Martin ....................................................................................557<br />
Hafez, I. ..................................................................................................708<br />
Haft, Annelise......................................................................................316<br />
Haga, Kazunori ......................................................................................70<br />
Hagemann, Thorsten .......................................................................507<br />
Haggarty, Beth .......................................................................................46<br />
Hajri, Amor ..........................................................................................655<br />
Hakim, Chady H. ................................................. 166, 175, 295, 613<br />
Halama, Niels ......................................................................................405<br />
Halbert, Christine L. ...............................................................117, 443<br />
Hall, Bryan C. .......................................................................................447<br />
Halonen, Paavo J. ..................................................................................97<br />
Haluska, Jr, Paul .....................................................................................17<br />
Hamada, Hir<strong>of</strong>umi ............................................................................643<br />
Hamada, Katsuyuki ..........................................................................627<br />
Hamazaki, Takashi ............................................................................545<br />
Hamid, O. ..................................................................................................15<br />
Hamilton, Sara E. ...............................................................................270<br />
Hammer, Caroline ........................................................................ 33, 90<br />
Hammerschmidt-Kamper, Christiane ......................................725<br />
Hampson, David R. ...........................................................................390<br />
Hamzaoui-Nord, Silke .....................................................................405<br />
Han, Hye-Eun ......................................................................................380<br />
Han, Jingfen..........................................................................................248<br />
Han, Joohee ................................................................................408, 414<br />
Han, Joohee ................................................................................705, 742<br />
Han, Jun-Ming .....................................................................................720<br />
Han, Moon-Hee ..................................................................................143<br />
Han, Na ..................................................................................................151<br />
Han, Sang W. ................................................................... 265, 582, 658<br />
Han, Sung Yeol ....................................................................................375<br />
Han, Su-Youne .....................................................................................722<br />
Han, Zongchao ....................................................................................344<br />
Handy, Chalonda R. ...........................................................................287<br />
Hannigan, Timothy ...........................................................................611<br />
Hansen, C. .............................................................................................708<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013<br />
153
Abstract Author Index<br />
Hansen, Gesine ......................................................................................84<br />
Hanson, Leah R. .................................................................................364<br />
Haodi, A. ................................................................................................682<br />
Happle, Christine ..................................................................................84<br />
Harbottle, Richard P. ..............................................................346, 354<br />
Hardcastle, Jayson J. ................................................................. 23, 641<br />
Harding, Cary O. .................................................................................354<br />
Harding-Smith, Rebekka E. ...........................................................460<br />
Hareendran, Sangeetha ..................................................................331<br />
Harel, Asaff ...........................................................................................478<br />
Hargrove, Phillip W................................................................... 26, 424<br />
Harmon, John W. ................................................................................616<br />
Harper, Jane ................................................................................. 59, 508<br />
Harper, Scott ........................................................................................295<br />
Harper, Scott Q..........................................................................171, 182<br />
Harrington, Kevin ........................................................... 18, 395, 652<br />
Hart, Stephen L. ............................................................ 158, 434, 435<br />
Hartemink, Alexander J. .................................................................241<br />
Hartig, Jörg S. ......................................................................................554<br />
Hartmann, Lynn ....................................................................................17<br />
Hartweck, Linn ...................................................................................169<br />
Harvey, Brandon K. ...........................................................................382<br />
Hasegawa, Mamoru ..................................................... 237, 432, 442<br />
Hasegawa, Naoyuki ..........................................................................643<br />
Hashimoto, Tadafumi ......................................................................283<br />
Hashimoto-Tamaoki, Tomoko ............................................218, 638<br />
Haskins, Mark .....................................................................................360<br />
Haskins, Mark E. ...................................................................................87<br />
Haso, Waleed .......................................................................................325<br />
Hassan, Namir .......................................................................................59<br />
Hassan, Namir J. .................................................................................508<br />
Hata, Taishi ..........................................................................................749<br />
Hatley, Mark E. ....................................................................................566<br />
Hauck, Bernd.............................................................................529, 547<br />
Haugwitz, Michael ............................................................................532<br />
Haurigot, Virginia .............................................................................488<br />
Hauser, Michael ..................................................................................295<br />
Hauswirth, William W. ..............................55, 115, 223, 545, 596<br />
Havlicek, David F. .................................................................................11<br />
Hayashita-Kinoh, Hiromi .....................................................120, 608<br />
Haynes, Russell D. .............................................................................255<br />
He, Bo .....................................................................................................367<br />
He, Chenxia ..........................................................................................731<br />
He, Ran ....................................................................................4, 326, 512<br />
He, Xianghui ..............................................................................207, 404<br />
He, Zhijian ............................................................................................691<br />
Hearl, William G. ................................................................................186<br />
Hebeler, Robert ..................................................................................624<br />
Heczey, Andras ......................................................................................12<br />
Heffernan, Thomas P. .......................................................................503<br />
Heffner, Garrett C. ...................................................................251, 258<br />
Heffner, Garrett .....................................................................................37<br />
Heiland, Teri L. ...................................................................................186<br />
Heinz, Niels ................................................................................320, 429<br />
Heiss, Christian ..................................................................................369<br />
Heiss, Kirsten ......................................................................................725<br />
Heitfeld, Suzanne ..............................................................................270<br />
Heldermon, Coy D. ............................................................................332<br />
Heller, Glenn ...........................................................................................61<br />
Heller, Kristin N. ............................................................... 91, 172, 291<br />
Heller, Richard .......................................................................................99<br />
Hemming, Richard ............................................................................379<br />
Hemminki, Akseli ............................................... 501, 680, 704, 730<br />
Hendry, Jason ......................................................................................593<br />
Henley, Jill E. ........................................................................................183<br />
Henley, Jill ...................................................................................184, 487<br />
Henley, Tom .........................................................................................551<br />
Henson, Karissa .................................................................................500<br />
Hentzschel, Franziska .....................................................................725<br />
Heo, Jeong ........................................................................................ 19, 22<br />
Hergenrother, Paul J. ........................................................................403<br />
Hermann, Alex ....................................................................................655<br />
Hermetet, Gregory ...............................................................................50<br />
Hernandez, Frank J. ..........................................................................509<br />
Hernandez, Luiza I. ...........................................................................509<br />
Herschman, Harvey R. .....................................................................448<br />
Hershield, Michael ..........................................................................307<br />
Herzog, Roland W. .............................................. 109, 190, 446, 669<br />
Heslop, Helen E. ............................................................ 299, 305, 398<br />
Hess, Richard ......................................................................................437<br />
Hetzel, Miriam ....................................................................................429<br />
Hew, Paggy ...........................................................................................403<br />
154<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013
Abstract Author Index<br />
Hickey, Ray D. ......................................................................................579<br />
Hickman, Suzanne E. .......................................................................304<br />
Hickman, Theresa ................................................................................19<br />
Hicks, Martin J. ........................................................................... 11, 727<br />
High, Katherine A. ..................... 8, 110, 488, 498, 529, 547, 733<br />
Higuchi, Takashi.......................................................................365, 753<br />
Hilje, Roman ........................................................................................320<br />
Hillestad, Matthew L. .............................................................583, 748<br />
Hinderer, Christian J.........................................................................110<br />
Hirai, Yukihiko ....................................................................................601<br />
Hiramoto, Takafumi ............................................................................86<br />
Hiraoka, Kei ................................................................................. 70, 522<br />
Hirata, Roli ..............................................................................................81<br />
Hiriyanna, Suja ...................................................................................709<br />
Hironaka, Kohei .................................................................................601<br />
Hiroshima, Kenzo ..............................................................................216<br />
Hirsch, Matthew L. ............................................................................268<br />
Hirschberg, Henry ............................................................................220<br />
Hirst, Rob A. .........................................................................................435<br />
Hirvinen, Mari ....................................................................................501<br />
Hishikawa, Shuji ................................................................................121<br />
Hitt, Mary ..............................................................................................403<br />
Ho, Ivy A. W. .........................................................................................254<br />
Ho, Michelle .........................................................................................492<br />
Hoeben, Rob C. ...................................................................................313<br />
Hogrel, Jean-Yves ...............................................................................734<br />
Holczbauer, Agnes.............................................................................696<br />
Holditch, Sara J ...................................................................................746<br />
H<strong>of</strong>fman, Brad E. ................................................................................190<br />
H<strong>of</strong>fman, Lauren ................................................................................648<br />
Hoherr, Sean E. ........................................................................481, 583<br />
Hogarth, Michael ...............................................................................161<br />
Hogg, Claire ..........................................................................................435<br />
Holczbauer, Agnes.............................................................................531<br />
Holder, Melanie N. ................................................................................33<br />
Hole, Patrick ........................................................................................264<br />
Holley, Lenton .....................................................................................660<br />
Hollis, Roger P. ............................................................................ 26, 492<br />
Holmes, Michael C. .....................................46, 107, 183, 184, 486,<br />
.......................................................................... 487, 488, 504, 568, 678<br />
Holt, Robert A. ....................................................................................115<br />
Holy, Jon ................................................................................................657<br />
Holzbaur, Erika L. F. ..........................................................................116<br />
Hom, Kellie ..............................................................................................44<br />
Hoon, Pak Seoung .............................................................................699<br />
Hope, Bruce T. .....................................................................................382<br />
Horkay, Iren .........................................................................................147<br />
Horvath, Staci .....................................................................................479<br />
Hosseini, Sayed Mohammd ...........................................................616<br />
Hotta, Akitsu .............................................................................357, 715<br />
Houghton, Frank ................................................................................239<br />
Howard, Carolann .............................................................................110<br />
Howard, Douglas B. ..........................................................................382<br />
Howe, Annett .........................................................................................48<br />
Howe, Steven J. .............................................................. 434, 435, 678<br />
Howell, Craig A. ..................................................................................509<br />
Howes, Rob ..........................................................................................245<br />
Hoxie, James A. ......................................................................................46<br />
Hoyos, Valentina ......................................................................392, 394<br />
Hoyos, Valentina ................................................................................684<br />
Hsieh, Matthew .......................................................................... 30, 428<br />
Hsieh, Pei-fang ....................................................................................723<br />
Hsu, David ..................................................................................474, 476<br />
Hu, Chuhong ........................................................................................497<br />
Hu, Huimin ...........................................................................................702<br />
Hu, Jim....................................................................................................433<br />
Hu, Ruozhen ........................................................................................712<br />
Hu, Yun...................................................................................................550<br />
Hu, Zebin ...............................................................................................555<br />
Hua, Giau ...............................................................................................474<br />
Hua, Kevin L............................................................................................46<br />
Huang, George ....................................................................................285<br />
Huang, Jeremiah ................................................................................287<br />
Huang, K.W. ..........................................................................................682<br />
Huang, Liang .......................................................................................457<br />
Huang, Patricia .........................................................................474, 476<br />
Huang, Wenlin ....................................................................................194<br />
Huang, Yuping.....................................................................................574<br />
Huard, Johnny ........................178, 179, 250, 281, 290, 611, 614<br />
Hubbard, S. M. ........................................................................................40<br />
Huch, Meritxell ......................................................................................85<br />
Hudecek, Michael ..............................................................................744<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013<br />
155
Abstract Author Index<br />
Hudry, Eloise .............................................................................283, 540<br />
Hudson, J. A. ............................................................................................40<br />
Huffaker, Thomas B. .........................................................................712<br />
Hughes, Jonathan ....................................................................507, 561<br />
Hui, Daniel H. ......................................................................................110<br />
Huls, Helen ...........................................10, 198, 431, 502, 649, 650<br />
Hulsker, Margriet ..............................................................................609<br />
Humbert, L. ..........................................................................................694<br />
Hung, Chien-Ya ...................................................................................723<br />
Hung, Ming-Chuan ............................................................................723<br />
Hung, Tsuing-Jeu ...............................................................................723<br />
Hunter, David W. ................................................................................102<br />
Hurton, Lenka V. ......................................................................502, 621<br />
Hurwitz, Brian ....................................................................................747<br />
Hurwitz, Mary Y. ................................................................................131<br />
Hurwitz, Richard L. ..........................................................................131<br />
Hussain, Syed-Rehan A. ..................................................................171<br />
Hustedt, Jason ........................................................................................44<br />
Huston, Marshall W. ...............................................................275, 521<br />
Hutnick, Natalie A. ...............................................................................45<br />
Hutnick, Natalie .................................................................................341<br />
Hwang, M. ................................................................................................40<br />
Hwang, Misako ........................................................................... 36, 592<br />
Hwang, Tae-Ho .............................................................................. 19, 22<br />
Hwang, Yihharn .................................................................................106<br />
Hwu, Patrick ........................................................................................270<br />
Hwu, Wuh-Liang ........................................................................ 60, 376<br />
Hyang, Kim Ji .......................................................................................699<br />
Hyde, Stephen C. ................................................................................460<br />
Hyland, Kendra A. .............................................................................102<br />
Hyman, Bradley T. .............................................................................283<br />
Hyman, Bradley .................................................................................540<br />
Hyodo, Ichinosuke ............................................................................643<br />
Iankov, Ianko D. ..................................................................................641<br />
Iankov, Ianko .......................................................................................417<br />
Ibanez, Carlos .....................................................................................676<br />
Ibañez, Carlos .....................................................................................703<br />
Ida, Hiroyuki ........................................................................................365<br />
Ida, Hiroyuki ........................................................................................753<br />
Idirene, Idir ..........................................................................................399<br />
Ido, Hiroyuki .......................................................................................651<br />
Igarashi, Tsutomu .............................................................................228<br />
Iglesias, Diana .....................................................................................247<br />
Ihara, Hirotaka ...................................................................................150<br />
Iijima, Osamu ......................................................................................228<br />
Iinuma, Shin............................................................................................80<br />
Iizuka, Sayoko .....................................................................................365<br />
Ikawa, Eriko ............................................................................................80<br />
Ikawa, Yasuhiro ..................................................................................437<br />
Ikeda, Hiroaki ........................................................................................16<br />
Ikeda, Yasuhiro ......................................................................................68<br />
Ikeda, Yasuhiro .........................................................................726, 746<br />
Ikemoto, Tomokazu ..........................................................................121<br />
Ilett, Elizabeth .......................................................................................18<br />
Ilia, Fishbein ........................................................................................144<br />
Ilmarinen, T .........................................................................................732<br />
Imbrogno, Kara ..................................................................................290<br />
Imbrogno, Kayla ................................................................................611<br />
Impey, Helen ...........................................................................................48<br />
Inagaki, Aki ..........................................................................................703<br />
Inagaki, Akihito .......................................................................... 70, 522<br />
Ingham, Sheila J. M............................................................................265<br />
Inoue, Hiroyuki ......................................................69, 432, 440, 442<br />
Inoue, Makoto ................................................................ 237, 432, 442<br />
Inoue, Tomoko ....................................................................................442<br />
Iqball, Sharifah ......................................................................................56<br />
Irons, Eric E. ........................................................................................477<br />
Ishibashi, Hidetoshi .........................................................................120<br />
Ishii, Masaru ...........................................................................................80<br />
Islam, Saleem .........................................................................................57<br />
Ito, Hiroshi ...........................................................................................627<br />
Ito, Tomoko ..........................................................................................627<br />
Ivan, Alferiev S. ...................................................................................144<br />
Ivanovska, Irena I. .............................................................................523<br />
Ivics, Zoltan ..........................................................................................347<br />
Iwamoto, Takeo ..................................................................................585<br />
Iyombe, Jean-Paul ...................................................................293, 389<br />
Izsvak, Zsuzsanna .............................................................................347<br />
Izuka, Sayoko ......................................................................................585<br />
Jackson, Mark W.................................................................................276<br />
Jackson, Rineka .....................................................................................10<br />
Jacob, Aurélien ......................................................................................52<br />
156<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013
Abstract Author Index<br />
Jacobs, Edwin H. ................................................................................275<br />
Jagadeesh, Jayashree ........................................................................307<br />
Jäger, Dirk ...................................................................................405, 639<br />
Jahr, Holger ..........................................................................................275<br />
Jakobsen, Bent ....................................................................................508<br />
Jakobsen, Bent K. ....................................................................... 59, 298<br />
Jamet, Thibaud ....................................................................33, 90, 605<br />
Janas, Michelle L. ...............................................................................209<br />
Jang, Jae-Hyung ....................................................................................... 3<br />
Janssen, Paul M. L. .............................................................................291<br />
Janumala, Hadassah .........................................................................160<br />
Jares, Alexander ....................................................................................29<br />
Jarjour, Jordan ................................................................ 188, 193, 318<br />
Jaulin, Nicolas .....................................................................................111<br />
Javier, Francisco .................................................................................678<br />
Jay, Christopher M. ................................... 206, 272, 464, 510, 625<br />
Jayandharan, Giridhara R....................................................114, 328,<br />
................................................................331, 337, 536, 537, 542, 549<br />
Jena, Bipulendu .....................................................................................10<br />
Jensen, Michael C. ..............................................................................744<br />
Jensen, S ................................................................................................682<br />
Jeon, Daejong ......................................................................................602<br />
Jeong, Eu <strong>Gene</strong> ....................................................................................565<br />
Jeong, Hwa Yeon ................................................................................159<br />
Jeong, Ji Hoon ............................................................................349, 572<br />
Jerome, Keith R. ............................................................ 187, 188, 193<br />
Jessup, John M. ...................................................................................217<br />
Ji Hoon, Jeong ......................................................................................573<br />
Ji, Qingzhou ..........................................................................................243<br />
Jiang, Shuxia ........................................................................................511<br />
Jiang, Wei ..............................................................................................194<br />
Jiang, Yuanyuan ..................................................................................216<br />
Jiang, Yuhang .......................................................................................691<br />
Jiang, Zhong .........................................................................................512<br />
Jiang, Ziyue Karen .............................................................................215<br />
Jiao, L.R. .................................................................................................682<br />
