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, 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105 Abstract Withdrawn from Presentation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
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