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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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 />
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