Jie, Feilong ..................................................................................221, 563<br />
Jimenez-Orozco, Alejandro ...........................................................564<br />
Jin, Sheng-Yu ........................................................................................312<br />
Jing, Yuqi ...............................................................................................637<br />
Joglekar, Alok V. ........................................................................492, 571<br />
Johana, Nuryanti ......................................................................363, 580<br />
Johnson, Aaron ......................................................................................23<br />
Johnson, Aime K. ....................................................36, 377, 590, 592<br />
Johnson, Christopher .......................................................................660<br />
Johnson, J. A. ...........................................................................................40<br />
Johnson, Larry G. ...............................................................................439<br />
Johnson, Mai ........................................................................................215<br />
Johnston, Jennifer M. .......................................................................528<br />
Johnston, Sandra K. .............................................................................25<br />
Jolly, Douglas J...........................................................................676, 703<br />
Jone, Takako ...........................................................................................93<br />
Jones, Jeanne .......................................................................................351<br />
Jones, Pamela ......................................................................................139<br />
Jones, Steven J. M. ..............................................................................115<br />
Jones, Tara ............................................................................................473<br />
Jonlin, Erica .............................................................................................27<br />
Jordon, Andrea P. ..................................................................................46<br />
Joséphine, Charlène .........................................................................225<br />
Joseph, Glorioso C. ............................................................................416<br />
Joseph, Lauren ....................................................................................656<br />
Jossot, Laurent....................................................................................399<br />
Jost, Laurent ........................................................................................261<br />
Joubert, Romain ..............................................33, 90, 240, 605, 606<br />
Joung, J. Keith ............................................................................490, 738<br />
Jubeli, Emile .........................................................................................755<br />
Juckem, Laura .....................................................................................454<br />
Jund, Michelle D. ................................................................................270<br />
June, Carl H. .............................................................59, 298, 508, 623<br />
Jung, Hyesook .....................................................................................151<br />
Jung, Keun-Hwa ............................................................ 143, 598, 602<br />
Jung, Seungmoon ...............................................................................602<br />
Jung, Ulrike ..........................................................................................456<br />
Kabadi, Ami M. .........................................................................170, 491<br />
Kabanov, Alexander V. ...........................................................691, 693<br />
Kabasakal, Levent .............................................................................452<br />
Kaeppel, Christine.............................................................................274<br />
Kaestner, Klaus ...................................................................................581<br />
Kagele, Dominique A. ......................................................................712<br />
Kahn, Jason .............................................................................................44<br />
Kakarla, Sunitha.................................................................................400<br />
Kalaivani, V. ................................................................................536, 537<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013<br />
157
Abstract Author Index<br />
Kaler, Stephen G. ......................................................................383, 586<br />
Kaliberov, Sergey A. ..........................................................................560<br />
Kaliberova, Lyudmila N. .................................................................560<br />
Kalinin, N. .............................................................................................724<br />
Kalle, Christ<strong>of</strong> von .............................................................................210<br />
Kalos, Michael ................................................................... 59, 298, 508<br />
Kalra, Mamta .......................................................................................396<br />
Kamijima, Shuichi ..................................................................... 70, 522<br />
Kaminoh, Yuuki J. ..............................................................................180<br />
Kühn, Anna Luisa ..............................................................................387<br />
Kaminsky, Stephen M. ...............................11, 106, 373, 589, 727<br />
Kan, Shih-hsin .....................................................................................586<br />
Kanaan, Nicholas M. .........................................................................596<br />
Kaneda, Yasuhumi ...............................................................................80<br />
Kaneshiro, Kayleigh .........................................................................709<br />
Kang, Dae Hwang .................................................................................22<br />
Kang, Elizabeth ..................................................................................308<br />
Kang, Eunah .........................................................................................634<br />
Kang, Kevin ..........................................................................................574<br />
Kang, Robert ........................................................................................290<br />
Kang, Soyeong ....................................................................................375<br />
Kankaldyyan, Vazgen .......................................................................648<br />
Kannan, Vivekanandan ...................................................................508<br />
Kao, Chinghai .........................................................................................70<br />
Kapoor, Neena ....................................................................................307<br />
Kapp, Kerstin ......................................................................................686<br />
Kapranov, Philipp ..............................................................................681<br />
Karikó, Katalin ..........................................................................146, 147<br />
Karlen, Andrea D. ..............................................................................495<br />
Karli, Eerika .........................................................................................501<br />
Karponi, Garie........................................................................................61<br />
Karponi, Garyfalia ..................................................................... 27, 427<br />
Kasahara, Noriyuki ...... 70, 218, 522, 530, 638, 676, 682, 703<br />
Kasahara, Yuko N. ..............................................................................176<br />
Kashiwakura, Yuji ..............................................................................237<br />
Kaspar, Roger ......................................................................................652<br />
Kassner, Paul .............................................................................339, 550<br />
Katakowski, Joseph ..........................................................................205<br />
Kataoka, Kazunori ............................................................................350<br />
Kato, Kazunori ....................................................................................216<br />
Katz, Michael G. ..................................................................................374<br />
Kaufman, Michael L. ............................................................................26<br />
Kaufmann, Johanna K. .....................................................................554<br />
Kaufmann, Kerstin B. ..........................................................................66<br />
Kaur, Balveen ............................................................................409, 747<br />
Kauss, M. Ariel ....................................................................................542<br />
Kawabata, Masato ...................................................................192, 654<br />
Kawalekar, Omkar U. ........................................................................623<br />
Kawamura, Kiyoko ............................................................................216<br />
Kawano, Yasuhiro.................................................................................78<br />
Kawashima, Rei ..................................................................................643<br />
Kay, Mark A. ........................................... 7, 191, 271, 277, 343, 436<br />
Kaye, Frederic J. .................................................................................335<br />
Kean, Leslie ..........................................................................................650<br />
Kearns-Jonker, Mary ........................................................................468<br />
Kebriaei, Partow ...................................................................................10<br />
Keech, Naomi .........................................................................................48<br />
Keilman, George W. ..........................................................................140<br />
Kelleher, Michelle ..............................................................................232<br />
Kells, Adrian P. ............................................................... 288, 594, 599<br />
Kemp, C. J. .............................................................................................381<br />
Kemp, Christopher H. ......................................................................386<br />
Kennedy, Douglas R. .........................................................................311<br />
Kenny, Gavin D. ...................................................................................158<br />
Keramaris, Elizabeth .......................................................................296<br />
Kesari, Santosh ...................................................................................676<br />
Keshavjee, Shaf ...................................................................................433<br />
Ketzer, Patrick .....................................................................................554<br />
Khalique, Nada Abdul ......................................................................755<br />
Khan, Amir S. ..................................................................... 45, 341, 397<br />
Khan, Iram .......................................................................... 32, 318, 673<br />
Khargharia, Sanjib ...............................................................................41<br />
Khati, Makobetsa ...............................................................................189<br />
Khattak, Shahryar .............................................................................322<br />
Khayter, Cyd .........................................................................................490<br />
Khokher, Zeshan ......................................................................152, 620<br />
Khosravi-Far, R. .....................................................................................15<br />
Kicenuik, Kristin ................................................................................139<br />
Kichaev, Gleb .......................................................................................153<br />
Kiem, Hans-Peter ........................................25, 249, 311, 421, 673<br />
Kienle, Eike ................................................................................122, 123<br />
Kikuchi, Yasuhi .....................................................................................80<br />
158<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013
Abstract Author Index<br />
Kildebeck, Eric ..........................................................................324, 489<br />
Kim, Byung Sun ..................................................................................602<br />
Kim, Chang Won ...................................................................................19<br />
Kim, Dae Wook ...................................................................................138<br />
Kim, Dae-Sung ....................................................................................593<br />
Kim, Dong-Eun ...................................................................................159<br />
Kim, Hak Kyun ....................................................................................436<br />
Kim, Hyesung ......................................................................................256<br />
Kim, Hyun Ah ......................................................................................717<br />
Kim, Hyun .............................................................................................703<br />
Kim, Jaesung ..............................................................................640, 645<br />
Kim, Jane ..................................................................................................42<br />
Kim, Jeong-Min .............................................................. 143, 598, 602<br />
Kim, Ji Hyang .......................................................................................722<br />
Kim, Jin Tac ..........................................................................................722<br />
Kim, Jin-Hee .........................................................................................598<br />
Kim, Joon-Hyung ..................................................................................75<br />
Kim, Joseph J. ........................................................................................... 9<br />
Kim, Kenneth ............................................................................183, 487<br />
Kim, Keun-Sik .....................................................................................159<br />
Kim, Kwang S. .....................................................................................756<br />
Kim, Manho ................................................................................143, 602<br />
Kim, Marie ............................................................................................287<br />
Kim, Mi Kyung ........................................................................................22<br />
Kim, Mikyung ......................................................................................151<br />
Kim, Minjung .......................................................................................645<br />
Kim, Na Hyung ....................................................................................711<br />
Kim, Nak Won ...........................................................................349, 572<br />
Kim, Pyung-Hwan .............................................................................645<br />
Kim, Seung Geun...................................................................................22<br />
Kim, Seung U. ......................................................................................756<br />
Kim, So-Yoon ........................................................................................598<br />
Kim, Su Jeong ......................................................................................138<br />
Kim, Sung Wan .............................................................. 634, 640, 645<br />
Kim, Sung Wan .........................................................................685, 717<br />
Kim, Tae Won ......................................................................................565<br />
Kim, Tae-il ............................................................................................645<br />
Kim, Taekyun ......................................................................................151<br />
Kim, Yoon Jun ......................................................................................233<br />
Kim, Yoon S. .........................................................................................672<br />
Kimura, Takahiro ..................................................................................70<br />
Kincaid, Tiffany ..................................................................................529<br />
Kinchington, Paul R. .........................................................................591<br />
King, Christiaan D. ...............................................................................43<br />
King, Glenn ...........................................................................................139<br />
King, Oliver .............................................................................................93<br />
Kinnon, Christine ..............................................................................678<br />
Kinoh, Hiromi H. ................................................................................176<br />
Kipps, Thomas H. ..............................................................................502<br />
Kirn, David H. ................................................................................. 19, 22<br />
Kiroyan, Jason B. ................................................................................468<br />
Kishnani, Priya ...................................................................................276<br />
Kitagawa, Koichi ................................................................................192<br />
Kitaura, Kumiko .................................................................................150<br />
Kitchen, Scott G. ....................................................................................48<br />
Kizzire, Koby ..........................................................................................41<br />
Kleuss, Christiane .............................................................................686<br />
Klimczak, Ryan R. ................................................................................... 5<br />
Klingelhutz, Aloysius .......................................................................587<br />
Kloss, Christopher C. ........................................................................728<br />
Knight, Scott W. ..................................................................................243<br />
Knott, Christine ..................................................................................153<br />
Knovich, Mary Ann ...........................................................................253<br />
Knox, Van W. ...........................................................................................87<br />
Knutson, Keith ............................................................................ 17, 652<br />
Kobayashi, Hiroshi ..................................................................365, 585<br />
Kobayashi, Hiroshi ............................................................................753<br />
Kobayashi, Masao .................................................................................86<br />
Kobinger, Gary P. ................................................................................379<br />
Kocak, E. ................................................................................................695<br />
Koch, Walter ........................................................................................444<br />
Kochanek, Stefan .....................................................................126, 484<br />
Koeberl, Dwight D. ..................................................................276, 618<br />
K<strong>of</strong>ie, Robert M. ................................................................................283<br />
Kohn, Donald B.............................................26, 307, 492, 494, 571<br />
Kohnke, Monica .................................................................................224<br />
Kolarich, Andrew R. .........................................................................332<br />
Koldej, Rachel ........................................................................................48<br />
Komori, Junji ..........................................................................................79<br />
Konopka, Krystyna ...........................................................................576<br />
Koo, Taeyoung ....................................................................................734<br />
Koon, E. Colin ......................................................................................503<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013<br />
159
Abstract Author Index<br />
Kopin, Etana ........................................................................................542<br />
Kornegay, Joe ..........................................................................................92<br />
Kortum, Amanda ...............................................................................419<br />
Kosasih, Paula .....................................................................................744<br />
Kostomitsopoulos, N. .......................................................................682<br />
Kottke, Timothy .....................................................18, 395, 505, 652<br />
Kotton, Darrell N. ..............................................................................107<br />
Kovacs, Krisztina ...............................................................................637<br />
Koya, Richard C. .................................................................................302<br />
Koyama, Yoshiyuki ............................................................................627<br />
Kozarsky, Karen .................................................................................364<br />
Kozlowski, Merek ..............................................................................418<br />
Kozlowski, Miroslaw ........................................................................236<br />
Kozyak, Benjamin..............................................................................391<br />
Kraeusslich, Hans-Georg ......................................................123, 514<br />
Kramer, Philipp ..................................................................................107<br />
Krammer, Peter H. .............................................................................204<br />
Kraus, Jan P. .........................................................................................583<br />
Krauss, Jürgen........................................................................................21<br />
Kreader, Carol A. ................................................................................243<br />
Kreppel, Florian ............................................................ 126, 134, 484<br />
Krishnagopal, Akshaya .........................................................331, 337<br />
Krishnamoorthy, Janani..................................................................431<br />
Krishnan, Aparna ..............................................................................476<br />
Krouse, Allen ............................................................................... 30, 428<br />
Kruck, Christa .....................................................................................379<br />
Krutzke, Lea ...............................................................................126, 484<br />
Kubo, Shuji ...................................................................... 216, 218, 638<br />
Kucharski, Cezary .............................................................................162<br />
Kuhn, Joseph .............................................. 199, 402, 503, 624, 625<br />
Kuhn, N. C .............................................................................................381<br />
Kuhn, Nathan C. .................................................................................386<br />
Kuhn, Nathan M. ................................................................................596<br />
Kuivanen, Antti ......................................................................................97<br />
Kulikovskaya, Irina .................................................................298, 508<br />
Kulkarni, Aditya .................................................................................745<br />
Kumar, Padmasini .........................199, 203, 206, 272, 317, 402,<br />
.................................................................................... 503, 624, 625, 633<br />
Kumar, Priti ..........................................................................................238<br />
Kumar, Sanjay .....................................................................................754<br />
Kumaresan, Pappanaicken ............................................................431<br />
Kumari, Monika..................................................................................549<br />
Kume, Akihiro ...........................................................................121, 651<br />
Kunkel, Hana ..........................................................................................66<br />
Kunkel, Louis M. ....................................................................................93<br />
Kuo, Chung-Jung ................................................................................253<br />
Kuo, Jennifer ........................................................................................215<br />
Kurosawa, Yoshikazu .......................................................................629<br />
Kurtzberg, Joanne ................................................................................62<br />
Kustikova, Olga S. ..............................................................................425<br />
Kutner, Robert ........................................................37, 251, 258, 674<br />
Kutner, Robert H. .....................................................................260, 262<br />
Kutschera, Ina .....................................................................................279<br />
Kuzmina, L............................................................................................724<br />
Kwak, Larry W. ...................................................................................626<br />
Kwiatkowski, Stanislaw ..................................................................714<br />
Kwon, Churl-Su ...................................................................................387<br />
Kwon, Minjung ...................................................................................151<br />
Kwon, Oh-Joon ....................................................................................634<br />
Kwon, Young Jik ............................................................... 42, 163, 220<br />
Kwong, King F. ....................................................................................513<br />
Kyba, Michael ............................................................................168, 169<br />
Kyeong, Lee Hyeon ...........................................................................699<br />
Kyuri, Lee ..............................................................................................573<br />
Lachmann, Nico ............................................................... 84, 320, 429<br />
Lad, Yatish ............................................................................................232<br />
Ladd, Bryan M. ....................................................................................102<br />
Laer, Dorothee von ...........................................................................195<br />
Lagasse, Eric ...........................................................................................79<br />
Lagresle-Peyrou, Chantal .....................................................313, 661<br />
Lai, Charles P. ......................................................................................103<br />
Lai, May C. ................................................................................................54<br />
Lai, Zhaohui .........................................................................................207<br />
Lake, John R. ........................................................................................102<br />
Lakshmipathy, Uma ..........................................................................244<br />
Lalezari, Jay .............................................................................................58<br />
Lam, Paula YP .....................................................................................254<br />
Lamb, Lawrence ................................................................................587<br />
Lamont, Jeffrey ...................................................................................624<br />
Landoli, Joseph .................................................................................676<br />
Landry, Breanne ................................................................................462<br />
Langlet-Bertin, Bérangère ................................................................52<br />
160<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013
Abstract Author Index<br />
Lankester, Arjen C. ............................................................................313<br />
Lapointe, M-O. ....................................................................................694<br />
Laprise, Stephanie ............................................................................115<br />
Lapteva, Natalia ....................................................................................14<br />
Larcher, Fernando .............................................................................347<br />
Larcher, Thibaut ................................................................................111<br />
Larochelle, Andre .................................................................................65<br />
Larosa, Fabrice ...................................................................................399<br />
Larsen, Helfe .......................................................................................755<br />
Latella, Maria Carmela ....................................................................347<br />
Lau, K.-H William ..............................................................................468<br />
Lauer, Anthony ...................................................................................454<br />
Laufs, Stephanie .......................................................................210, 518<br />
Laurent, Louise ..................................................................................465<br />
Lawlor, Michael W. .................................................................... 33, 605<br />
Lawlor, Michael .......................................................................... 90, 473<br />
Lawrence, Roger ................................................................................448<br />
Lawson, Lee Ann ..................................................................................57<br />
Lay, Frank .............................................................................................616<br />
Lazar, Daniel ........................................................................................189<br />
Lazarski, Christopher A. .................................................................737<br />
Le Clerre, Diane ..................................................................................401<br />
Le Guiner, Caroline .................................................................111, 734<br />
Le Roy, Florence .................................................................................294<br />
Le, Mickaël ...........................................................................................240<br />
Le, Steven Q..........................................................................................586<br />
Leaver, T. ................................................................................................708<br />
Lebel, Carl ................................................................................................83<br />
