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, Saturday, May 18, 2013<br />
Poster Session III, continued<br />
3:45 PM - 5:45 PM<br />
Room: Exhibit Hall C/D<br />
Musculo-skeletal <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> II<br />
605<br />
MTMR2 Ameliorates the Phenotype <strong>of</strong> Myotubular<br />
Myopathy in Mice<br />
Nathalie Danièle, Thibaud Jamet, Christelle Moal, Romain Joubert,<br />
Nadia Messaddeq, Michael W. Lawlor, Alban Vignaud, Anna<br />
Buj-Bello.<br />
606<br />
Restricting MTM1 Transgene Expression to Skeletal<br />
Muscle in AAV-Mediated <strong>Gene</strong> <strong>Therapy</strong> for Myotubular<br />
Myopathy<br />
Romain Joubert, Christelle Moal, Alban Vignaud, Samia Martin,<br />
Isabelle Richard, Philippe Moullier, Alan H. Beggs, Martin K.<br />
Childers, Anna Buj-Bello.<br />
607<br />
Long Term Intra-Articular Expression <strong>of</strong> PRG4 Prevents<br />
Post-Traumatic Osteoarthritis<br />
Zhechao Ruan, Vincenzo Cerullo, Kilian Guse, Ayelet Erez, Brian<br />
Dawson, Frank Gannon, Brendan Lee.<br />
608<br />
Immune Tolerance Induction in Canine X-Linked<br />
Muscular Dystrophy with Trans-Placental rAAV9-<br />
Microdystrophin Transduction<br />
Hiromi Hayashita-Kinoh, Hironori Okada, Yuko Nitahara-Kasahara,<br />
Tomoko Chiyo, Naoko Yugeta, Takashi Okada, Shin’ichi Takeda.<br />
609<br />
Low Dystrophin Levels Increase Survival and Improve<br />
Muscle Pathology and Function in Dystrophin/Utrophin<br />
Double Knockout Mice<br />
Maaike van Putten, Margriet Hulsker, Courtney Young, Peter A. ‘t<br />
Hoen, Gert-Jan B. van Ommen, Annemieke M. Aartsma-Rus.<br />
610<br />
Limb Girdle Muscular Dystrophy 2I: <strong>Gene</strong>ration <strong>of</strong> a<br />
New Mouse Model and Test <strong>of</strong> Therapeutic Approaches<br />
by rAAV2/9 <strong>Gene</strong> Transfer<br />
Evelyne Gicquel, Karine Charton, Nathalie Daniele, Isabelle<br />
Richard.<br />
611<br />
Hepatocyte Growth Factor Contributes to the Improved<br />
Pathology <strong>of</strong> mdx:p65+/- Mice<br />
Jonathan D. Proto, Ying Tang, Aiping Lu, Minakshi Poddar, Sarah<br />
Beckman, Kayla Imbrogno, Timothy Hannigan, Wendy Mars, Bing<br />
Wang, Johnny Huard.<br />
612<br />
Urine Biomarkers for Duchenne Muscular Dystrophy<br />
and Its Animal Models<br />
Jérémy Rouillon, Thibaut Léger, Jean-Michel Camadro, Tomas Voit,<br />
Fedor Svinartchouk.<br />
613<br />
Systemic <strong>Gene</strong> Transfer in Neonatal Dogs Using<br />
Tyrosine Mutant AAV-1,6 and9<br />
Chady H. Hakim, Yongping Yue, Jin-Hong Shin, Regina R. Williams,<br />
Keqing Zhang, Arun Srivastava, Bruce F. Smith, Dongsheng Duan.<br />
614<br />
Effect <strong>of</strong> Angiogenesis on the Regenerative Capacity <strong>of</strong><br />
ACL-Derived CD34+ <strong>Cell</strong>s in ACL Reconstruction<br />
Koji Takayama, Tang Ying, Wang Bing, Freddie Fu, Johnny Huard.<br />
615<br />
The Phenotype <strong>of</strong> Dysferlin-Deicient Mice Is Not<br />
Rescued by AAV-Mediated Transfer <strong>of</strong> Anoctamin 5<br />
Karine Charton, Laurence Suel-Petat, François Monjaret, Nathalie<br />
Bourg-Alibert, Carinne Roudaut, Evelyne Gicquel, Bjarne Udd,<br />
Isabelle Richard.<br />
616<br />
Transfection <strong>of</strong> Murine Dermis Via Topical DNA<br />
Plasmid Delivery<br />
Chunqing Dou, Guy P. Marti, Lixin Liu, Frank Lay, Donald J. Rees,<br />
Aaron T. Tabor, Junkai Du, Sayed Mohammd Hosseini, Amir Mehdi<br />
Ansari, Ali Karim Ahmed, James A. Williams, John W. Harmon.<br />
617<br />
Clinical Trial Readiness for <strong>Gene</strong> Replacement to the<br />
Diaphragm: Translational Respiratory Endpoints in<br />
Ventilator-Dependent Neuromuscular Diseases<br />
Barbara K. Smith, Melissa A. Goddard, Martin K. Childers, Barry J.<br />
Byrne.<br />
618<br />
Adjunctive b2-Agonist <strong>Therapy</strong> Enhances Traficking <strong>of</strong><br />
Acid a-Glucosidase to Lysosomes in the Skeletal Muscle<br />
<strong>of</strong> Mice with Pompe Disease<br />
Dwight D. Koeberl, Songtao Li, Benjamin Farah, Andrew Bird, Paul<br />
M. Yen, Sarah P. Young.<br />
619<br />
Comparative In Vitro Potency <strong>of</strong> a Novel Disc-Derived<br />
<strong>Cell</strong> Population (Discospheres) and Mesenchymal Stem<br />
<strong>Cell</strong>s for the Treatment <strong>of</strong> Degenerative Disc Disease<br />
Lara I. Silverman, Galina Dulatova, Terry Tandeski, Kevin T. Foley.<br />
620<br />
Functional Analysis <strong>of</strong> GNE Expression Plasmids in<br />
Immortalized Human Myoblasts<br />
Yadira Valles-Ayoub, Rosangela Carbajo, Lucia Sandoval, Jorge<br />
Garcia-Figueroa, Sarah Stein, Zeshan Khokher, Daniel No, Babak<br />
Darvish, Jacques P. Tremblay, Daniel Darvish.<br />
Cancer Immunotherapy III<br />
621<br />
Tethered IL-15 Mutein on CD19-Speciic T <strong>Cell</strong>s<br />
Sustains Persistence When Tumor Antigen Is Low and<br />
Can Treat Minimal Residual Disease<br />
Lenka V. Hurton, Kirsten Switzer, Harjeet Singh, Marie A. Forget,<br />
Tiejuan Mi, Simon Olivares, Richard E. Champlin, Dean A. Lee,<br />
Laurence J. N. Cooper.<br />
Saturday, May 18, 2013<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013<br />
133
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