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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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
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