Leber, Mathias F. ................................................................................639<br />
Lebrun, J-J. ............................................................................................694<br />
Lebuhotel, Céline .....................................................................401, 683<br />
Lecomte, Christophe ...........................................................................52<br />
Lecomte, Emilie ....................................................................................76<br />
Ledent, Philippe .................................................................................261<br />
Lee, Albert ............................................................................................472<br />
Lee, Allyson ..........................................................................................283<br />
Lee, Amy ...................................................................................................44<br />
Lee, Brendan .............................................................................128, 607<br />
Lee, Bum Sup ......................................................................................565<br />
Lee, Dahye ............................................................................................238<br />
Lee, Daniel W. ......................................................................................325<br />
Lee, Dean A. .........................................................................................621<br />
Lee, Dean ..............................................................................................431<br />
Lee, Donghoon ....................................................................................443<br />
Lee, Doo Sung .....................................................................................640<br />
Lee, Edward .........................................................................................229<br />
Lee, Ellen K. .........................................................................................497<br />
Lee, Gary K. .............................................................................................58<br />
Lee, Gary ...............................................................................................183<br />
Lee, Gwang-Hyun ..............................................................................159<br />
Lee, Hong J. ..........................................................................................756<br />
Lee, Hyeon Kyeong ...........................................................................722<br />
Lee, Hyeon Youl ..................................................................................138<br />
Lee, Hyun Woo ...................................................................................722<br />
Lee, J. .......................................................................................................708<br />
Lee, Jae Chul ........................................................................................710<br />
Lee, Janet ..............................................................................................308<br />
Lee, Jangwook .....................................................................................238<br />
Lee, Jihyun ............................................................................................623<br />
Lee, Jong Young ..................................................................................143<br />
Lee, Jun Woo ...........................................................................................22<br />
Lee, Jung Eun.......................................................................................572<br />
Lee, Jung-Sun ......................................................................................130<br />
Lee, Ki Jeong ..............................................................................339, 550<br />
Lee, Kuen Yong ...................................................................................238<br />
Lee, Kwan Hee ....................................................................................565<br />
Lee, Kyung-Mi .....................................................................................654<br />
Lee, Kyuri ..............................................................................................349<br />
Lee, Lily .................................................................................................532<br />
Lee, Min Sang ............................................................................349, 572<br />
Lee, Minhyung ..........................................................................685, 717<br />
Lee, Narae .............................................................................................195<br />
Lee, Ni-Chung .............................................................................. 60, 376<br />
Lee, Sang Kun ............................................................................143, 602<br />
Lee, Sang-Kyung.................................................................................238<br />
Lee, Soonhee .......................................................................................151<br />
Lee, Soon-Tae ................................................................. 143, 598, 602<br />
Lee, Tao-Chen .....................................................................................723<br />
Lee, Won Jai .........................................................................................130<br />
Lee, Yeon Sook .......................................................................................19<br />
Lee, Youngsook...................................................................................685<br />
Lee, Yu Kyung .........................................................................................22<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013<br />
161
Abstract Author Index<br />
Leen, Ann ..............................................................................................396<br />
Leen, Ann M. ........................................................................................398<br />
Leger, Adrien ..........................................................................................76<br />
Leger, Andrew J. .................................................................................453<br />
Léger, Thibaut .....................................................................................612<br />
Legg, Alison..........................................................................................508<br />
Lei-butters, Diana CM......................................................................535<br />
Lemaire, Laetitia ................................................................................401<br />
Lemoine, Nicholas ............................................................................561<br />
Lemoine, Nicholas R. .......................................................................507<br />
Lenarsky, Carl .....................................................................................203<br />
Leng, Qixin ..............................................................................................44<br />
Leo, Simone ...............................................................................309, 517<br />
Lerch, Thomas F. ................................................................................336<br />
Leroy, Stanley G. .................................................................................592<br />
Leslie, George J. .....................................................................................46<br />
Lesueur, Léa L. ....................................................................................148<br />
Letteri, Rachel .....................................................................................157<br />
Leung, A. ................................................................................................708<br />
Leung, Kitty .........................................................................................433<br />
Levin, Bernard R. ..................................................................................48<br />
Levine, Bruce L. ................................................................ 59, 298, 508<br />
Levy, Matthew.....................................................................................205<br />
Levy, Robert J. ................................................................ 144, 366, 372<br />
Levy, Robert .........................................................................................374<br />
Levy, Seth G. .........................................................................................548<br />
Li, Baozheng ..............................................................................541, 542<br />
Li, Brian ....................................................................................................29<br />
Li, Fanying ...............................................................................................99<br />
Li, Feng ...................................................................................................345<br />
Li, Haitang ............................................................................................668<br />
Li, Hojun ................................................................................................488<br />
Li, Hongmei ..........................................................................................715<br />
Li, Hongtao ...........................................................................................406<br />
Li, Hongwei ..................................................................... 221, 533, 563<br />
Li, Huapeng ............................................................................................... 4<br />
Li, Jia ........................................................................................4, 326, 512<br />
Li, Jianbin ....................................................................................367, 370<br />
Li, Jing.....................................................................................................155<br />
Li, Jinlong ..............................................................................................221<br />
Li, Juan .............................................................................. 367, 370, 449<br />
Li, Leping ..............................................................................................242<br />
Li, Li B. ...................................................................................................266<br />
Li, Liyuan ..............................................................................................136<br />
Li, Mengxin ............................................................................4, 128, 326<br />
Li, Pengpeng ........................................................................................255<br />
Li, Pingjuan ..........................................................................................467<br />
Li, Shaoyong .........................................................................4, 128, 326<br />
Li, Shulin ...............................................................................................139<br />
Li, Songtao ............................................................................................618<br />
Li, Tianbai .............................................................................................533<br />
Li, Tiansen ............................................................................................709<br />
Li, Wei .....................................................................................................752<br />
Li, Wenpeng .........................................................................................195<br />
Li, Xiaodan ............................................................................................404<br />
Li, Xiaomiao ...............................................................................328, 542<br />
Li, Xiaoming .........................................................................................664<br />
Li, Xuan S...............................................................................................107<br />
Li, Yan .....................................................................................................129<br />
Li, Yi ........................................................................................................640<br />
Li, Yonghai ............................................................................................529<br />
Li, Zongyi ..............................................................................................729<br />
Liadi, Ivan .............................................................................................393<br />
Liang, Zhibing .....................................................................................533<br />
Liao, Sophia H. Y. ................................................................................356<br />
Liao, Wei ................................................................................................107<br />
Lidonnici, Maria Rosa ......................................................................420<br />
Lieber, Andre .................................................13, 201, 303, 483,552,<br />
.......................................................................... 679, 680, 689, 704, 729<br />
Lifshitz, Samuel ..................................................................................503<br />
Liikanen, Ilkka ....................................................................................730<br />
Lim, In J. .................................................................................................756<br />
Lin, Amy ......................................................................................676, 703<br />
Lin, Chi-Chen .......................................................................................348<br />
Lin, Michael Z. .....................................................................................255<br />
Lin, P. .......................................................................................................708<br />
Lin, Yanni ....................................................................................489, 567<br />
Lin, Yun ........................................................................................339, 550<br />
Lin, Ziguang .........................................................................................474<br />
Lindqvist, Johan M. ...........................................................................173<br />
Lindsay, Cory R. ..................................................................................324<br />
Linette, Gerald P. ........................................................................ 59, 508<br />
162<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013
Abstract Author Index<br />
Ling, Changquan ....................................................75, 118, 314, 328<br />
Ling, Chen ........................ 75, 118, 314, 328, 333, 334, 541, 542<br />
Lipinski, Daniel M. ............................................................................229<br />
Lipshutz, Gerald S. ..................................................................497, 571<br />
Lipton, Jack W. ....................................................................................386<br />
Lisowski, Leszek .................................................................7, 191, 277<br />
Litterman, Adam J. ............................................................................270<br />
Little, Dianne .......................................................................................276<br />
Litzky, Leslie ...........................................................................................59<br />
Liu, C. ......................................................................................................682<br />
Liu, Chao Lien .....................................................................................670<br />
Liu, Da<strong>of</strong>eng ............................................................................................12<br />
Liu, Dexi .......................................................................................242, 750<br />
Liu, Enli ..................................................................................................299<br />
Liu, Guei-Sheung ......................................................................230, 740<br />
Liu, Guihua ...........................................................................................473<br />
Liu, Hao .................................................................................................300<br />
Liu, Jian ..................................................................................................295<br />
Liu, Lingfeng ........................................................................................744<br />
Liu, Lixin ...............................................................................................616<br />
Liu, Mingyao ........................................................................................433<br />
Liu, Pingguo .........................................................................................208<br />
Liu, Shi-he ...................................................................................272, 659<br />
Liu, Shu-Fen .........................................................................................723<br />
Liu, X.X. ..................................................................................................682<br />
Liu, Xiang ..............................................................................................219<br />
Liu, Xiu Y. ...............................................................................................509<br />
Liu, Xiuying ..........................................................................................511<br />
Liu, Yanxin ............................................................................................752<br />
Liu, Yijia .................................................................................................455<br />
Liu, Yuli .....................................................................................................87<br />
Liu, Yu-Ping .............................................................................................20<br />
Liu, Zhongda ........................................................................................214<br />
Livingston, Justin ...............................................................................208<br />
Llewellyn, Nick .........................................................................184, 487<br />
Lo, Mandy .............................................................................................322<br />
Loeb, Keith R. ......................................................................................140<br />
Logan, Grant J. .....................................................................................356<br />
Logg, Christopher R. ...................................................... 70, 522, 703<br />
Lombardo, Angelo ....................................................... 469, 486, 504<br />
Lomeli-R, Christian Y. ......................................................................564<br />
Lonard, David M. ...............................................................................459<br />
Long, Harry .............................................................................................17<br />
Lopez, Patricia ....................................................................................476<br />
Lopez, Veronica M. ............................................................................635<br />
Lorain, Stéphanie ..............................................................................225<br />
Lorincz, Matthew ..............................................................................322<br />
Lostal, William.......................................................................................98<br />
Lothrop Jr, Clinton D. .......................................................................498<br />
Louis, Chrystal ....................................................................................396<br />
Low, Walter C. ...........................................................................364, 495<br />
Lu, Aiping ..................................................... 178, 179, 250, 290, 611<br />
Lu, Jiamiao ............................................................................................271<br />
Lu, Maomin ..........................................................................................533<br />
Lu, Pei Juan ..........................................................................................296<br />
Lu, Peijuan ............................................................................................451<br />
Lu, Qi Long .................................................................................296, 451<br />
Lu, Rong....................................................................................................29<br />
Lu, Taihe ................................................................................................671<br />
Lu, Yi .......................................................................................................136<br />
Lu, Yuan ............................................................................... 75, 118, 335<br />
Lu, Yuanqing ........................................................................................545<br />
Lu, Zhi Hong ........................................................................................560<br />
Lu, Zhuo-zhuang ......................................................................644, 729<br />
Lucas, Cherry ......................................................................................232<br />
Lucke, Colleen .......................................................................................... 9<br />
Luke, Jeremy ........................................................................................185<br />
Lum, Lawrence ...................................................................................711<br />
Luna Velez, María Victoria.............................................................330<br />
Lundberg, Cathryn ...............................................................................99<br />
Luo, Jie .........................................................................................221, 563<br />
Luo, Jun .....................................................................................................96<br />
Luo, Xianghua .....................................................................................414<br />
Luo, Yuxia .............................................................................................338<br />
Lüttge, Doreen .......................................................................................84<br />
Lutzko, Carolyn ..................................................................................426<br />
Lyde, Randolph ....................................................................................... 9<br />
Lydie, Guigand ....................................................................................111<br />
Lyle, Meghan .......................................................................................670<br />
Lythgoe, Mark F. .......................................................................158, 434<br />
Ma, Hsin-I .............................................................................................720<br />
Ma, Wenqin ................................................................................541, 542<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013<br />
163
Abstract Author Index<br />
Ma, Yongjie ...........................................................................................750<br />
Ma, Zhijun .............................................................................................671<br />
Mac, Helen ............................................................................................224<br />
MacAldaz, Margarita E. ...................................................................390<br />
MacDonald, Kelvin D. .......................................................................361<br />
Machen, Laurie .........................................................................141, 149<br />
Machluf, Marcelle ..............................................................................719<br />
Machuca, Tiago N. .............................................................................433<br />
Machuca-R, Catalina ........................................................................534<br />
Mack, David .........................................................................................473<br />
Mackall, Crystal L. .............................................................................325<br />
MacLaren, Robert E. .........................................................................229<br />
MacVicar, B. ..........................................................................................708<br />
Madinaveitia-V, Juan A. ...................................................................246<br />
Madoiwa, Seiji .....................................................................................121<br />
Madtes, David K. ................................................................................117<br />
Maeder, Morgan L. ............................................................................738<br />
Magee, Mitchell J. ...............................................................................624<br />
Maggio, Aurelio .....................................................................................61<br />
Magnani, Zulma..................................................................................504<br />
Magno, Aaron L. ....................................................................................54<br />
Maguire, A. ..............................................................................................53<br />
Maguire, Albert M. ..................................................................224, 227<br />
Maguire, Casey A. ......................................................... 103, 540, 751<br />
Maguire-Zeiss, Kathleen .................................................................457<br />
Mah, Cathryn S. .....................................................................................57<br />
Mahadevan, Lavanya ............................................................................. 9<br />
Mahajan, Vivek ...................................................................................693<br />
Mahdipoor, Parvin ............................................................................213<br />
Mahendravada, Aruna ............................................................. 49, 300<br />
Maiti, Sourindra N. ............................................................................626<br />
Maiti, Sourindra .......................................................................431, 502<br />
Maitland, Stacy ..........................................................1, 384, 387, 595<br />
Majka, Marcin .....................................................................................714<br />
Makkia, Rasha .....................................................................................344<br />
Malani, Nirav .............................................................................306, 310<br />
Male, Frances ......................................................................................310<br />
Malech, Harry L. ......................................................................... 82, 308<br />
Malerba, Alberto ................................................................................734<br />
Malik, Alock ................................................................................. 92, 391<br />
Malik, Punam ......................................................................................664<br />
Maltais, Chantale ..................................................................................83<br />
Malvilio, Fulvio ...................................................................................734<br />
Mamchaoui, Kamel ...........................................................................491<br />
Mandel, R. J. .........................................................................................381<br />
Mandel, Ronald J. .....................................................................386, 721<br />
Mandelli, Giacomo ............................................................................420<br />
Manfredsson, F. P. ..............................................................................381<br />
Manfredsson, Fredric P. ........................................................386, 596<br />
Mangena, Ramu .................................................................................551<br />
Maniati, Eleni ......................................................................................507<br />
Mann, Trenae ......................................................................................200<br />
Mannioui, Cécile Schiffer ...............................................................683<br />
Manoli, Irini .........................................................................................280<br />
Mao, Qin ................................................................................................512<br />
Mao, Sunnie .........................................................................................692<br />
Mao, Yingying .....................................................................................533<br />
Maples, Phillip B. ..................................... 199, 203, 206, 234, 272,<br />
....................... 352, 402, 464, 503, 510, 570, 577, 624, 625, 633<br />
Maranzano, Michael .........................................................................276<br />
Marchetti, Dario .................................................................................392<br />
Marcus, Nancy ....................................................................................211<br />
Mardini, Osama ..................................................................................103<br />
Marescau, Bart ...................................................................................497<br />
Margaritis, Paris ................................................................................733<br />
Margulies, Kenneth B. .....................................................................374<br />
Marinova, Ekaterina ............................................................................12<br />
Markert, Chad .....................................................................................473<br />
Marktel, Sarah ....................................................................................420<br />
Markusic, David .................................................................................669<br />
Markusic, David M. ...........................................................................109<br />
Marolleau, Beatrice .............................................................................76<br />
Marquez, Laura I. ...............................................................................466<br />
Marrocco, Elena .................................................................................539<br />
Mars, Wendy ........................................................................................611<br />
Marsh, A. ..................................................................................................15<br />
Marshall, K. .............................................................................................53<br />
Marsic, Damien ........................................................................335, 548<br />
Martell, L. A. ............................................................................................15<br />
Marti, Guy P. .........................................................................................616<br />
Martin, D. R. ............................................................................................40<br />
Martin, Daniel ........................................................................................57<br />
164<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013
Abstract Author Index<br />
Martin, Douglas R. .......................................36, 377, 387, 590, 592<br />
Martin, John .........................................................................................338<br />
Martin, Samia .......................................................................33, 90, 606<br />
Martinelli, Valentina ........................................................................368<br />
Martinez-F, Francisco ................................................. 246, 534, 564<br />
Maruyama, Kazuo ...................................................................142, 145<br />
Marzulli, Marco ........................................................................416, 566<br />
Massie, Bernard .................................................................................135<br />
Mastaglio, Sara ...................................................................................504<br />
Masurier, Carole ............................................................................ 33, 90<br />
Mata, Melinda .....................................................................................400<br />
Matalon, Reuben ...................................................................................35<br />
Matar, Majed ........................................................................................461<br />
