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 85<br />
ABSTRACTS<br />
FRIDAY, JUNE 3<br />
651<br />
Oligonucleotide-Mediated <strong>Gene</strong> Repair Restores Full<br />
Length SMN mRNA Expression in Mutant-SMN<br />
Murine Fibroblasts<br />
Emanuela Bruscia, Rafal Ochalski, Michael Rice, Giuseppe<br />
Novelli, Federica Sangiuolo, Diane Krause<br />
652<br />
Adeno-Associated Vector Delivery <strong>of</strong> Gutamate Transporters<br />
in an ALS Mouse Model<br />
Christine Haenggeli, Brian Kaspar, Jeffrey Rothstein<br />
653<br />
Injection <strong>of</strong> AAV Directly into the Spinal Cord Increases<br />
Levels <strong>of</strong> <strong>Gene</strong> Expression<br />
Christine Haenggeli, Jeffrey D. Rothstein, Brian K. Kaspar<br />
654<br />
Quantifying GDNF Levels in the Spinal Cord after<br />
Intracerebral Delivery <strong>of</strong> rAAV5 GDNF<br />
Kevin D. Foust, Paul J. Reier, Ronald J. Mandel, Terence R.<br />
Flotte<br />
Neurologic: Advances in Vectors, Delivery and Imaging<br />
655<br />
Compacted DNA Nanoparticles Effectively Transfect<br />
Brain <strong>Cell</strong>s In Vitro and In Vivo<br />
David M. Yurek, Anita Fletcher-Turner, Mark J. Cooper<br />
656<br />
Development <strong>of</strong> Viral Vectors To Mediate Neuron-<br />
Specific <strong>Gene</strong> Silencing<br />
Colin P. Glover, Alison S. Bienemann, Beverly L. Davidson,<br />
James B. Uney<br />
657<br />
A Nucleic Acid-Based Transgene Reporter for In Vivo<br />
MRI<br />
Eric T. Ahrens, Clinton S. Robison, William F. Goins<br />
658<br />
Development and Evaluation <strong>of</strong> an Infusion Device for<br />
AAV-Mediated <strong>Gene</strong> Delivery to the Brain<br />
Jurg M. Sommer, Laura M. Sanftner, Haihwa Kao, Peter H.<br />
Smith, Brian M. Suzuki, Joseph Vargas, John R. Forsayeth,<br />
Janet Cunningham, Krys S. Bankiewicz, Jan Bernal Ruth<br />
Lessard, Glenn Pierce, Kirk W. Johnson<br />
659<br />
The Plasticity <strong>of</strong> Hematopoietic Stem <strong>Cell</strong>s (HSC): Rat<br />
HSC Transduced In Situ by rSV40 Vectors Differentiate<br />
into Multiple Lineages <strong>of</strong> CNS <strong>Cell</strong>s<br />
Jean-Pierre Louboutin, Bianling Liu, Beverley S. Reyes,<br />
Elizabeth J. Van Bockstaele, David S. Strayer<br />
660<br />
Quantitative Comparison <strong>of</strong> AAV Serotypes AAV2,<br />
AAV5, and AAVrh.10 Efficiency for CNS <strong>Gene</strong> <strong>Therapy</strong><br />
Following Intracranial <strong>Gene</strong> Delivery<br />
Dolan Sondhi, Daniel A. Peterson, Elizabeth Vassallo, Christine<br />
T. Sanders, Jamie A. Stratton, Neil R. Hackett, Guangping<br />
Gao, James M. Wilson, Ronald G. Crystal<br />
661<br />
Efficient SV40-Mediated <strong>Gene</strong> Transfer to the Brain<br />
Jean-Pierre Louboutin, Beverley S. Reyes, Elizabeth J. Van<br />
Bockstaele, David S. Strayer<br />
662<br />
The Tracking <strong>of</strong> Exogenous <strong>Cell</strong>s in the Injured Central<br />
Nervous System: Viral Vector Labeling Versus an Intrinsic<br />
<strong>Gene</strong>tic Marker<br />
Bas Blits, Brandon M. Kitay, Andre R. Sanchez, Christian M.<br />
Andrade, Damien D. Pearse<br />
Neurologic: Sensory<br />
663<br />
Protection Against Aminoglycoside-Induced Ototoxicity<br />
by AAV Vector-Mediated GDNF <strong>Gene</strong> Transfer into the<br />
Cochlea<br />
Yuhe Liu, Takashi Okada, Kuniko Shimazaki, Tatsuya<br />
Nomoto, Kianoush Sheykholeslami, Shin-Ichi Muramatsu,<br />
Hiroaki Mizukami, Akihiro Kume, Shuifang Xiao, Keiichi<br />
Ichimura Keiya Ozawa<br />
664<br />
Regeneration <strong>of</strong> Sensory <strong>Cell</strong>s in the Inner Ear <strong>of</strong><br />
Deafened Mature Guinea Pigs<br />
Donald L. Swiderski, Masahiko Izumikawa, Douglas E.<br />
Brough, Yehoash Raphael<br />
665<br />
Experiences with Delivery <strong>of</strong> Nanoparticles to the Mouse<br />
Cochlea<br />
Mark Praetorius, Kim Baker, Bernhard Schick, Peter K.<br />
Plinkert, Hinrich Staecker<br />
666<br />
LV with Different Promoters Mediated <strong>Gene</strong> Expression<br />
in the Inner Ear<br />
Maoli Duan, Anders Fridberger, Dongguang Wei, Cecilia<br />
Lundberg<br />
667<br />
Lentiviral <strong>Gene</strong> Transfer <strong>of</strong> RPE65 cDNA in Knock-Out<br />
Mouse Models <strong>of</strong> Leber Congenital Amaurosis<br />
Corinne Kostic, Alexis-Pierre Bemelmans, Muriel Jaquet, Dana<br />
Hornfeld, William W. Hauswirth, Janis Lem, Zhongyan Wang,<br />
Daniel F. Schorderet, Francis L. Munier, Andreas Wenzel Yvan<br />
Arsenijevic
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