Final Program - American Society of Gene & Cell Therapy

Abstract Directory, Friday, May 17, 2013
Friday, May 17, 2013
Poster Session II, continued
5:30 PM - 7:30 PM
Room: Exhibit Hall C/D
Neurologic Gene & Cell Therapy I
379
Intravenous AAV9 Based Gene Therapy Corrects a
Mouse Model of Sandhoff Disease
Jagdeep S. Walia, Alexander Bello, Naderah Altaleb, Christa Kruck,
Richard Hemming, Gary P. Kobinger, Barbara Triggs-Raine.
380
A Speciic Cleavage of Amyloid-b by a Viral Protease
NIa Prevents the Amyloid-b-Mediated Mitochondrial
Dysfunction
Woo Jin Park, Bae-Hyun Shin, Hye-Eun Han, Sang Min Park.
381
Viral Vector Mediated Overexpression of Pleiotrophin
for Disease Modiication in Parkinson’s Disease
S. E. Gombash, F. P. Manfredsson, C. J. Kemp, S. L. Wohlgenant, N. C
Kuhn, S. M. Fleming, R. J. Mandel, T. H. Collier, C. E. Sortwell.
382
Infrared Fluorescent Protein (iRFP) as a Reporter for
Monitoring and Modulating Neurons
Christopher T. Richie, Lowella V. Fortuno, Douglas B. Howard,
Leslie R. Whitaker, Bruce T. Hope, Brandon K. Harvey.
383
Fetal Brain-Directed AAV5 and AAV9 Gene Therapy
Results in Rapid, Robust, and Persistent Transgene
Expression in Mouse Choroid Plexus Epithelia
Marie Reine Haddad, Eun-Young Choi, Anthony Donsante, Patricia
M. Zerfas, Stephen G. Kaler.
384
Intravenous Administration of AAV9-mbgal in a GM1
Gangliosidosis Mouse Model Expresses Enzyme in the
CNS and Delays Disease Onset with Gender Differences
Cara M. Weismann, Jennifer Ferreira, Stacy Maitland, Quin Su,
Guangping Gao, Miguel Sena-Esteves.
385
Gene Delivery of ATP6 by a Mitochondrial Targeting
Sequence Modiication of AAV9 Capsid VP2 Rescues
Cells with Mutated T8993G MtDNA Responsible for
Neuropathy Ataxia and Retinitis Pigmentosa and
Expresses in the Mouse CNS
John Guy, Huijan Yuan.
386
rAAV2/5-Mediated Gene Transfer Is Less Eficient
in the Aged Rat Brain: Examination of a-Synuclein-
Mediated Neurodegeneration in the Nigrostriatal
System
Sara E. Gombash, Fredric P. Manfredsson, Christopher H. Kemp,
David L. Fischer, Nathan C. Kuhn, Allyson Cole-Strauss, Ronald J.
Mandel, Sheila M. Fleminig, Timothy J. Collier, Jack W. Lipton, Caryl
E. Sortwell.
387
Safety Study of Intracranial AAVrh8-Mediated Gene
Transfer of beta-Hexosaminidase in Non-Human
Primates
Diane Golebiowski, Imramsjah M. J. van der Bom, Churl-Su Kwon,
Stacy Maitland, Anna Luisa Kühn, Nina Bishop, Elizabeth Curren,
Nilsa Silva, Andrew Miller, Susan Westmoreland, Matthew Gounis,
Douglas R. Martin, Wael Asaad, Miguel Sena-Esteves.
388
Targeting Human SOD1 Using AAV Mediated RNAi in a
Mouse Model of Amyotrophic Lateral Sclerosis
Lorelei I. Stoica, Gabriella Toro, Robert Brown, Christian Mueller,
Miguel Sena-Esteves.
389
TALEFrat#8–VP64 Increases Frataxin Gene and Protein
Expression in Fibroblasts of Friedreich Patients and of
Mouse Model
Jacques P. Tremblay, Pierre Chapdelaine, Jean-Paul Iyombe, Joël
Rousseau, Zoé Coulombe.
390
Transduction of the Central Nervous System Following
Intra-Cerebroventricular Administration of Adeno-
Associated Viral Vectors to Neonatal and Juvenile Mice
Shervin Gholizadeh, Sujeenthar Tharmalingam, Margarita E.
MacAldaz, David R. Hampson.
391
Serial and Endpoint Biomarkers for Cardiorespiratory
Function in a Canine Model of DMD Provide an
Integrative View of Disease Pathogenesis and Response
to Therapy
Andrew Mead, Alock Malik, Mihail Petrov, Martin Childers, Janet
Bogan, Benjamin Kozyak, Hansell Stedman.
590 (Moved from Poster Session III)
Intracranial AAV Gene Therapy Extends the Life Span
of GM2 Gangliosidosis Cats >Four-Fold with No Clinical
Evidence of Vector Toxicity
Allison M. Bradbury, Victoria J. McCurdy, Aime K. Johnson, Heather
Gray-Edwards, Brandon L. Brunson, Ashley N. Randle, Nancy R.
Cox, Miguel Sena-Esteves, Douglas R. Martin.
116
Final Program SALT LAKE CITY, UTAH May 15–18, 2013