Matern, Dietrich .................................................................................583<br />
Matrai, Janka .......................................................................................496<br />
Matrobattista, Enrico ......................................................................707<br />
Matsui, Hideto ....................................................................................357<br />
Matsumoto, Priscila K. ..........................................................582, 658<br />
Mattar, Citra .................................................................... 363, 430, 580<br />
Matthews, Carolyn M. ......................................................................503<br />
Mattoo, Abid ........................................................................................217<br />
Maus, Marcela V. ...................................................................................59<br />
Mavilio, Fulvio ............................................................................ 76, 347<br />
Maynard, Ryan ....................................................................................525<br />
Mays, Lauren E. .....................................................................................71<br />
Mazarakis, Nicholas D. .......................................................................67<br />
Mazzacurati, Lucia ..................................................................416, 566<br />
Mazzieri, Roberta ..............................................................................301<br />
McAnulty, Robin J. .............................................................................434<br />
McBride, Jodi .......................................................................................284<br />
McCaffrey, Anton P. ...........................................................................454<br />
McCart, Andrea ..................................................................................561<br />
McCarty, Douglas ..................................................................................39<br />
McCarty, Douglas M. ...............................................................125, 597<br />
McClure, Diane ...................................................................................461<br />
McCown, Thomas J. ..........................................................................112<br />
McCraw, Dustin ..................................................................................336<br />
McCurdy, V. J. ..........................................................................................40<br />
McCurdy, Victoria J. ..............................................36, 377, 590, 592<br />
McDoom, Isaam ....................................................................................55<br />
McElroy, Amber N. ............................................................................738<br />
McGinley, Lisa M. ...............................................................................475<br />
McGinn, Arlo N. ..................................................................................685<br />
McGrath, John A. ................................................................................578<br />
McGrath, John ........................................................................................80<br />
McInerny, Simone C. .........................................................................115<br />
McIvor, R. Scott .................................................... 102, 364, 447, 495<br />
Mckay, Tristan R. ...............................................................................435<br />
McKenna, Robert ..................................................................................71<br />
McLeod, Cameron W. .......................................................................158<br />
McNeish, Iain ......................................................................................561<br />
McNiece, Ian ...........................................................................................10<br />
McVicar, Daniel W..............................................................................308<br />
Mead, Andrew ............................................................................ 92, 391<br />
Meadows, Aaron ........................................................................ 39, 597<br />
Medrano, Ruan Felipe V. .................................................................630<br />
Medzikovic, Lejla ...............................................................................369<br />
Mei, Zhuyong .......................................................................................299<br />
Melcher, Alan ...........................................................18, 395, 505, 652<br />
Melo, Sandra ........................................................................................277<br />
Memmott, Trevor ..............................................................................208<br />
Mendeleeva, L. ....................................................................................724<br />
Mendell, Jerry R. ....................................................91, 172, 291, 295<br />
Mendoza, Janess ................................................................................341<br />
Mendoza, Janess M. ..........................................................................153<br />
Meng, Xianmei ....................................................................................468<br />
Merchan, Jaime ...................................................................................637<br />
Merigan, William H. ............................................................................... 5<br />
Merkel, Christian A. ..........................................................................630<br />
Merkel, Olivia ......................................................................................711<br />
Merkert, Sylvia ......................................................................................84<br />
Merlin, Simone .........................................................................315, 469<br />
Merten, Otto W. .....................................................................................52<br />
Merwin, Miles J. ..................................................................................344<br />
Messaddeq, Nadia ........................................................ 240, 278, 605<br />
Metelitsa, Leonid ..................................................................................12<br />
Metzge, Mark ..........................................................................................30<br />
Metzger, G. ............................................................................................695<br />
Metzger, Joseph M. ............................................................................538<br />
Metzger, Mark .....................................................................................428<br />
Meunier, M. ..........................................................................................694<br />
Meyer, Nancy .......................................................................................336<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013<br />
165
Abstract Author Index<br />
Mi, Tiejuan ...................................................................... 198, 502, 621<br />
Miao, Carol H. ....................................................... 101, 140, 665, 670<br />
Miao, Lei ................................................................................................691<br />
Michel, Gérard .......................................................................................62<br />
Midoux, Patrick ..................................................................................342<br />
Miest, Tanner S. ..................................................................................415<br />
Mikals, Kyle .............................................................................................71<br />
Mikkelsen, Tom ..................................................................................676<br />
Miko, Edit ..............................................................................................147<br />
Milazi, Stephanie ...............................................................................451<br />
Milczarek, Olga ...................................................................................714<br />
Miller, A. D. ...........................................................................................117<br />
Miller, Andrew ....................................................................................387<br />
Miller, Auston ......................................................................................168<br />
Miller, Christopher P. .......................................................................423<br />
Miller, Christophre P.........................................................................419<br />
Miller, Dusty ........................................................................................443<br />
Miller, Erin ............................................................................................542<br />
Miller, Jeffrey C. ..................................................................................184<br />
Miller, Jr, Francis J. .............................................................................511<br />
Miller, Timothy J. ...............................................................................500<br />
Millington, Michelle L. ........................................................................48<br />
Millroy, Laura ......................................................................................189<br />
Mills, Jason A. ......................................................................................224<br />
Mimuro, Jun .........................................................................................121<br />
Min Sang, Lee ......................................................................................573<br />
Mineno, Junichi ................................................................ 16, 629, 651<br />
Mingozzi, Federico ..................................................................110, 498<br />
Mingozzi, Frederico ............................................................................... 8<br />
Mintz, P.J. ...............................................................................................682<br />
Mintzer, Esther ...................................................................................188<br />
Mir, Lluis M. .........................................................................................148<br />
Miranda, Maria V. ..............................................................................632<br />
Mishra, Suparna .................................................................................307<br />
Miskin, James ......................................................................................232<br />
Mitchell, Erin .................................................................................. 33, 90<br />
Mitchell, James B. ..............................................................................743<br />
Mitchell, Marilyn...................................................................................92<br />
Mithbaokar, Pratibha .......................................................................553<br />
Mitrophanous, Kyriacos ......................................................... 56, 232<br />
Mitsuyasu, Ronald ................................................................................58<br />
Mittal, Suresh K. .................................................................................628<br />
Miura, Yoshi .........................................................................................414<br />
Miura, Yoshiaki .............................................................. 408, 705, 742<br />
Mixson, Archibald J. .............................................................................44<br />
Miyake, Koichi ................................................................ 228, 358, 601<br />
Miyake, Noriko .........................................................................228, 358<br />
Miyamoto, Shohei..................................................69, 432, 440, 442<br />
Mizandara, M. .....................................................................................682<br />
Mizoguchi, Yoko ....................................................................................86<br />
Mizuguchi, Hiroyuki .........................................................................482<br />
Mizukami, Hiroaki ..................................................................121, 651<br />
Mizushima, Tsunekazu ...................................................................749<br />
Moal, Christelle ...............................................33, 90, 240, 605, 606<br />
Moats, Rex A. .......................................................................................648<br />
Mochizuki, Shinji ..................................................................................86<br />
Modlich, Ute...............................................................................422, 425<br />
Moi, Davide ................................................................................301, 486<br />
Moisyadi, Stefan .................................................................................569<br />
Mok, Steven..........................................................................................302<br />
Mokhtari, Saloomeh .........................................................................253<br />
Monassier, Laurent ...........................................................................278<br />
Monette, Sébastien .................................................................373, 589<br />
Monjaret, François ............................................................................615<br />
Montazeri Aliabadi, Hamidreza ..................................................213<br />
Montgomery, Chrystal .....................................................................171<br />
Montgomery, Chrystal L. ........................................................ 91, 291<br />
Montiel-Martínez, Ana G. ...............................................................604<br />
Montini, Eugenio ..........................28, 63, 64, 233, 309, 517, 666<br />
Montus, Marie .....................................................................................734<br />
Moon, Jangsup ....................................................................................598<br />
Mooney, Michael ................................................................................378<br />
Moore, Malcolm A.S. .........................................................................727<br />
Moore, Theodore ...............................................................................307<br />
Mookherjee, Suddhasil ...................................................................709<br />
Moore, Andrea R. ...............................................................................681<br />
Mooreield, Emily ..............................................................................473<br />
Moreo, Andrew ..................................................................................182<br />
Morgan, Mary .........................................................................................65<br />
Morgan, Richard A. ...........................................................................716<br />
Mori, Masaki ........................................................................................749<br />
Moon, Anne ..................................................................................... 19, 22<br />
166<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013
Abstract Author Index<br />
Morishita, Naoya .....................................................................192, 654<br />
Moritz, Thomas ......................................................84, 320, 422, 429<br />
Morrison, Kyle P. ................................................................................140<br />
Morrison, Nancy ...................................................................................36<br />
Morrow, Matthew P .............................................................................45<br />
Moser, Anne ............................................................................................37<br />
Mosquea, Leocadia M. .....................................................................453<br />
Moss, Bernard .....................................................................................681<br />
Mota, Daniel ........................................................................................656<br />
Moughon, Diana .................................................................................215<br />
Moullier, Philippe .........................33, 76, 90, 111, 445, 606, 734<br />
Moulton, Hong M. ..............................................................................451<br />
Mrugala, Maciej M. ...............................................................................25<br />
Mu, Anbin .............................................................................................374<br />
Mu, Dakai ..............................................................................................540<br />
Mu, Dakai ..............................................................................................751<br />
Mu, Xiaodong ......................................................................................179<br />
Mucci, Adele ...........................................................................................84<br />
Mueller, Ann-Kristin ........................................................................725<br />
Mueller, Christian ..................................... 211, 361, 388, 603, 735<br />
Mueller, Martin ...................................................................................123<br />
Muenzer, Joseph .................................................................................495<br />
Muether, Nadine ................................................................................557<br />
Mukhopadhyay, Debabrata ...........................................................652<br />
Mulhern, Orla ......................................................................................184<br />
Mulhern, Orla L. .................................................................................487<br />
Müller-Kuller, Uta .................................................................................66<br />
Muñoz-Fuentes, Rosa M. ................................................................604<br />
Munye, Mustafa M. ..................................................................434, 435<br />
Muramatsu, Hiromi ................................................................146, 147<br />
Muramatsu, Shin-ichi .............................................................. 60, 376<br />
Murlidharan, Giridhar .......................................................................... 2<br />
Murrey, Darren ........................................................................... 39, 597<br />
Murugan, Dorai ..................................................................................754<br />
Mussolino, Claudio ...........................................................................539<br />
Muthumani, Karuppiah ............................................................. 9, 341<br />
Muthuswamy, Jit ................................................................................100<br />
Myers, Doug ............................................................................................14<br />
Myles, Devin J. ............................................................................. 45, 397<br />
Naash, Muna I. ....................................................................................344<br />
Naissi, Naiseh .........................................................................688, 713<br />
Nagabhushan Kalburgi, Sahana ..................................................112<br />
Nagamoto, Yasuhito .........................................................................482<br />
Nagasaki, Masao ...................................................................................86<br />
Nair, Nisha ....................................................................................... 8, 496<br />
Nair, Sukesh C. ....................................................................................114<br />
Najbauer, Joseph ................................................................................656<br />
Najjar, Amer .........................................................................................650<br />
Nak Won, Kim .....................................................................................573<br />
Nakade, Koji .........................................................................................643<br />
Nakai, Hiroyuki ...............................................72, 78, 119, 124, 259<br />
Nakajima, Yoshiyuki ............................................................................79<br />
Nakamura, Kazuhiro ...........................................................................86<br />
Nakamura, Masataka .......................................................................651<br />
Nakamura, Shin-ichiro ....................................................................482<br />
Nakanishi, Yoichi ...................................................69, 432, 440, 442<br />
Nakano, Yuki .......................................................................................440<br />
Nakano, Yuri ........................................................................................643<br />
Nakashima, Hiroshi ..........................................................................690<br />
Nakauchi, Hiromitsu ...................................................... 86, 365, 753<br />
Naldini, Luigi ........................................ 63, 64, 197, 233, 301, 309,<br />
......................................................................... 323, 441, 469, 486, 496,<br />
.................................................................................... 504, 517, 666, 667<br />
Nam, Hye Yeong .................................................................................645<br />
Nam, Hyun-Joo ......................................................................................71<br />
Nam, Jeong A. ......................................................................................572<br />
Nam, Kihoon ..............................................................................685, 717<br />
Nan, Zenhong ......................................................................................495<br />
Nan, Zhenhong ...................................................................................364<br />
Narayan, Poornima K. L. .................................................................267<br />
Nargarkhan, Simon ...........................................................................169<br />
Nariai, Naoki ..........................................................................................86<br />
Narukawa, Munetoshi .....................................................................530<br />
Narusawa, Megumi .................................................................432, 442<br />
Nathan, Erica .......................................................................................156<br />
Nathwani, Amit ........................................................................363, 580<br />
Naughton, Bartholomew J. .................................................... 39, 597<br />
Nawa, Akihiro .....................................................................................627<br />
Nayak, Ramesh ...................................................................................426<br />
Nayak, Sushrusha ..............................................................................446<br />
Naylor, Stuart .............................................................................. 56, 232<br />
Neal, Tawny L. .....................................................................................174<br />
Neduva, Victor ....................................................................................309<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013<br />
167
Abstract Author Index<br />
Negishi, Yoichi ..........................................................................142, 145<br />
Negre, Olivier ......................................................................................674<br />
Neiswender, James............................................................................212<br />
Nektaria, Andronikou ......................................................................706<br />
Nelson, Carol A. ..................................................................................453<br />
Nelson, Jared .......................................................................................263<br />
Nemnaitis, John ..................................................................................317<br />
Nemunaitis, Derek ............................................................................273<br />
Nemunaitis, John J. ....................................15, 199, 203, 206, 219,<br />
...........................................234, 272, 273, 351, 352, 402, 459, 464,<br />
............................................479, 503, 510, 570, 577, 624, 625, 659<br />
Nerem, Robert M. ..............................................................................475<br />
Nettelbeck, Dirk .................................................................................483<br />
Nettelbeck, Dirk M. ................................................................... 21, 554<br />
Ng, Philip...............................................................................................353<br />
Ngo, Minhtran C. ................................................................................396<br />
Nguyen, Tran T....................................................................................690<br />
Nichol, Ge<strong>of</strong>f ...........................................................................................58<br />
Nichols, Timothy ...............................................................................667<br />
Nicolay, Jan P. .......................................................................................204<br />
Nicolis di Robilant, Benedetta .....................................................197<br />
Nicholls, J.P. ..........................................................................................682<br />
Nichols, Timothy C............................................................................733<br />
Nicholson, David ................................................................................755<br />
Nicolson, Sarah C. .................................................................................74<br />
Nicolson, Tamara ..................................................................................48<br />
Nie, Daibang ........................................................................................281<br />
Niemeyer, Glenn P. ............................................................................498<br />
Nienhuis, Arthur W. ..........................................................................672<br />
Nienhuis, Arthur ................................................................................702<br />
Nieses, Amanda ..................................................................................468<br />
Nilsson, Bradley L. ...............................................................................43<br />
Nilsson, Jonas ......................................................................................321<br />
Nimgaonkar, Vishwajit ....................................................................239<br />
Niopek, Dominik ................................................................................514<br />
Nishikawa, Shinichi ..........................................................................753<br />
Nishimura, Junichi ............................................................................749<br />
Nitahara-Kasahara, Yuko .....................................................120, 608<br />
Nixon, Alan J. ...........................................................89, 257, 297, 718<br />
No, Daniel ...................................................................................152, 620<br />
Noble, Misty L. ..........................................................................101, 140<br />
Noh, Moon Jong ..................................................................................565<br />
Norris, James .......................................................................................219<br />
Norvell, Fabienne ....................................................................206, 633<br />
Noske, David P. ...................................................................................321<br />
Noskov, Alexander ............................................................................465<br />
Novic<strong>of</strong>f, Sarah .........................................................................497, 571<br />
Nowrouzi, Ali ............................................................................204, 274<br />
Noyes, Marcus B.................................................................................485<br />
Nunan, Robert ....................................................................................273<br />
Nurro, Jussi .............................................................................................97<br />
Nusco, Edoardo ..................................................................................353<br />
Nygaard, Sean C. ................................................................................316<br />
Oakes, Benjamin L. ...........................................................................485<br />
Obata, Yuichi .......................................................................................643<br />
Obeng-Adjei, Nyamekye ....................................................................45<br />
O’Callaghan, Christopher ...............................................................435<br />
Ochala, Julien ......................................................................................173<br />
O’Connell, Ryan M. ............................................................................712<br />
O’Connor, Colleen ..............................................................................650<br />
O’Connor, Colleen M. ........................................................................653<br />
O’Connor, Geraldine .........................................................................308<br />
Oda, Yusuke .........................................................................................145<br />
Odom, Guy L. .......................................................................................543<br />
O’Donnell, Patricia ............................................................................360<br />
O’Donnell, Patricia A. ............................................................... 87, 289<br />
Ogino, Atsushi .....................................................................................216<br />
Ogris, Manfred ....................................................................................134<br />
Oh, Eonju...............................................................................................129<br />
Oh, Il Ung ..............................................................................................375<br />
Oh, Jonathan ........................................................................................503<br />
Ohashi, Kazuo ............................................................................. 79, 237<br />
Ohashi, Toya ..............................................................................365, 585<br />
Ohashi, Toya ........................................................................................753<br />
Ohlfest, John R. ...................................................................................270<br />
Ohlin, K. Elisabet ...............................................................................631<br />
Ohlstein, Jason ....................................................................................444<br />
Ohmine, Ken ........................................................................................651<br />
Ohmori, Tsukasa ......................................................................121, 237<br />
Ohnami, Shumpei ..............................................................................642<br />
Ojeda, Sergio R. .....................................................................................78<br />
Okada, Hironori ............................................................ 120, 176, 608<br />
Okada, Takashi .............................................................. 120, 176, 608<br />
168<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013
Abstract Author Index<br />
Okamoto, Sachiko ..................................................................... 16, 629<br />
Okano, Teruo ............................................................................... 79, 237<br />
Old, Matthew .......................................................................................747<br />
Olivares, Simon ........................10, 198, 502, 621, 649, 650, 653<br />
Oliviera, Anna V. .................................................................................160<br />
