Abstract Directory, Wednesday, May 15, 2013 Oral Abstract Session 138 Sensory (Ophthalmic and Auditory) <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> 4:20 PM - 5:20 PM Room: 150 ABC 53 4:20 PM Retinal <strong>Gene</strong> <strong>Therapy</strong> May Alter Connectivity <strong>of</strong> Visual Pathways M. Ashtari, L. Cyckowski, G. Zhang, P. Cook, K. Marshall, J. Wellman, J. Gee, A. Vossough, K. Shindler, A. Maguire, J. Bennett. Wednesday, May 15, 2013 54 4:35 PM <strong>Gene</strong> <strong>Therapy</strong> for Wet-AMD: Progress Report on a Phase I/II Clinical Trial Elizabeth P. Rakoczy, May C. Lai, Cora Pierce, Aaron L. Magno, Richard J. Samulski, David J. Dismuke, Joshua C. Grieger, Thomas W. Chalberg, Mark S. Blumenkrnz, Martyn French, Ian J. Constable. 55 4:50 PM Preclinical Safety Studies for AAV2-MerTK <strong>Gene</strong> <strong>Therapy</strong> Vector for Retinitis Pigmentosa Thomas J. Conlon, Wen-Tao Deng, Renee C. Ryals, Kirsten E. Erger, Travis L. Cossette, Shannon E. Boye, Isaam McDoom, Nathalie Clemente, Brian Cleaver, Mark Potter, Corinne Abernathy, Sanford L. Boye, William H. Hauswirth. 56 5:05 PM Glaucoma-GT, a Novel <strong>Gene</strong> <strong>Therapy</strong> Treatment for Primary Open-Angle Glaucoma Katie Binley, Scott Ellis, Vicky Scripps, Sharifah Iqball, Stuart Naylor, Kyriacos Mitrophanous. <strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013 91
Thursday, May 16, 2013 Abstract Directory, Thursday, May 16, 2013 Oral Abstract Session 230 Clinical <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> Oral Abstract Session 2:00 PM - 4:00 PM Room: Ballroom A 57 2:00 PM Acid alpha-Glucosidase <strong>Gene</strong> Replacement <strong>Therapy</strong> to the Diaphragm in Ventilator-Dependent Pompe Disease: One-Year Respiratory Motor Outcomes Barbara K. Smith, Daniel Martin, Lee Ann Lawson, Cathryn S. Mah, Saleem Islam, Thomas Conlon J. Conlon, Dawn Phillips, Shelley Collins, Barry J. Byrne. 58 2:15 PM Long Term CD4 Reconstitution in HIV Subjects Receiving ZFN CCR5 Modiied CD4 T-<strong>Cell</strong>s (SB-728-T) May Be Attributed to the Sustained Durability <strong>of</strong> the Central Memory T-<strong>Cell</strong> Subset Gary K. Lee, Joumana Zeidan, Jay Lalezari, Ronald Mitsuyasu, Shelley Wang, Marty Giedlin, Ge<strong>of</strong>f Nichol, Winson Tang, Dale Ando, Raick-Pierre Sekaly. 59 2:30 PM Cardiovascular Toxicity and Titin Cross-Reactivity <strong>of</strong> Afinity-Enhanced TCR-Engineered T <strong>Cell</strong>s Against HLA-A1 Restricted MAGE-A3 Antigen Marcela V. Maus, Gerald P. Linette, Edward A. Stadtmauer, Aaron P. Rapoport, Bruce L. Levine, Lyndsey A. Emery, Leslie Litzky, Gwendolyn K. Binder-Scholl, Dominic P. Smethurst, Andrew B. Gerry, Nick J. Pumphrey, Alan Bennet, Joanna Brewer, Jane Harper, Namir Hassan, Bent K. Jakobsen, Michael Kalos, Carl H. June. 60 2:45 PM <strong>Gene</strong> <strong>Therapy</strong> for Aromatic L-Amino Acid Decarboxylase Deiciency: Report <strong>of</strong> Treatment for 8 Patients Wuh-Liang Hwu, Shin-ichi Muramatsu, Sheng-Hong Tseng, Kai- Yuan Tzen, Ni-Chung Lee, Yin-Hsiu Chien, Richard O. Snyder, Barry Byrne, Chun-Hwei Tai, Ruey-Meei Wu. 61 3:00 PM Safe G-CSF Mobilization <strong>of</strong> Thalassemia Patient CD34+ <strong>Cell</strong>s and Effective Globin <strong>Gene</strong> Transduction for Therapeutic Infusion Farid Boulad, Xiuyan Wang, Clare Taylor, Jinrong Qu, Leda Ferro, Garie Karponi, Shirley Bartido, Patricia Giardina, Glenn Heller, Susan E. Prockop, Aurelio Maggio, Michel Sadelain, Isabelle Riviere. 