FINAL PROGR AM - American Society of Gene & Cell Therapy
FINAL PROGR AM - American Society of Gene & Cell Therapy
FINAL PROGR AM - American Society of Gene & Cell Therapy
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<strong>AM</strong>ERICAN SOCIETY OF GENE THERAPY • Final Program 49<br />
ABSTRACTS<br />
THURSDAY, JUNE 2<br />
48 3:45 PM<br />
The Long-Term Effects <strong>of</strong> SERCA2a Overexpression in<br />
Large Animal Model <strong>of</strong> Heart Failure<br />
Yoshiaki Kawase, Ryuichi Yoneyama, Kozo Hoshino, Jennifer<br />
McGregor, Thanh-Thao Ton-Nu, Tom G. Neilan, Robert A.<br />
Levine, Judy Hung, Motoya Hayase, Federica del Monte Roger<br />
Hajjar<br />
49 4:00 PM<br />
Evaluation <strong>of</strong> Catheter-Based Deliveries <strong>of</strong> 10um Gelatin<br />
Hydrogel Microsphere, a Novel Non-Viral Vehicle, in a<br />
Porcine Heart<br />
Kozo Hoshino, Ryuichi Yoneyama, Yoshiaki Kawase, Alec M.<br />
De Grand, John V. Frangioni, Yasuhiko Tabata, Takeshi<br />
Kimura, Toru Kita, Roger J. Hajjar, Motoya Hayase<br />
50 4:15 PM<br />
Transcript Pr<strong>of</strong>iling Identifies Novel Role for SGK1 in<br />
Promoting Cardiomyocyte Survival<br />
Takuma Aoyama, Takashi Matsui, Mikhail Novikov, Anthony<br />
Rosenzweig<br />
Oral Abstract Session 245<br />
Neurodegenerative Diseases<br />
Room: 263/267<br />
51 2:00 PM<br />
Adenoviral BDNF/Noggin-Induced Neurogenesis from<br />
Endogenous Neural Stem <strong>Cell</strong>s Delays Motor Impairment<br />
and Extends Survival in a Transgenic Model <strong>of</strong><br />
Huntington’s Disease<br />
Sung-Rae Cho, Eva Chmielnicki, Aris Economides, Steven A.<br />
Goldman<br />
52 2:15 PM<br />
AAV-Delivered RNAi Improves <strong>Cell</strong>ular and Motor<br />
Phenotypes in a Mouse Model for Huntington’s Disease<br />
Scott Q. Harper, Patrick D. Staber, Xiaohua He, Ines H.<br />
Martins, Qinwen Mao, Henry L. Paulson, Robert M. Kotin,<br />
Beverly L. Davidson<br />
53 2:30 PM<br />
Post-Transcriptional Suppression <strong>of</strong> Striatal Mutant<br />
Huntingtin Leads to Mild Phenotypic Improvements in<br />
the R6/1 Mouse<br />
Edgardo Rodriguez, Eileen Denovan-Wright, Kevin Nash,<br />
Alfred S. Lewin, Ronald J. Mandel<br />
54 2:45 PM<br />
Lentiviral-Mediated Silencing <strong>of</strong> SOD1 through RNA<br />
Interference Delays Disease Onset and Progression in a<br />
Mouse Model <strong>of</strong> ALS<br />
Cedric Raoul, Patrick Aebischer<br />
55 3:00 PM<br />
Lentiviral-Mediated Silencing <strong>of</strong> Mutant SOD-1 Using<br />
RNAi Causes Long Term Correction <strong>of</strong> ALS in a<br />
Transgenic Mouse Model<br />
Gareth S. Ralph, Pippa A. Radcliffe, Denise M. Day, Janine M.<br />
Carthy, Susan M. Kingsman, Kyriacos A. Mitrophanous,<br />
Mimoun Azzouz, Nicholas D. Mazarakis<br />
56 3:15 PM<br />
Amelioration <strong>of</strong> Neurodegenerative and Neuropathological<br />
Alterations in a Transgenic Model <strong>of</strong> Alzheimer’s<br />
Disease: Targeting BACE1 with Small Interfering RNAs<br />
Oded Singer, Robert A. Marr, Edward Rockenstein, Leslie<br />
Crews, Fred H. Gage, Inder M. Verma, Eliezer Masliah<br />
57 3:30 PM<br />
<strong>Therapy</strong> for Alzheimer’s Disease by Neprilysin <strong>Gene</strong><br />
Transfer<br />
Robert A. Marr, Edward Rockenstien, Atish Mukherjee, Mark<br />
S. Kindy, Louis B. Hersh, Fred H. Gage, Eliezer Masliah,<br />
Inder M. Verma<br />
58 3:45 PM<br />
Rett Syndrome Is Reversible and Treatable by MeCP2<br />
<strong>Gene</strong> <strong>Therapy</strong> into the Striatum in Mice<br />
Ken-ichiro Kosai, Akira Kusaga, Takeo Isagai, Koji Hirata,<br />
Satoshi Nagano, Yoshiteru Mur<strong>of</strong>ushi, Tomoyuki Takahashi,<br />
Sachio Takashima, Toyojiro Matsuishi<br />
59 4:00 PM<br />
rAAV-Mediated Nigral Parkin Over-Expression Is<br />
Neuroprotective in the 6-OHDA Rat Model <strong>of</strong><br />
Parkinson’s Disease<br />
Fredric P. Manfredsson, Alfred S. Lewin, Nicholas Muzyczka,<br />
Corinna Burger, Ronald J. Mandel<br />
60 4:15 PM<br />
Recovery <strong>of</strong> Vestibular Function Induced by math1 <strong>Gene</strong><br />
Delivery<br />
Hinrich Staecker, Mark Praetorius, Douglas E. Brough<br />
Oral Abstract Session 246<br />
<strong>Gene</strong> <strong>Therapy</strong> for Infections and Vaccines<br />
Room: 230/231<br />
61 2:00 PM<br />
Higher In Vivo Maintenance, Biodistribution and<br />
Immunopotency <strong>of</strong> Dendritic <strong>Cell</strong> Vaccines Self-Differentiated<br />
through Lentiviral Vector Programming<br />
Richard C. Koya, Takahiro Kimura, Antoni Ribas, Nori<br />
Kasahara, Renata Stripecke<br />
62 2:15 PM<br />
Transduction <strong>of</strong> CD34+ Hematopoietic Progenitor <strong>Cell</strong>s<br />
with a SIV-Based Lentiviral Vector Expressing Antisense<br />
SIV Env Protects CD4+ T <strong>Cell</strong> Progeny from<br />
SIVmac239 Infection<br />
S. E. Braun, F. Eng, M. Connole, C. Chen, B. Dropulic, G.<br />
K. Binder, X. Lu, R. P. Johnson
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