Olsen, Curtis ........................................................................................685<br />
Olsen, John C. ......................................................................................535<br />
Olson, Erik R. .......................................................................................102<br />
Olson, Susan ........................................................................................466<br />
Olzzewska, Malgorzata ......................................................................50<br />
O’Malley, Bert W. ................................................................................459<br />
O’Malley, Thomas ..............................................................................360<br />
Omata, Daiki ........................................................................................145<br />
O’Neill, Kim ..........................................................................................208<br />
Orchard, Paul .........................................................................................62<br />
Orentas, Rimas J. ................................................................................325<br />
Orl<strong>of</strong>f, Lisa A. .......................................................................................472<br />
Oro, Anthony E. ..................................................................................277<br />
Ortiz, Luis ...................................................................................333, 334<br />
Ortuno, Danny ..........................................................................339, 550<br />
Ortved, Kyla ...............................................................................297, 718<br />
Osborn, Mark J. .............................................................. 270, 584, 738<br />
Ostertag, Derek ..................................................................................676<br />
Ostrowska, Alina .......................................................... 137, 465, 470<br />
Otsu, Makoto .......................................................................................365<br />
Otus, Mokoto .......................................................................................753<br />
Ou, K........................................................................................................708<br />
Ou, Wu ....................................................................................................467<br />
Ousterout, David G. ................................. 167, 177, 241, 292, 491<br />
Owen, John ...........................................................................................253<br />
Owens, Jesse B. ...................................................................................569<br />
Oyster, Nick ..........................................................................................178<br />
Ozaki, Suzuka ......................................................................................192<br />
Ozawa, Keiya .............................................................................121, 651<br />
Özbas-Turan, Suna ............................................................................452<br />
Özkan, Naziye......................................................................................452<br />
Pagliara, Daria ....................................................................................300<br />
Pahujani, Shweta ..................................................................................66<br />
Pai, M. .....................................................................................................682<br />
Pak, Seung Hoon ................................................................................722<br />
Palliser, Debborah .............................................................................205<br />
Palmer, Donna ....................................................................................353<br />
Palomares, Laura A...........................................................................604<br />
Pan, Changchuan ...............................................................................194<br />
Pan, Dao............................................................................... 38, 248, 665<br />
Pan, Xiufang ................................................................................. 98, 166<br />
Pandha, Hardev ................................................................ 18, 395, 652<br />
Pandya, Jhell ........................................................................................333<br />
Pang, Li ..................................................................................................439<br />
Pangallo, Joey ......................................................................................673<br />
Pangallo, Joseph .................................................................................318<br />
Pangle, Amanda K. ............................................................................439<br />
Papapetrou, Eirini ...................................................................679, 680<br />
Papayannopoulou, Thalia ...................................................... 27, 427<br />
Pappen, Beena O. ............................199, 206, 402, 479, 503, 624<br />
Pâques, Frédéric ................................................................................731<br />
Pardi, Norbert .....................................................................................146<br />
Parelkar, Sangram S..........................................................................157<br />
Parise, Carolina B. .............................................................................658<br />
Park, Byung Ho ......................................................................................22<br />
Park, Dong-Kyu ..................................................................................598<br />
Park, Hana ..................................................................................497, 571<br />
Park, Jae .................................................................................................506<br />
Park, Jeonghyun .................................................................................151<br />
Park, Ki Dae .........................................................................................375<br />
Park, Sang Min ....................................................................................380<br />
Park, Sung Young ...............................................................................572<br />
Park, Woo Jin .......................................................................................380<br />
Park, Yeo Myeong ..............................................................................565<br />
Park, Yong Serk ..................................................................................159<br />
Parney, Ian ............................................................................................417<br />
Parovichnikova, E. .............................................................................724<br />
Partridge, Emily A. ............................................................................418<br />
Partridge, Terence A. ..........................................................................93<br />
Paruzynski, Anna ..............................................................................204<br />
Parvizi, Paria .......................................................................................755<br />
Pasceri, Peter ............................................................................322, 593<br />
Paschon, David E. ....................................................................107, 488<br />
Passineau, Michael J. ..............................................................141, 149<br />
Pastore, Nunzia ........................................................................353, 499<br />
Pastor-Flores, Ana R. .......................................................................604<br />
Patel, Anant B......................................................................................150<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013<br />
169
Abstract Author Index<br />
Patel, Bella .................................................................................... 24, 411<br />
Patel, Manij ..........................................................................................535<br />
Patt, Richard ...........................................................................................19<br />
Paulard, Anais .....................................................................................674<br />
Paun, Marla ..........................................................................................140<br />
Pavani, Giulia .......................................................................................110<br />
Payan, Emmanual ..............................................................................674<br />
Payne, Kimberly J. .............................................................................468<br />
Peccate, Cecile ....................................................................................225<br />
Pei, Nana ...............................................................................................221<br />
Pellin, Danilo ..........................................................................................63<br />
Pence, Casey ........................................................................................461<br />
Peng, Kah Whye ....................................................................................17<br />
Peng, Kah-Whye ......................................................................... 20, 413<br />
Peng, L. ...................................................................................................682<br />
Peng, Rui-Yun ......................................................................................105<br />
Penn, Marc S. .......................................................................................500<br />
Pennell, Christopher A. ...................................................................270<br />
Perdomini, Morgane ........................................................................278<br />
Pereboeva, Larisa ..............................................................................587<br />
Perez, Giovani B. ................................................................................582<br />
Perez, Omar .........................................................................................703<br />
Perez-Pinera, Pablo ........................................... 167, 241, 292, 491<br />
Perkins, Edward .................................................................................657<br />
Perlingeiro, Rita C. R. .......................................................................168<br />
Perlingeiro, Rita .................................................................................169<br />
Perna, Serena K. .................................................................................300<br />
Perrigue, Patrick M. ..........................................................................656<br />
Persikov, Anton V. ..............................................................................485<br />
Persons, Derek A. ............................................................ 26, 235, 424<br />
Persson, Jonas ...........................................................................201, 679<br />
Pertschuk, Dan ...................................................................................676<br />
Pestina, Tamara I. ..............................................................................424<br />
Peterson, Jonathan ...........................................................................544<br />
Petrey, Aaron .......................................................................................359<br />
Petrov, Mihail ......................................................................................391<br />
Petrov, Mike ............................................................................................92<br />
Petry, Harald ........................................................................................274<br />
Pettigrew, Amy ...................................................................................461<br />
Phalon, Connor .............................................................. 206, 570, 633<br />
Phaltane, Ruhi.....................................................................................422<br />
Phan-Lai, Vy ............................................................................................13<br />
Phelan, Dane........................................................................................335<br />
Phillips, Dawn ........................................................................................57<br />
Phillips, Lucy .......................................................................................453<br />
Picard, Capucine ................................................................................306<br />
Piccolo, Pasquale .......................................................... 353, 362, 553<br />
Pichon, Chantal ..................................................................................342<br />
Piekna, Alina .......................................................................................593<br />
Piepenhagen, Peter A. .....................................................................453<br />
Pierce, Cora .............................................................................................54<br />
Pierciey, John ..........................................................................................37<br />
Pierciey, Jr, Francis H. .........................................................................51<br />
Pierciey, Jr, Francis J. ..............................................................251, 258<br />
Pierre-Destine, Lorraine ................................................................373<br />
Pierson, Tyler M. ................................................................................378<br />
Pigeon, Lucie .......................................................................................342<br />
Pike-Overzet, Karin ................................................................313, 319<br />
Pirollo, Kathleen F. ............................................................................273<br />
Pivirotto, Phillip ............................................................ 288, 594, 599<br />
Plati, Tiziana ........................................................................................309<br />
Plesa, Gabriela ....................................................................................508<br />
Plewa, Cherylene .....................................................................339, 550<br />
Poddar, Minakshi .....................................................................179, 611<br />
Podetz-Pedersen, Kelly M. ...................................................364, 495<br />
Podhajcer, Osvaldo L. .......................................................................635<br />
Podsak<strong>of</strong>f, Gregory M. .....................................................................110<br />
Poirot, Laurent .............................................................. 401, 683, 731<br />
Polach, Kevin J. ...................................................................................461<br />
Poliani, Pietro L. .................................................................................661<br />
Ponde, Datta ........................................................................................289<br />
Ponder, Katherine P. ............................................................................87<br />
Ponnazhagan, Selvarangan ...........................................................548<br />
Ponnusamy, Ravi ...............................................................................497<br />
Poppante, Kimberly ............................................................................33<br />
Poptani, Harish...................................................................................289<br />
Porada, Christopher .........................................................................253<br />
Portales-Casamar, Elodie ...............................................................115<br />
Porteus, Matthew H. ..............................................................324, 489<br />
Porteus, Matthew ..............................................................................471<br />
Posey Jr, Avery D. ...............................................................................623<br />
Potter, Mark ............................................................................................55<br />
170<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013
Abstract Author Index<br />
Potter, Phil ............................................................................................648<br />
Poulard, Karine .....................................................................................90<br />
Powlesland, Alex................................................................................508<br />
Pozsgai, Eric R. ...................................................................................172<br />
Prabhakar, Shilpa ..............................................................................286<br />
Preston, Thomas J. ...............................................................................91<br />
Preuss, Meredith A. ..........................................................................560<br />
Price, Sandra ............................................................................... 30, 428<br />
Prill, Jan-Michael .....................................................................126, 484<br />
Prince, Timothy .................................................................................660<br />
Prockop, Susan E. .................................................................................61<br />
Proto, Jonathan D. ...................................................................179, 611<br />
Provasi, Elena .....................................................................................504<br />
Psatha, Nikoleta ......................................................................... 27, 427<br />
Puccio, Hélène ....................................................................................278<br />
Pugh, John A. T. ...................................................................................158<br />
Pule, Martin .........................................................................................434<br />
Pulicherla, Nagesh ...............................................................................77<br />
Pulido, Jose ................................................................................395, 652<br />
Pumphrey, Nick J. ...................................................................... 59, 298<br />
Pumphrey, Nick ..................................................................................508<br />
Pungente, Michael D. .......................................................................755<br />
Qasim, Waseem ..................................................................................578<br />
Qi, Feng ..................................................................................................404<br />
Qian, Haohua .......................................................................................709<br />
Qiao, Chunping .............................................................. 296, 367, 370<br />
Qin, Hong ..............................................................................................626<br />
Qin, Su ....................................................................................................326<br />
Qiu, Jianping ........................................................................................106<br />
Qiu, Yujie ...............................................................................................404<br />
Qu, Guang .............................................................................................529<br />
Qu, Jinrong ...................................................................................... 50, 61<br />
Quach, Lucy .........................................................................................373<br />
Quinn, Thomas P. ...............................................................................532<br />
Quintanilla, Rene H...........................................................................244<br />
Rabinowitz, Joseph ...................................................... 108, 444, 544<br />
Radu, Antoneta ...................................................................................236<br />
Rae, Dustin T. .......................................................................................525<br />
Raii, Arash ...........................................................................................727<br />
Ragan, Regina .....................................................................................163<br />
Raghavarchari, Nalini .........................................................................65<br />
Ragni, Margaret C. .............................................................................110<br />
Rahimov, Fedik ......................................................................................93<br />
Rait, Antonina .....................................................................................273<br />
Raju, Liji ................................................................................................755<br />
Rakoczy, Elizabeth P. ...........................................................................54<br />
Ramos, Carlos A. ......................................................................299, 684<br />
Ramos, Julian N. .................................................................................543<br />
Ramsay, E. .............................................................................................708<br />
Ramya, V. ...............................................................................................114<br />
Ranaldo, Gabriella .............................................................................469<br />
Randle, A. E. ............................................................................................40<br />
Randle, Ashley N. ...................................................36, 377, 590, 592<br />
Ranghetti, Anna .................................................................................301<br />
Ranzani, Marco .........................................................................233, 666<br />
Rao, Donald ................................................................................459, 659<br />
Rao, Donald D. .. 199, 206, 219, 234, 272, 317, 402, 510, 625<br />
Rao, Nalam M. .....................................................................................156<br />
Rapoport, Aaron P. .......................................................... 59, 298, 508<br />
Rauschhuber, Christina ..................................................................557<br />
Rawlings, David..................................................................................673<br />
Rawlings, David J. ..................................................32, 311, 318, 665<br />
Raya, Angel ...........................................................................................469<br />
Razban, Vahid .....................................................................................368<br />
Rea-Ramsey, Angela .........................................................................461<br />
Reay, Daniel P. ........................................................................................94<br />
Rebar, Edward J. ............................................................... 46, 184, 488<br />
Recchia, Alessandra .........................................................................347<br />
Redlich, Shira ......................................................................................366<br />
Redmond, Eugene .............................................................................465<br />
Reebye, V. ..............................................................................................682<br />
Reed, T. D. .................................................................................................15<br />
Rees, Donald J. ....................................................................................616<br />
Reid, Michael .......................................................................................656<br />
Reik, Andreas ......................................................................................504<br />
Reinhart, Bonnie......................................................................416, 566<br />
Reinhart, Bonnie L. ...........................................................................478<br />
Reiser, Jakob ........................................................................................467<br />
Rejiba, Soukaina ................................................................................655<br />
Remenyik, Eva ....................................................................................147<br />
Reuser, Arnold J. J. .............................................................................275<br />
Reutenauer, Laurence .....................................................................278<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013<br />
171
Abstract Author Index<br />
Rex, Tonia S. .........................................................................................226<br />
Reyon, Deepak ....................................................................................490<br />
Rezek, Valerie.........................................................................................48<br />
Rhee, Byungdoo .................................................................................151<br />
Rhodes, Eric ...............................................................................245, 551<br />
Riba, Michela .......................................................................................233<br />
Ribas, Antoni .......................................................................................302<br />
Ribeiro, Aline H. .................................................................................630<br />
Riberdy, Janice M. ..............................................................................235<br />
Rice, Jennifer .......................................................................................461<br />
Rice, Kevin G. ............................................................................... 41, 155<br />
Richard, Champlin ............................................................................431<br />
Richard, Isabelle ................................................. 294, 606, 610, 615<br />
Richie, Christopher T. ......................................................................382<br />
Richter, Miximilian ...................................................... 303, 483, 552<br />
Riddell, K. P. ............................................................................................40<br />
Riddell, Stan ...........................................................................................81<br />
Riddell, Stanley R. .............................................................................744<br />
Rieck, Alex ............................................................................................245<br />
Riggs, Arthur .......................................................................................474<br />
Riley, James L. .....................................................................................508<br />
Rinaldi, Fabrizio .......................................................................168, 169<br />
Riolobos, Laura .....................................................................................81<br />
Rissanen, Tuomas T. ............................................................................97<br />
Rival-Gervier, Sylvie .........................................................................322<br />
Rivers, Angela ...........................................................................333, 334<br />
Rivers, Angela E. ................................................................................542<br />
Rivière, Christel ............................................................................ 52, 90<br />
Rivière, Julie ........................................................................................662<br />
Riviere, Christel ....................................................................................33<br />
Riviere, Isabelle...............................................50, 61, 427, 506, 651<br />
Robbins, Joan M. ................................................................................676<br />
Robbins, Joan ......................................................................................703<br />
Robbins, Paul D. ....................................................................................94<br />
Roberts, Alison ...................................................................................678<br />
Roberts, Errin .....................................................................................453<br />
Roberts, Sean ......................................................................................681<br />
Roberts, Thomas C. ...........................................................................181<br />
Robison, Richard ...............................................................................208<br />
Rocca, Celine J. ............................................................... 493, 494, 546<br />
Rocca, Céline .......................................................................................678<br />
Roche, Francis P. ................................................................................631<br />
Rockey, William M. ............................................................................509<br />
Rockne, Russell C. ................................................................................25<br />
Rodino-Klapac, Louise R. ............................................. 91, 172, 291<br />
Rödl, Wolfgang ...................................................................................134<br />
Rodriguez, Stephen ..........................................................................263<br />
Roh, Jae-Kyu ................................................................... 143, 598, 602<br />
Romain, Gabrielle ..............................................................................393<br />
Romano, Josephine ..............................................................................46<br />
Romero, Zulema ....................................................................................26<br />
Rommelfanger-Konkol, Diana ...........................................505, 652<br />
Roncarolo, Maria Grazia .......................................................323, 441<br />
Rondon, Gabriela ..................................................................................10<br />
Rooney, Cliona M. ..............................14, 299, 396, 398, 400, 622<br />
Roovers, Onno ....................................................................................275<br />
Rosales, Cecilia .........................................................................363, 580<br />
Rose, Laura ................................................................................161, 162<br />
Rose-Hellkant, Teresa .....................................................................657<br />
Rosenberg, Jonathan B. .........................................................373, 589<br />
Rosenberg, Steven A. .......................................................................716<br />
Rosengurt, Enrique ..........................................................................659<br />
Ross, Joel ...............................................................................................593<br />
Rossi, John J. ............................................... 454, 456, 515, 668, 682<br />
Rossini, Silvano ..................................................................................420<br />
Roth, David B.......................................................................................324<br />
Roth, Justin C. ......................................................................................438<br />
Rothe, Michael .................................................................. 37, 422, 425<br />
Rottier, Peter .......................................................................................707<br />
Roudaut, Carinne ....................................................................294, 615<br />
Rouillon, Jérémy ................................................................................612<br />
Round, June L. .....................................................................................712<br />
Rousseau, Joel .....................................................................................293<br />
Rousseau, Joël ............................................................................. 83, 389<br />
Rowland, Elizabeth F. ......................................................................485<br />
Roy, Andre ............................................................................................495<br />
Roy, Aparna ................................................................................474, 476<br />
Roy-Chaudhuri, Biswajoy ..............................................................436<br />
Roy-Chaudhuri, Biswajoy ..............................................................701<br />