62 3:15 PM Hematopoietic Stem <strong>Cell</strong> <strong>Gene</strong> <strong>Therapy</strong> with Lentiviral Vector in 4 Patients with Cerebral X-Linked Adrenoleukodystrophy: Long-Term Outcome and Comparison <strong>of</strong> Eficacy with Allogeneic Hematopoietic Stem <strong>Cell</strong> Transplantation Paul Orchard, Nathalie Cartier, Salima Hacein-Bey-Abina, Joanne Kurtzberg, Maria Escolar, Jakub Tolar, André Baruchel, Jean-Hughes Dalle, Gérard Michel, Stéphane Blanche, Cynthia Bartholomä, Manfred Schmidt, Pierre Bougnères, Christ<strong>of</strong> van Kalle, Marina Cavazzana-Calvo, Alain Fischer, Patrick Aubourg. 676 3:30 PM Update on Toca 511 - a retroviral replicating vector in clinical trials for High Grade Glioma Douglas J Jolly, Joan M. Robbins, Derek Ostertag, Amy Lin, Carlos Ibanez, Harry E. Gruber, Noriyuki Kasahara, Timothy F. Cloughesy, Manish K. Aghi, Susan M. Chang, Michael A. Vogelbaum, Santosh Kesari, Tom Mikkelsen, Joseph Landoli, E. Antonio Chiocca, James Elder, Greg Foltz, Dan Pertschuk 299 3:45 PM Clinical Responses in Patients Infused with T Lymphocytes Redirected To Target -Light Immunoglobulin Chain Carlos A. Ramos, Barbara Savoldo, Enli Liu, Adrian P. Gee, Zhuyong Mei, Bambi Grilley, Cliona M. Rooney, Helen E. Heslop, Malcolm K. Brenner, Gianpietro Dotti. Oral Abstract Session 231 RNA Virus Vectors 2:00 PM - 4:00 PM Room: Ballroom B 63 2:00 PM Comprehensive Study <strong>of</strong> Lentiviral “Integrome” in WAS <strong>Gene</strong> <strong>Therapy</strong> Patients Luca Biasco, Cristina Baricordi, Francesca Dionisio, Andrea Calabria, Samantha Scaramuzza, Danilo Pellin, Clelia Di Serio, Christ<strong>of</strong> Von Kalle, Manfred Schmidt, Eugenio Montini, Luigi Naldini, Alessandro Aiuti. 64 2:15 PM In Vivo Safety Evaluation <strong>of</strong> a Novel Insulated SIN LV Reveals Genotoxicity Escape-Mechanisms Monica Volpin, Daniela Cesana, Alexandre Artus, Caroline Duros, Fabrizio Benedicenti, Lucia Sergi Sergi, Luigi Naldini, Odile Cohen- Haguenauer, Eugenio Montini. 65 2:30 PM No Impact <strong>of</strong> Lentiviral Transduction on Hematopoietic Stem and Progenitor <strong>Cell</strong> Telomere Length or <strong>Gene</strong> Expression Proile in the Rhesus Macaque Stephanie E. Sellers, Bogden Dumitriu, Mary Morgan, Colin O. Wu, Nalini Raghavarchari, Yanqin Yang, Naoya Uchida, John F. Tisdale, Robert E. Donahue, Andre Larochelle, Cynthia E. Dunbar. 92 <strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013
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ASGCT Officers and Board of Directo
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Outstanding New Investigator Awards
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Faculty List Thomas W. Chalberg, Jr
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Faculty List Ann M. Leen, PhD Baylo
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Faculty List Evan Y. Snyder, MD PhD
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General Meeting Information Continu
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General Meeting Information Speaker
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Salt Palace Convention Center Floor
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Salt Palace Convention Center Floor
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Program Schedule, Wednesday, May 15
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Abstract Author Index Bartel, Ronnd
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