Rozengurt, Nora .................................................................................497<br />
Ruan, Zhechao ....................................................................................607<br />
Rubin, David G. ...................................................................................589<br />
172<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013
Abstract Author Index<br />
Ruggiero, Eliana ......................................................................... 47, 204<br />
Ruiz, Autumn ......................................................................................407<br />
Rungta, R. ..............................................................................................708<br />
Runtsch, Marah C. .............................................................................712<br />
Ruozi, Giulia ................................................................................. 95, 368<br />
Rushworth, David .............................................................................202<br />
Russ, Jill .................................................................................................524<br />
Russell, David W......................................................................... 81, 266<br />
Russell, Rob .........................................................................................516<br />
Russell, Stephen J. ..........................................17, 20, 406, 407, 413<br />
Russo, Fabio .........................................................................................441<br />
Rusten, Myra C. ..................................................................................102<br />
Ryals, Renee C. .......................................................................................55<br />
Ryan, Robin .............................................................................................14<br />
Ryu, Byoung ................................................................................. 37, 674<br />
Ryu, Byoung Y. .......................................................................................51<br />
Sabapathy, Vikram ............................................................................754<br />
Sabine, Geiger-Schredelseker ......................................................307<br />
Sacher, Anna ........................................................................................123<br />
Sadelain, Michel ................................... 50, 61, 427, 506, 651, 728<br />
Sætrom, P. .............................................................................................682<br />
Sagara, Miyako ............................................................................ 69, 440<br />
Sagawa, Hiroaki .................................................................................532<br />
Sahel, José-Alain ................................................................................225<br />
Sahenk, Zarife .....................................................................................171<br />
Saito, Hiroki ...............................................................................192, 654<br />
Sakamaki, Ippei ..................................................................................626<br />
Sakamoto, Chika ........................................................... 432, 440, 442<br />
Sakata, Asuka ......................................................................................121<br />
Sakata, Yoichi ............................................................................121, 237<br />
Sakuma, Toshie .......................................................................... 68, 726<br />
Sakurai, Fuminori .............................................................................482<br />
Sakurai, Hidetoshi .............................................................................715<br />
Salazar, Andres M. .............................................................................270<br />
Salibi, N. ....................................................................................................40<br />
Salmon, Florence ...............................................................................274<br />
Salomon, Daniel R. ............................................................................493<br />
Salva, Emine ........................................................................................452<br />
Samara-Kuko, Ermira ......................................................................355<br />
Samaranch, Lluis .......................................................... 288, 594, 599<br />
Samoto, Vivian Y. ...............................................................................582<br />
Samulski, R. Jude .......................................6, 54, 74, 112, 268, 546<br />
San Sebastian, Waldy .................................................. 288, 594, 599<br />
Sanadiki, Sara .....................................................................................492<br />
Sanchez, Robbi ...................................................................................272<br />
Sandikin, Daren ..................................................................................430<br />
Sandoval, Lucia ........................................................................152, 620<br />
Sandoval-Zamora, Hugo .......................................................246, 564<br />
Sandoval-Zamora, Hugo E. ............................................................534<br />
Santiago, Yolanda ..............................................................................568<br />
Sanvito, Francesca ....................................................... 233, 323, 666<br />
Saori, Ukai ............................................................................................142<br />
Sapp, Peter ...........................................................................................603<br />
Sardesai, Niranjan Y. ....................................... 9, 45, 153, 341, 397<br />
Sardi, Pablo S..........................................................................................34<br />
Sargent, R. G. .......................................................................................472<br />
Sargent, Roy G. .........................................................................471, 575<br />
Sarkar, Debalina .................................................................................109<br />
Sartin, J. L. ................................................................................................40<br />
Sasakawa, Noriko ..............................................................................715<br />
Sather, Blythe D. ....................................................................................32<br />
Sato, Makoto ........................................................................................215<br />
Sato, Taku .............................................................................................753<br />
Savchenko, V. .......................................................................................724<br />
Savola, Paula ........................................................................................730<br />
Savoldo, Barbara .....................49, 197, 299, 300, 305, 392, 394<br />
Sawaguchi, Yoshikazu .....................................................................145<br />
Saydaminova, Kamola ................................................ 679, 680, 704<br />
Sayles, Leanne C. ................................................................................436<br />
Scala, Serena ...........................................................................................28<br />
Scaramuzza, Samantha ......................................................................63<br />
Scaria, Puthupparampil ..................................................................455<br />
Schaffer, David V. .................................................3, 5, 267, 327, 692<br />
Schambach, Axel .......................................................66, 84, 313, 425<br />
Scharenberg, Andrew M. .............188, 311, 318, 401, 673, 683<br />
Schech, Amanda .................................................................................636<br />
Scheel, Maria .......................................................................................283<br />
Scheff, Nicole .......................................................................................285<br />
Scherer, Lisa.........................................................................................668<br />
Schiedlmeier, Bernd ...............................................................320, 429<br />
Schiffer-Mannioui, Cécile ...............................................................401<br />
Schinco, Piercarla ..............................................................................469<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013<br />
173
Abstract Author Index<br />
Schiroli, Giulia .....................................................................................486<br />
Schlichting, Lisa .................................................................................497<br />
Schmidt, Manfred ...........................37, 47, 62, 63, 204, 210, 233,<br />
......................................................274, 279, 309, 518, 666, 678, 738<br />
Schmidt, Manuel ................................................................................686<br />
Schmidt, Sabine .................................................................................274<br />
Schneider, Lynda ...............................................................................721<br />
Schneppendahl, Johannes .............................................................178<br />
Schoield, Christine ..........................................................................551<br />
Scholler, John .......................................................................................623<br />
Schr<strong>of</strong>f, Matthias ................................................................................686<br />
Schubert, Kathryn .............................................................................466<br />
Schuermann, Nina ............................................................................516<br />
Schug, Jonathan .................................................................................581<br />
Schuldt, Ryan A. .................................................................................745<br />
Schwäble, Joachim ...............................................................................66<br />
Schwerk, Nicolaus ................................................................................84<br />
Scripps, Vicky .........................................................................................56<br />
Seabra, Miguel C. ...............................................................................227<br />
Sekaly, Raick-Pierre ...........................................................................58<br />
Selby, Peter ......................................................................... 18, 395, 652<br />
Sellers, Stephanie E. ............................................................................65<br />
Selot, Ruchita ......................................................................................549<br />
Semechkin, Ruslan ....................................................... 137, 465, 470<br />
Semenova, Ekaterina .......................................................................578<br />
Sen, Dwaipayan ......................................... 114, 331, 337, 536, 537<br />
Senac, Julien S. ....................................................................................280<br />
Sena-Esteves, Miguel ...................... 1, 36, 40, 93, 212, 286, 377,<br />
.......................................................384,387, 388, 590, 592, 595, 721<br />
Senzer, Neil ............................199, 206, 219, 234, 272, 273, 317,<br />
............................................459, 479, 503, 510, 570, 624, 625, 659<br />
Seo, Emiko ............................................................................................643<br />
Seog, Joonil ..............................................................................................44<br />
Sergi Sergi, Lucia ...................................................64, 486, 666, 667<br />
Sergio, Bossi ........................................................................................520<br />
Serrano-Pozo, Alberto .....................................................................540<br />
Servais, Laurent .................................................................................734<br />
Sessa, Maria .........................................................................................309<br />
Seth, Prem ............................................................................................555<br />
Seto, Jane T. ..........................................................................................543<br />
Sevin, Caroline ....................................................................................589<br />
Sganga, Leonardo ..............................................................................635<br />
Sgouramali, Eleni ..............................................................................427<br />
Shaban, Mona......................................................................................451<br />
Shah, Riddhi ........................................................................................345<br />
Shah, Sapana N. ..................................................................................451<br />
Shanley, Ryan ......................................................................................414<br />
Shapiro, Michael ...................................................................................44<br />
Sharma, Anurag .......................................................................104, 628<br />
Sharma, Jay ................................................................................493, 494<br />
Sharma, Nitasha .................................................................................288<br />
Sharma, Priyanka ..............................................................................267<br />
Sharma, Rajiv ......................................................................................488<br />
Sharon, David ......................................................................................403<br />
Shaw, Kit L. ...........................................................................................307<br />
Shayakhmetov, Dmitry M. ..............................................................477<br />
Shedlock, Devon J. ................................................................................45<br />
Shelke, Rajani ......................................................................................330<br />
Shen, Kang ............................................................................................255<br />
Shen, Shen ....................................................................................... 73, 77<br />
Shi, Gang................................................................................................361<br />
Shi, Hailiang .........................................................................................561<br />
Shi, Yijun ...............................................................................................378<br />
Shi, Yingai .............................................................................................473<br />
Shields, Jordan E. ...............................................................................663<br />
Shigemura, Katsumi .........................................................................192<br />
Shihabuddin, Lamya S. ............................................................ 34, 282<br />
Shiku, Hiroshi ............................................................................. 16, 629<br />
Shim, Inkyong .....................................................................................237<br />
Shima, Midori ......................................................................................357<br />
Shimada, Hideaki ..............................................................................216<br />
Shimada, Takashi .......................................................... 228, 358, 601<br />
Shimada, Yohta .........................................................................365, 753<br />
Shimada, Yota......................................................................................585<br />
Shimizu, Hiroyuki ..................................................................... 69, 440<br />
Shimizu, Kahori ..................................................................................482<br />
Shimizu, Saki .......................................................................................530<br />
Shin, Bae-Hyun ...................................................................................380<br />
Shin, Jeong-A. ......................................................................................238<br />
Shin, Jin-Hong ................................................................... 98, 166, 613<br />
Shin, Simon C. ...........................................................................665, 670<br />
Shin, Sug Kyun ....................................................................................685<br />
Shindler, K. ..............................................................................................53<br />
174<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013
Abstract Author Index<br />
Shirakawa, Toshiro .................................................................192, 654<br />
Shires, G. Thomas ..............................................................................402<br />
Shirley, Shawna .....................................................................................99<br />
Shirosaki, Chihiro ..............................................................................192<br />
Shiver, Rachel .........................................................................................45<br />
Shoemark, Amelia .............................................................................435<br />
Shontz, Kim .............................................................................................91<br />
Shontz, Kimberly M. .........................................................................291<br />
Shpall, Elizabeth J. ................................................................................10<br />
Shpall, Elizabeth ................................................................................650<br />
Shreiber, Claire A. ..............................................................................726<br />
Shum, Kato ...........................................................................................515<br />
Siametis, Athanasios ........................................................................427<br />
Sidman, Richard L. ...............................................................................34<br />
Silva, Anne ............................................................................................744<br />
Silva, Gabriela .....................................................................................160<br />
Silva, Michael E. ..................................................................................656<br />
Silva, Nilsa ............................................................................................387<br />
Silverman, Lara I. ...............................................................................619<br />
Silvin, Christopher ............................................................................307<br />
Simoneau, Mélanie ...........................................................................135<br />
Simpson, Elizabeth M. .....................................................................115<br />
Siner, Joshua I. ....................................................................................498<br />
Singh, Aruna ........................................................................................576<br />
Singh, Harjeet .....................................10, 393, 502, 621, 649, 650<br />
Singh, Mandeep S. .............................................................................229<br />
Singh, Martin .......................................................................................639<br />
Singh, Mona .........................................................................................485<br />
Singh, Purba ........................................................................................200<br />
Singh, Swati .........................................................................................673<br />
Singha, Swati ..........................................................................................32<br />
Skinner, Nicole E. B. ..........................................................................270<br />
Skog, Johan .................................................................................304, 751<br />
Skorik, Christian ......................................................................260, 262<br />
Skottman, H .........................................................................................732<br />
Slavcev, Roderick .....................................................................688, 713<br />
Slepushkin, Vladimir .......................................................................495<br />
S-Manickam, Devika .........................................................................691<br />
Smethurst, Dominic P. ............................................................. 59, 298<br />
Smirnova, Olga ...................................................................................581<br />
Smith, Andrew .......................................................................................88<br />
Smith, Barbara K. ...................................................................... 57, 617<br />
Smith, Barbara .......................................................................................90<br />
Smith, Bruce F. ....................................................................................613<br />
Smith, Catherine ................................................................................284<br />
Smith, Claire M. ..................................................................................435<br />
Smith, Julianne ...................................................................................401<br />
Smith, Julianne ...................................................................................683<br />
Smith, Krissy .......................................................................................660<br />
Smith, Trevor R. F. .............................................................................153<br />
Smogorzewska, Monika..................................................................307<br />
Snyder, Evan ........................................................................................465<br />
Snyder, Richard O. ........................................................................ 60, 76<br />
Soderini, Alejandro ...........................................................................635<br />
Soheili, Tayebeh .................................................................................662<br />
Sohn, Jihee ............................................................................................250<br />
Soker, Shay ...........................................................................................253<br />
Soker, Thomas .......................................................................................33<br />
Sokolic, Robert ...................................................................................307<br />
Soland, Melisa .....................................................................................253<br />
Sommerville, Laura ............................................................................... 2<br />
Sondhi, Dolan .................................................................... 11, 373, 589<br />
Song, Lina .............................................................................................603<br />
Song, Lina .............................................................................................735<br />
Song, Liujiang............................................................................328, 542<br />
Song, Shuxian ............................................................................101, 140<br />
Song, Sihong ........................................................................................545<br />
Song, Xiao-Tong ..................................................................................410<br />
Soo, Shin Back .....................................................................................699<br />
Sorrentino, Brian P. ..................................................... 308, 527, 671<br />
Sortwell, C. E. ......................................................................................381<br />
Sortwell, Caryl E. .....................................................................386, 596<br />
Sosale, Nisha G. ..................................................................................523<br />
South, Andy P. .....................................................................................578<br />
Sowers, Anastasia .............................................................................743<br />
Sparks, Jeff............................................................................................461<br />
Spencer, H. Trent ............................................................. 31, 528, 663<br />
Spencer, Jacqueline F. ......................................................................480<br />
Spillman, Michael ..............................................................................336<br />
Spires-Jones, Tara .............................................................................283<br />
Spitzweg, Christine ...........................................................................134<br />
Springer, Matthew L. ........................................................................369<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013<br />
175
Abstract Author Index<br />
Squadrito, Mario L. ...........................................................................301<br />
Sridharan, Arati M. ...........................................................................100<br />
Srinivasan, N..............................................................................114, 536<br />
Srivastava, Alok ......................................... 114, 331, 337, 536, 537<br />
Srivastava, Arun ....................................................75, 118, 314, 328,<br />
......................................................331, 333, 334, 541, 542, 545, 613<br />
Staal, Frank J. T. ........................................................................313, 319<br />
Stadtmauer, Edward A. ................................................. 59, 298, 508<br />
Stagg, Scott ...........................................................................................336<br />
Stamatoyannopoulos, George .............................................. 27, 427<br />
Stanek, Lisa M. ....................................................................................282<br />
Stanic, Bojana .....................................................................................511<br />
Starker, Colby G. .......................................................................584, 738<br />
Starnes, Joseph ...................................................................................664<br />
Steckler, Robert ........................................................................199, 624<br />
Stedman, Hansell ....................................................................... 92, 391<br />
Steet, Richard ......................................................................................359<br />
Stefankski, Jolanta ............................................................................506<br />
Stefano, Corna.....................................................................................520<br />
Stein, Libby ..........................................................................................492<br />
Stein, Sarah ................................................................................152, 620<br />
Steines, Benjamin R..........................................................................327<br />
Stewart, Phoebe L. ............................................................................477<br />
Stilhano, Roberta S. ..................................................... 265, 582, 658<br />
Stock, Stuart ........................................................................................555<br />
Stockard, Cecil R. ...............................................................................560<br />
Stoddard, Barry ........................................................................188, 318<br />
Stoddard, Barry L. .............................................................................193<br />
Stoica, Lorelei I. ..................................................................................388<br />
Stojdl, David F. ....................................................................................413<br />
Stok, Merel ...........................................................................................275<br />
Stoltzman, Carrie ...............................................................................450<br />
Stolz, Donna B. .......................................................................................94<br />
Stone, Daniel .................................................................. 187, 188, 193<br />
Storb, Rainer .......................................................................................443<br />
Stout, David .........................................................................................497<br />
Stoytchev, Ilko .....................................................................................569<br />
Strange, Wanda ..................................................................................351<br />
Strauss, Bryan E. ......................................................................519, 630<br />
Streeter, Jennifer ................................................................................511<br />
Streetz, Konrad...................................................................................514<br />
Stringer, C. Allen ................................................................................503<br />
Stripecke, Renata .................................................................................47<br />
Stupka, Elia ................................................................................233, 517<br />
Su, Leonard .............................................................................................92<br />
Su, Qin ........................................................................................4, 93, 512<br />
Su, Quin .................................................................................................384<br />
Su, Rui-Jun ............................................................................................468<br />
Su, Xiaomin ..........................................................................................457<br />
Subrizi, A ...............................................................................................732<br />
Sudha, G. ................................................................................................536<br />
Sudha, Govindarajan ........................................................................114<br />
Suel, Laurence ....................................................................................294<br />
Suel-Petat, Laurence ........................................................................615<br />
Suerbaum, Sebastian .......................................................................425<br />
Suerth, Julia D. ............................................................................ 66, 425<br />
Sugimoto, Mitsuhiko ........................................................................357<br />
Sui, Hongshu .......................................................................................535<br />
Suksanpaisan, Lukkana .....................................................................20<br />
Sullivan, Lisa ........................................................................................224<br />
Sumaroka, Marina V. ........................................................................374<br />
Sumaroka, Marina .............................................................................547<br />
Summers, Alex ....................................................................................119<br />
Sun, Baodong ......................................................................................276<br />
Sun, Helena C. .....................................................................................107<br />
Sun, Jiali ....................................................................................................14<br />
Sun, Ryan R. .........................................................................................140<br />
Sun, Ryan ..............................................................................................101<br />
Sun, Xiao-Xin ..........................................................................................72<br />
Sun, Xingshen .....................................................................................535<br />
Sun, Yu ...................................................................................................550<br />
Sung, Biin ..............................................................................................104<br />
Suppa, Erica .........................................................................................298<br />
Surace, Enrico Maria ........................................................................539<br />
Sutton, Debbie ....................................................................................508<br />
Su-Youne, Han .....................................................................................699<br />
Suzuki, Daichi .....................................................................................142<br />
Suzuki, Ryo .................................................................................142, 145<br />
Suzuki, Shingo ............................................................... 471, 472, 575<br />
Svinartchouk, Fedor ...............................................................445, 612<br />
Swanekamp, Ria L. ...............................................................................43<br />
Swanson, Kristin R. ..............................................................................25<br />
176<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013
Abstract Author Index<br />
Swanson, Magdalena I. ....................................................................115<br />
Swaroop, Anand .................................................................................709<br />
Sweeney, Colin L. ..................................................................................82<br />
Sweet-Cordero, E. A. .........................................................................436<br />
Swiderski, P. .........................................................................................682<br />
Switzer, Kirsten C. .............................................................................502<br />
Switzer, Kirsten ............................................................. 198, 621, 649<br />
Symonds, Ge<strong>of</strong>f P. .................................................................................48<br />
Synold, Timothy W. ...........................................................................648<br />
Sysol, Justin R. .....................................................................................280<br />
‘t Hoen, Peter A. .................................................................................609<br />
Tabar, Vivianne S. .................................................................................11<br />
Tabor, Aaron T. ....................................................................................616<br />
Tac, Kim Jin ..........................................................................................699<br />
Tachibana, Masashi ..........................................................................482<br />
Tada, Yuji ...............................................................................................216<br />
Taga, Yavuz ...........................................................................................697<br />
Tagalakis, Aristides D. ...........................................................158, 434<br />
Tagawa, Masatoshi ....................................................... 216, 638, 642<br />
Taherian, Mehran ..............................................................................286<br />
Tai, Alex .................................................................................................110<br />
Tai, Chun-Hwei ......................................................................................60<br />
Tai, Ming-Hong ...................................................................................230<br />
Tai, Ming-Hong ...................................................................................740<br />
Takafuji, Makoto ................................................................................150<br />
Takagi, Kazuko ...................................................................................627<br />
Takagi, Norio .......................................................................................142<br />
Takemasa, Ichiro................................................................................749<br />
Takagi-Kimura, Misato ..........................................................218, 638<br />
Takahashi, Hiroshi ............................................................................228<br />
Takahashi, Masamichi ............................................................. 70, 522<br />
Takapoo, Maysam ..............................................................................511<br />
Takayama, Koichi...................................................69, 432, 440, 442<br />
Takayama, Koji ...................................................................................614<br />
Takeda, Shin’ichi ........................................................... 120, 176, 608<br />
Takeda, Shuko .....................................................................................283<br />
Takesako, Kazuto ...............................................................................629<br />
Takesako, Kazutoh ............................................................................651<br />
Takeuchi, Ryo ......................................................................................193<br />
Takiguchi, Yuichi ................................................................................216<br />
Talbot, Kendra T. ...................................................................................45<br />
Talmon, Maria .....................................................................................469<br />
Tam, Hok-Hei.......................................................................................412<br />
Tam, Quincy .........................................................................................522<br />
Tam, Y. ....................................................................................................708<br />
Tamai, Katsuto .......................................................................................80<br />
Tamamoto, Atsuko ............................................................................638<br />
Tan, Jian .................................................................................................178<br />
Tan, Lay Geok ......................................................................................430<br />
Tan, Mengqun .....................................................................................328<br />
Tan, Menqun ........................................................................................542<br />
Tandeski, Terry ..................................................................................619<br />
Tandon, Manish ..................................................................................628<br />
Tang, Qiushi .........................................................................................211<br />
Tang, Qiushi .........................................................................................735<br />
Tang, Ru-Hang ....................................................................................367<br />
Tang, Terence ......................................................................................214<br />
Tang, Winson .........................................................................................58<br />
Tang, Ying ..........................................178, 179, 250, 281, 290, 611<br />
Tani, Kenzaburo ..............................................69, 86, 432, 440, 442<br />
Tannous, Bakhos A. ................................. 103, 321, 458, 540, 751<br />
Tapscott, Stephen H. ........................................................................443<br />
Taquet, Nicolas .............................................................. 352, 464, 633<br />
Tarantal, Alice F. .................................................................................542<br />
Targovnik, Alexandra M. ................................................................632<br />
Tatsumi, Kohei ............................................................................ 79, 237<br />
Tatsumi, Koichiro ..............................................................................216<br />
Taylor, Catherine................................................................................214<br />
Taylor, Clare ............................................................................................61<br />
Taylor, J. .................................................................................................708<br />
Taylor, Walton ...........................................................................199, 624<br />
Tayton-Martin, Helen K. .................................................................298<br />
Teckman, Jeffery ................................................................................211<br />
Templeton, Nancy S. .................................................... 464, 570, 625<br />
Tengood, Jillian E. ..............................................................................144<br />
Tenhu, H ................................................................................................732<br />
Tenney, Rebecca .................................................................................444<br />
Terada, Naohiro .................................................................................545<br />
Terjung, Ronald L. .............................................................................166<br />
Tesfay, Mulu Z. ....................................................................................413<br />
Teunissen, Erik ...................................................................................707<br />
Thakore, Pratiksha I. ............................... 167, 170, 177, 292, 491<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013<br />
177
Abstract Author Index<br />
Thakur, Archana, ...............................................................................711<br />
Tharion, George .................................................................................754<br />
Tharmalingam, Sujeenthar ...........................................................390<br />
Themeli, Maria ...................................................................................728<br />
Theobald, Narda ................................................................................308<br />
Thiel, William H. ................................................................................511<br />
Thokala, Radhika .......................................................... 431, 502, 649<br />
Thomas, Amelia H. ...............................................................................51<br />
Thomas, Carole ..................................................................................185<br />
Thompson, David H. .........................................................................745<br />
Thompson, Jill .........................................................18, 395, 505, 652<br />
Thompson, John .................................................................................214<br />
Thony, Beat ..........................................................................................354<br />
Thorgeirsson, Snorri S. ...................................................................217<br />
Thorpe, Robin .....................................................................................526<br />
Thrasher, Adrian J. ................................... 306, 313, 578, 661, 678<br />
Throm, Robert E. ...............................................................................672<br />
Thyme, Summer ................................................................................318<br />
Ti juan, Mi .............................................................................................649<br />
Tian, Gengwen .......................................................................................12<br />
Tian, Jie ..................................................................................................133<br />
Tiberghien, Pierre .............................................................................399<br />
Tisdale, John ................................................................................ 30, 428<br />
Tisdale, John F. .......................................................................................65<br />
Tobin, Lisa A. .......................................................................................513<br />
Todorow, Carlyn A.............................................................................236<br />
Tolar, Jakub ........................................................................ 62, 584, 738<br />
Tolmachova, Tanya ...........................................................................227<br />
Tomalia, D.A. ........................................................................................682<br />
Tomita, Kyoko .....................................................................................482<br />
Tompkins, Josh ...................................................................................474<br />
Tonon, Giovanni .................................................................................233<br />
Torbett, Bruce ........................................................................................32<br />
Torgerson, Troy ..................................................................................673<br />
Torgerson, Troy R. ............................................................................311<br />
Toro, Gabriela .....................................................................................603<br />
Toro, Gabriella ....................................................................................388<br />
Torok-Storb, Beverly ..............................................................419, 423<br />
Torrance, Chris ...................................................................................551<br />
Torres-Vega, Miguel A. .....................................................................604<br />
Torry, Donald S. ..................................................................................200<br />
Toso, Erik ..............................................................................................169<br />
Toth, Karoly .........................................................................................480<br />
Tournaire, Benoit .................................................................................76<br />
Towe, Christopher ............................................................................438<br />
Trabalza, Antonio .................................................................................67<br />
Trabucco, Leonardo .........................................................................516<br />
Tran, Chy-Anh .....................................................................................668<br />
Tran, Dat ...............................................................................................431<br />
Treger, Janet ..............................................................................522, 530<br />
Tremblay, Jacques P. ...................................83, 293, 389, 491, 620<br />
Tricoli, Lucas ..........................................................................................44<br />
Triggs-Raine, Barbara .....................................................................379<br />
Trobridge, Grant ................................................................................673<br />
Trobridge, Grant D. .................................................................311, 525<br />
Troupes, Andrew ..................................................................................77<br />
Trump, Lisa ..........................................................................................426<br />
Truong, Lynn .......................................................................................183<br />
Tsai, Hsin-Yin.......................................................................................545<br />
Tsai, Mu-Rou ........................................................................................723<br />
Tsai, Richard K. ...................................................................................523<br />
Tsai, Shengdar Q. ...............................................................................490<br />
Tschudy-Seney, Benjamin ..............................................................468<br />
Tseng, Sheng-Hong ..............................................................................60<br />
Tsui, Christopher ...............................................................................302<br />
Tsuji, Kohichiro .....................................................................................86<br />
Tsujita, Maristela ...............................................................................519<br />
Tsukahara, Tomonori ............................................................121, 651<br />
Tuan, Rocky .........................................................................................178<br />
Turchiano, Giandomenico .............................................................347<br />
Turner, Lorianne S. ...........................................................................660<br />
Turner, Mallory A. ...................................................................132, 481<br />
Turtle, Cameron J. .............................................................................744<br />
Turtle, Cameron ....................................................................................81<br />
Turunen, Heikki T..............................................................................330<br />
Tzen, Kai-Yuan .......................................................................................60<br />
Uchibori, Ryosuke ...................................................................121, 651<br />
Uchida, Naoya ......................................................................30, 65, 428<br />
Uchino, Junji ........................................................................................558<br />
Udd, Bjarne ..........................................................................................615<br />
Uemura, Mamoru ..............................................................................749<br />
Ueno, Kazuko .........................................................................................86<br />
178<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013
Abstract Author Index<br />
Ugai, Hideyo...............................................................................127, 558<br />
Ugen, Kenneth E. .................................................................................... 9<br />
Uitto, Jouni ..............................................................................................80<br />
Ukovich, Laura ....................................................................................368<br />
Ul-Hasan, Tahira ................................................................................542<br />
Uludag, Hasan ...................................................... 161, 162, 213, 462<br />
Ungerechts, Guy ............................................................ 405, 554, 639<br />
Ur, Sarah ......................................................................................493, 494<br />
Ur, Sarah M. ..........................................................................................546<br />
Urabe, Masashi .........................................................................121, 651<br />
Urbinati, Fabrizia .................................................................................26<br />
Uribe, Misael .......................................................................................604<br />
Urnov, Fyodor D. ................................................................................568<br />
Ürenden, Gökçe ..................................................................................204<br />
Urschitz, Johann.................................................................................569<br />
Urtti, A ....................................................................................................732<br />
Usas, Arvydas ............................................................................178, 290<br />
Ustoa, Maria.........................................................................................534<br />
Utoh, Rie ...................................................................................................79<br />
Vázquez-Carcaño, Lizbeth M. .......................................................604<br />
Vähä-Koskela, Markus .....................................................................501<br />
Valdmanis, Paul N. ............................................................................436<br />
Valles-Ayoub, Yadira ..............................................................152, 620<br />
Van de Graaf, Benjamin ........................................................373, 589<br />
van der Bom, Imramsjah M. J. ......................................................387<br />
van der Horst, Gijsbertus T. J. .......................................................147<br />
van der Ploeg, Ans T. ........................................................................275<br />
van der Velden, Guus .......................................................................661<br />
van der Vos, Kristan E. ....................................................................304<br />
van Dongen, Jacques J. M. ..............................................................313<br />
van Helsdingen, Yvette ....................................................................521<br />
Van Hoppe, Stephanie .....................................................................458<br />
van Kalle, Christ<strong>of</strong> ................................................................................62<br />
van Logtenstein, Richard ...............................................................274<br />
van Ommen, Gert-Jan B. .................................................................609<br />
van Putten, Maaike ...........................................................................609<br />
van Rijn, Sjoerd ..................................................................................321<br />
van Til, Nico P. .....................................................................................275<br />
van Til, Niek P............................................................................521, 661<br />
Van Vliet, Kim .................................................... 4, 71, 333, 334, 541<br />
Van Wittenberghe, Laetitia ...............................................33, 76, 90<br />
Vandenberghe, Luk H. ............................................................. 71, 330<br />
VandenDriessche, Thierry ..........................8, 354, 355, 496, 667<br />
Vandertop, W. Peter ..........................................................................321<br />
Varadarajan, K. S. ...............................................................................150<br />
Varadarajan, Navin ...........................................................................393<br />
Varela-Rohena, Angel ......................................................................508<br />
Varga, Monika .....................................................................................369<br />
Vargas-Jerónimo, Roxana Y. ..........................................................604<br />
Vasireddy, Vidyullatha ..........................................................224, 227<br />
Vatakis, Dimitrios N. ...........................................................................48<br />
Vazin, Tandis ............................................................................................ 3<br />
vemula, Sai vikram V. .......................................................................628<br />
Venditti, Charles P. ..................................................................113, 280<br />
Vera, Juan ..............................................................................................400<br />
Vera, Juan F. ................................................................................398, 622<br />
Veres, Gabor .....................................................37, 51, 251, 258, 674<br />
Verga, Michael ....................................................................................124<br />
Vergara-Silva, A. ....................................................................................15<br />
VerHeul, Ross ......................................................................................745<br />
Verhoeyen, Els ....................................................................................662<br />
Vetrini, Francesco .............................................................................353<br />
Vetter, Alexandra ...............................................................................134<br />
Vickers, Selwyn M. ............................................................................414<br />
Viecelli, Hiu Man ................................................................................354<br />
Vignaud, Alban ................................................33, 90, 240, 605, 606<br />
Vile, Richard ............................................................18, 395, 505, 652<br />
Villa, Anna ............................................................................................661<br />
Villamizar, Olga ..................................................................................235<br />
Villaverde, Marcela S. ......................................................................632<br />
Vincent, Karen ....................................................................................338<br />
Vincent, Phil ........................................................................................264<br />
Virdi, Kulpreet S. ................................................................................134<br />
Visel, Meike ............................................................................................... 5<br />
Visser, Trudi P. ....................................................................................275<br />
Vite, Charles H. ...................................................................................289<br />
Vitek, Libor ..........................................................................................353<br />
Vizcaino-Dorado, Pablo ..................................................................534<br />
Vlahu, Alexander ...............................................................................745<br />
Vogelbaum, Michael A. ....................................................................676<br />
Vogl, Dan T. ...........................................................................................298<br />
Voit, Thomas .............................................................................225, 734<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013<br />
179
Abstract Author Index<br />
Voit, Tomas .................................................................................445, 612<br />
Volpin, Monica ............................................................................ 64, 666<br />
von Kalle, Christ<strong>of</strong> ......................................63, 204, 233, 274, 279,<br />
.............................................309, 405,518, 639, 666, 674, 678, 738<br />
Vossough, A. ............................................................................................53<br />
Voytas, Daniel F. .......................................................................584, 738<br />
Voziyanov, Yuri ...................................................................................345<br />
Voziyanova, Eugenia ........................................................................345<br />
Vrecenak, Jesse ...................................................................................418<br />
Vrecenak, Jesse D. ..............................................................................236<br />
Vuidepot, Annelise ............................................................................508<br />
Vulin, Adeline ......................................................................................180<br />
Wack, Katy ...............................................................................................94<br />
Waddington, Simon ..................................................... 363, 430, 580<br />
Wadsworth, Sam................................................................................338<br />
Wagemaker, Gerard ..................................................... 275, 521, 661<br />
Wagner, C. R. ........................................................................................710<br />
Wagner, Ernst .....................................................................................134<br />
Wagner, John E. ..................................................................................738<br />
Wagner, Kathryn R. ..............................................................................93<br />
Wake, Kenjiro .........................................................................................79<br />
Walia, Jagdeep S. ................................................................................379<br />
Walker, Christopher M. ......................................................................91<br />
Walker, Edith .......................................................................................195<br />
Walker, Timothy.................................................................................284<br />
Wallace, Kirby .....................................................................................461<br />
Wallace, Lindsay ......................................................................171, 295<br />
Wallace, Lindsay M. ..........................................................................182<br />
Wallraven, Gladice ...............199, 203, 206, 351, 402, 577, 625<br />
Walsh, C. ................................................................................................708<br />
Walters, Jewell N. ..............................................................................397<br />
Wambua, Gerald ................................................................................465<br />
Wanatabe, Ayumi ..............................................................................432<br />
Wang, Beibei ...........................................................................................69<br />
Wang, Bing ........................................178, 179, 250, 281, 290, 611<br />
Wang, Chi-Hsien ......................................................................296, 370<br />
Wang, Dan ................................................................................................. 4<br />
Wang, Gensheng ................................................................................106<br />
Wang, Hongjie ...........................................................................483, 552<br />
Wang, Hongmei ..................................................................................308<br />
Wang, Hua ..................................................................................105, 644<br />
Wang, Jianbin ................................................46, 107, 183, 184, 487<br />
Wang, Jinhui ........................................................................................544<br />
Wang, Jiwei ..........................................................................................507<br />
Wang, Juan ...........................................................................................457<br />
Wang, Lan ................................................................................................11<br />
Wang, Lina .......................................................................... 75, 118, 314<br />
Wang, Lisa ............................................................................................400<br />
Wang, Li-Sheng .........................................................................105, 644<br />
Wang, Minghui....................................................................................127<br />
Wang, Ningqing ..................................................................................154<br />
Wang, Pengju.............................................................................507, 561<br />
Wang, Ping ...........................................................................................360<br />
Wang, Pin-Yi.........................................................................................412<br />
Wang, Qingtao ....................................................................................194<br />
Wang, Shelley .........................................................................................58<br />
Wang, Shengyao .................................................................................563<br />
Wang, Sheri ..........................................................................................281<br />
Wang, Weijun ......................................................................................703<br />
Wang, Xi.................................................................................................163<br />
Wang, Xiang .........................................................................................562<br />
Wang, Xia<strong>of</strong>ei ......................................................................................512<br />
Wang, Xiaoyin .....................................................................................369<br />
Wang, Xin ..............................................................................................752<br />
Wang, Xiuyan .......................................................................50, 61, 506<br />
Wang, Xuefeng ....................................................................................665<br />
Wang, Xueqian .......................................................................................32<br />
Wang, Yaohe ..............................................................................507, 561<br />
Wang, Yuan ..............................................................75, 118, 314, 328<br />
Wang, Yuhan ................................................................................ 85, 579<br />
Wang, Yupeng .....................................................................................318<br />
Wang, Zejing ........................................................................................443<br />
Wang, Zhaohui .................................206, 219, 234, 317, 510, 625<br />
Wang, Zhimin ............................................................................141, 149<br />
Ware, Tierra................................................................................. 39, 597<br />
Warischalk, Jayme K. ............................................................................ 6<br />
Wasielewska, Teresa ...........................................................................50<br />
Wasserman, Wyeth W. .....................................................................115<br />
Watanabe, Natsumi ..........................................................................237<br />
Watanabe, Norihiro ................................................................398, 622<br />
Watson, Rachael S. ...............................................................................88<br />
Watts, Ashlee E. .....................................................................................89<br />
180<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013
Abstract Author Index<br />
Watts, Korashon L. ............................................................................249<br />
Wayne, Alan .........................................................................................307<br />
Wayne, Alan S. ....................................................................................325<br />
Weagel, Evita .......................................................................................208<br />
Weaver, Eric .........................................................................................583<br />
Weaver, Matthew S. .............................................................................96<br />
Weber, Gerrit .......................................................................................392<br />
Weber, Nicholas D. ............................................................................193<br />
Weber, Nick ................................................................................187, 188<br />
Webber, Beau R. .................................................................................738<br />
Weeden, Timothy ..............................................................................453<br />
Wei, Dong ..............................................................................................281<br />
Wei, Jie ......................................................................................................12<br />
Wei, Lisa L. ...........................................................................................737<br />
Wein, Nicolas .......................................................................................180<br />
Weinberg, Marc S. .............................................................................189<br />
Weiner, David B. ................................................ 9, 45, 341, 374, 397<br />
Weinstein, John ..................................................................................267<br />
Weismann, Cara M. .................................................................384, 595<br />
Weiss, Brendan...................................................................................298<br />
Weissman, Drew ......................................................................146, 147<br />
Weissman, Irving ..................................................................................29<br />
Weitzel, Richard .................................................................................428<br />
Welch, Peter .........................................................................................706<br />
Wellman, J. ...............................................................................................53<br />
Wendland, Rebecca ..........................................................................104<br />
Wengerter, Brian C. ...........................................................................205<br />
Wentworth, Bruce M. .......................................................................453<br />
Werling, Natalie .................................................................................526<br />
Wernersson, Karin ............................................................................209<br />
Westin, Erik .........................................................................................587<br />
Westmoreland, Susan ......................................................................387<br />
Wetzke, Martin ......................................................................................84<br />
Wherley, Jennifer ..................................................................................26<br />
Whisenant, Thomas .........................................................................493<br />
Whitaker, Leslie R. ............................................................................382<br />
White, Edward A. ...............................................................................158<br />
Whitlock, B. K.........................................................................................40<br />
Wiekmeijer, Anna-Sophia ....................................................313, 319<br />
Wielgosz, Matthew M. .....................................................................672<br />
Wierda, William G. ............................................................................502<br />
Wilber, Andrew ................................................................ 26, 200, 235<br />
Wild, A ....................................................................................................708<br />
Wilkinson, Leslie ...............................................................................461<br />
Willems, Jessica ............................................................................ 8, 496<br />
Wille-Reece, Ulrike ..............................................................................45<br />
Williams, Daniel .................................................................................298<br />
Williams, James ..................................................................................185<br />
Williams, James A. .............................................. 186, 261, 263, 616<br />
Williams, Regina R. ...........................................................................613<br />
Williams, Richard D. .........................................................................374<br />
Wilson, D. U. ............................................................................................40<br />
Wilson, Heather .................................................................................400<br />
Wilson, James ...........................................................................108, 444<br />
Wilson, James M. ............................................................. 71, 128, 360<br />
Wilson, John R. ...................................................................................622<br />
Wilson, Matthew H. ..........................................................................269<br />
Wilson-Robles, Heather M. ...........................................................653<br />
Winslow, Monte .................................................................................436<br />
Wise, Megan ........................................................................................341<br />
Witt, William .......................................................................................281<br />
Witten, J .................................................................................................718<br />
Witting, Burghardt ...........................................................................686<br />
Wohlgenant, S. L. ...............................................................................381<br />
Wojno, Adam P. ...................................................................................231<br />
Wojton, Jeffrey ....................................................................................747<br />
Wold, William S. M. ...........................................................................480<br />
Wolf, Stephan ......................................................................................274<br />
Wolf, Walter .........................................................................................703<br />
Wolfe, John H. ............................................................................116, 289<br />
Wolfe, Thomas G. ...............................................................................550<br />
Wolfe, Tom ...........................................................................................339<br />
Wolstein, Orit .........................................................................................48<br />
Woltjen, Knut ......................................................................................715<br />
Won, Chong-Hyun .............................................................................602<br />
Wong, Jr, Kamehameha K. ..............................................................542<br />
Wong, Suet Ping .......................................................................346, 354<br />
Wong, Sunnie Y. ..................................................................................488<br />
Woo, Lee Hyun ...................................................................................699<br />
Wood, Steve .........................................................................................339<br />
Wood, Travis........................................................................................183<br />
Woodard, Lauren E. ..........................................................................269<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013<br />
181
Abstract Author Index<br />
Woodle, Martin ...................................................................................455<br />
Wooley, Dawn P. .................................................................................267<br />
Worgall, Stefan ...................................................................................104<br />
Wourms, Michael ...............................................................................664<br />
Wright, J. Fraser ........................................ 110, 312, 529, 547, 663<br />
Wu, Bo ....................................................................................................451<br />
Wu, Chuanfeng ......................................................................................29<br />
Wu, Chu-Tse ...............................................................................105, 644<br />
Wu, Colin O. .............................................................................................65<br />
Wu, I.-Huan ..........................................................................................453<br />
Wu, James .............................................................................................659<br />
Wu, Jing .................................................................................................433<br />
Wu, Joseph ...........................................................................................474<br />
Wu, Lily ..................................................................................................215<br />
Wu, Ruey-Meei ......................................................................................60<br />
Wu, Ruilin .............................................................................................476<br />
Wu, Susan ................................................................................................87<br />
Wu, Wendy ...........................................................................................104<br />
Wu, Xiaolin ...........................................................................................308<br />
Wu, Xin ...................................................................................................749<br />
Wu, Zhijian ...........................................................................................709<br />
Würdinger, Thomas ..........................................................................321<br />
Wyszynski, Mike ................................................................................339<br />
Xia, Danny.............................................................................................184<br />
Xia, Xueqing .........................................................................................139<br />
Xiao, Bin ......................................................................................296, 367<br />
Xiao, Feng-jun ...........................................................................105, 644<br />
Xiao, Juan ..............................................................................................312<br />
Xiao, Ru ..................................................................................................330<br />
Xiao, Weidong .....................................................................................681<br />
Xiao, Xiao ..................................................... 167, 296, 367, 370, 449<br />
Xie, Jun .............................................................................................. 4, 512<br />
Xie, Qing ................................................................................................336<br />
Xie, Yi ......................................................................................................727<br />
Xie, Yili ...................................................................................................513<br />
Xie, Yuran ..............................................................................................711<br />
Xing, Elizabeth M. .................................................................................87<br />
Xu, Ding .................................................................................................448<br />
Xu, Lei .....................................................................................................296<br />
Xu, Weidong .........................................................................................555<br />
Xu, Wenqin ...........................................................................................524<br />
Xu, Yang ....................................................................................................49<br />
Xu, Zhili ..................................................................................................133<br />
Xue, Shu-Ya ...........................................................................................644<br />
Yadak, Rana ..........................................................................................521<br />
Yaguchi, Chiemi ..................................................................................228<br />
Yagyu, Shigeki .....................................................................................684<br />
Yakovief, Galina ..................................................................................495<br />
Yamada, Takeshi ................................................................................643<br />
Yamaguchi, Kiyoshi..............................................................................86<br />
Yamaguchi, Naoto .............................................................................216<br />
Yamamoto, Masato .................................................................408, 414<br />
Yamamoto, Masato .................................................................705, 742<br />
Yamamoto, Motoko ...........................................................................358<br />
Yamamoto, Shohei ...............................................................................86<br />
Yamamoto, Takashi ..........................................................................715<br />
Yamamoto, Yuki .................................................................................642<br />
Yamanaka, Shinya .............................................................................715<br />
Yamatmoto, Hir<strong>of</strong>umi ......................................................................749<br />
Yamazaki, Yoshiyuki .........................................................................601<br />
Yan, Jenny J. .............................................................................................46<br />
Yan, Jenny ...................................................................................183, 487<br />
Yan, Jian ............................................................................... 45, 341, 397<br />
Yan, Junjie .............................................................................................164<br />
Yan, Linna .............................................................................................750<br />
Yan, Pearlly...........................................................................................412<br />
Yan, Renhe ............................................................................................533<br />
Yan, Ziying ............................................................................................535<br />
Yancey, Heather ..................................................................................660<br />
Yáñez-Muñoz, Rafael J .....................................................................678<br />
Yang, Hsiao T. ......................................................................................166<br />
Yang, Hyunwoo ..................................................................................602<br />
Yang, Maria .................................................................................. 45, 341<br />
Yang, Qing .............................................................................................290<br />
Yang, Shan ............................................................................................216<br />
Yang, Shu ...............................................................................................374<br />
Yang, Xia<strong>of</strong>ang ....................................................................................512<br />
Yang, Yanqin ...........................................................................................65<br />
Yang, Yong ............................................................................................562<br />
Yang, Yue-feng ..........................................................................105, 644<br />
Yang, Yu-Lin .........................................................................................723<br />
Yannaki, Evangelia .................................................................... 27, 427<br />
182<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013
Abstract Author Index<br />
Yanovich, Saul .....................................................................................298<br />
Yaseen, Nahi Y. ....................................................................................647<br />
Yazicioglu, Mustafa ...........................................................................110<br />
Ye, Jing Jing ...........................................................................................476<br />
Ye, Jingjing ............................................................................................474<br />
Ye, Xun ....................................................................................................680<br />
Yeagy, Brian A. ....................................................................................493<br />
Yeger, Herman.....................................................................................433<br />
Yeghiazarians, Yerem .......................................................................369<br />
Yen, Paul M. ..........................................................................................618<br />
Yeoh, George C. T................................................................................356<br />
Yeung, Jonathan .................................................................................433<br />
Yezzi, Michael H. ................................................................................472<br />
Yin, Lu ......................................................................................................... 5<br />
Yin, Yue ..................................................................................................644<br />
Ying, Tang .............................................................................................614<br />
Ylä-Herttuala, Seppo ...........................................................................97<br />
Yliperttula, M ......................................................................................732<br />
Yockman, James W. ...........................................................................645<br />
Yoder, Mervin C. .......................................................................328, 542<br />
Yokoi, Kentaro ....................................................................................753<br />
Yokoi, Takayuki ..................................................................................753<br />
Yokoyama, Kazunari K. ...................................................................643<br />
Yoo, Hyuk Sang ...................................................................................256<br />
Yoo, James.............................................................................................253<br />
Yoo, Ji Young ........................................................................................747<br />
Yoon, Hye-Jin .......................................................................................602<br />
Yoon, Sea Young .................................................................................289<br />
Yoon, Sung Won .................................................................................143<br />
Yoshihara, Chieko ..............................................................................627<br />
Yoshioka, Akira ......................................................................................79<br />
You, Yezhi ..............................................................................................164<br />
Young, Brittany A. .............................................................................480<br />
Young, Courtney.................................................................................609<br />
Young, Sarah P. ....................................................................................618<br />
Youssouian, H. ......................................................................................15<br />
Yu, Feng .................................................................................................410<br />
Yu, Juehua ...................................................................................272, 659<br />
Yu, Jun-Ge .............................................................................................747<br />
Yu, Yang .......................................................................................206, 633<br />
Yu, Zhiqiang .........................................................................................164<br />
Yuan, Huijan ........................................................................................385<br />
Yuan, Zhenhua ............................................................... 367, 370, 449<br />
Yue, Yongping .............................................................................. 98, 613<br />
Yue, Yougping ......................................................................................166<br />
Yugeta, Naoko .....................................................................................608<br />
Yulyana, Yulyana ................................................................................254<br />
Yumul, Roma .............................................. 201, 303, 483, 552, 704<br />
Yun, Chae-Ok ................129, 130, 556, 634, 640, 645, 679, 689<br />
Yvon, Eric ...................................................................................... 12, 394<br />
Zabner, Joseph ....................................................................................327<br />
Zacchigna, Serena ..................................................................... 95, 368<br />
Zacharoulis, D .....................................................................................682<br />
Zaiss, Anne K. ......................................................................................448<br />
Zamorano-Carrillo, Absalom ........................................................ 604<br />
Zamore, Phillip D. ..............................................................................512<br />
Zanaboni, Laura .................................................................................420<br />
Zanatta, Daniela B. ............................................................................519<br />
Zanetti, Gianluigi .....................................................................309, 517<br />
Zaraspe, Kim .......................................................................................125<br />
Zaraspe, Kimberly ................................................................................39<br />
Zavaljevski, Maja ...............................................................................450<br />
Zeidan, Joumana ...................................................................................58<br />
Zelenaia, Olga ......................................................................................547<br />
Zentella-Dehesa, Alejandro ...........................................................534<br />
Zentilin, Lorena .....................................................................................95<br />
Zerfas, Patricia M. ..............................................................................383<br />
Zervou, Fani ............................................................................................27<br />
Zhang, Chao .........................................................................................562<br />
Zhang, Chunbo..........................................................................242, 750<br />
Zhang, Daoning .....................................................................................44<br />
Zhang, Fang ................................................................................313, 661<br />
Zhang, Feijie ........................................................................................271<br />
Zhang, Feng ..........................................................................................677<br />
Zhang, G. ...................................................................................................53<br />
Zhang, Jane ..............................................................................................48<br />
Zhang, Jianying ...................................................................................747<br />
Zhang, Jingang ..........................................................................533, 563<br />
Zhang, Jingwan ...................................................................................559<br />
Zhang, Jingyu.......................................................................................217<br />
Zhang, Jun .............................................................................................136<br />
Zhang, Keqing ................................................................... 98, 166, 613<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013<br />
183
Abstract Author Index<br />
Zhang, Lei ..................................................................................... 46, 184<br />
Zhang, Ming ............................................................................................49<br />
Zhang, Mingdi ................................................................ 285, 478, 591<br />
Zhang, Na ..............................................................................................563<br />
Zhang, Qun-wei ........................................................................105, 644<br />
Zhang, Ting ..........................................................................................449<br />
Zhang, Tracy Y. .......................................................................................93<br />
Zhang, Wenli ........................................................................................557<br />
Zhang, Xiao-Bing ...............................................................................468<br />
Zhang, Xichen ......................................................................................239<br />
Zhang, Yan ............................................................................................369<br />
Zhang, Yanling ....................................................................................221<br />
Zhang, YiYi ...........................................................................................729<br />
Zhang, Yu ...............................................................................4, 128, 411<br />
Zhang, Yuanyuan ...............................................................................473<br />
Zhang, Zhao .........................................................................................247<br />
Zhang, Zhenwei ..................................................................................555<br />
Zhao, Allan ...........................................................................................281<br />
Zhao, Chunli .........................................................................................174<br />
Zhao, Chunxia ...........................................................................370, 449<br />
Zhao, Hui Fen ......................................................................................424<br />
Zhao, Huiren ..............................................................................339, 550<br />
Zhao, Li ..................................................................................................105<br />
Zhao, Na ......................................................................................207, 404<br />
Zhao, Yangbing ...................................................................................623<br />
Zhao, Yuan ............................................................................................526<br />
Zheng, Changyu ..................................................................................743<br />
Zheng, Dexian .....................................................................................752<br />
Zheng, Qifa ...........................................................................................214<br />
Zheng, X. Long ....................................................................................312<br />
Zheng, Yanyan .....................................................................................436<br />
Zheng, Zhaohui ...................................................................................298<br />
Zhong, Li ............................................................4, 128, 326, 328, 542<br />
Zhou, Betty ...........................................................................................455<br />
Zhou, Dongming ................................................................................562<br />
Zhou, Guisheng .........................................................................272, 659<br />
Zhou, Jiehua .........................................................................................454<br />
Zhou, Michelle ....................................................................................115<br />
Zhou, Qinghui......................................................................................164<br />
Zhou, Shangzhen ...................................... 110, 312, 488, 498, 733<br />
Zhou, Shang-Zhen .............................................................................547<br />
Zhou, Sheng .........................................................................................671<br />
Zhu, Liwei ...................................................................................207, 404<br />
Zimmermann, Wolfgang .................................................................658<br />
Zingone, Adriana ...............................................................................531<br />
Zolotukhin, Irene ...............................................................................190<br />
Zolotukhin, Sergei ...................................................................335, 548<br />
Zoltick, Philip K. .................................................................................523<br />
Zou, Jizhong ............................................................................................82<br />
Zourelias, Lee ............................................................................141, 149<br />
Zwaka, Thomas ..................................................................................169<br />
184<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013