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 />
Thursday, May 16, 2013<br />
Poster Session I, continued<br />
4:00 PM - 6:00 PM<br />
Room: Exhibit Hall C/D<br />
Musculo-skeletal <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong> I<br />
168<br />
Activation, Expansion, and In Vivo Regenerative<br />
Potential <strong>of</strong> Pax3-Transduced Satellite <strong>Cell</strong>s: A Potential<br />
<strong>Cell</strong>-Based <strong>Therapy</strong> for Duchenne Muscular Dystrophy<br />
Antonio Filareto, Robert W. Arpke, Darko Bosnakovski, Radbod<br />
Darabi, Fabrizio Rinaldi, Auston Miller, Michael Kyba, Rita C. R.<br />
Perlingeiro.<br />
169<br />
<strong>Gene</strong>tic Correction <strong>of</strong> Facioscapulohumeral Dystrophy<br />
Using Telomere Insertion: Potential for Therapeutic<br />
Application<br />
Fabrizio Rinaldi, Linn Hartweck, Darko Bosnakovski, Erik<br />
Toso, Abhijit Dandapat, Radbod Darabi, Thomas Zwaka, Simon<br />
Nargarkhan, Rita Perlingeiro, Michael Kyba.<br />
170<br />
Enhanced Myogenic Differentiation Induced by<br />
Engineered MyoD Transcription Factors<br />
Ami M. Kabadi, Pratiksha I. Thakore, Charles A. Gersbach.<br />
171<br />
Viral Mediated Acute Mouse Model for Myotonic<br />
Dystrophy Type1<br />
Syed-Rehan A. Hussain, Lindsay Wallace, Danielle Grifin, Chrystal<br />
Montgomery, Scott Q. Harper, Zarife Sahenk, K. Reed Clark.<br />
172<br />
Pre-Clinical Eficacy Study <strong>of</strong> Beta-Sarcoglycan <strong>Gene</strong><br />
Transfer<br />
Eric R. Pozsgai, William E. Grose, Danielle A. Grifin, Kristin N.<br />
Heller, Jerry R. Mendell, Louise R. Rodino-Klapac.<br />
173<br />
The Cardiac Alkali Myosin Light Chain Can Restore<br />
Skeletal Muscle Function in a Mouse Model <strong>of</strong> Nemaline<br />
Myopathy<br />
Johan M. Lindqvist, Julien Ochala.<br />
176<br />
Engraftment <strong>of</strong> Mesenchymal Stromal <strong>Cell</strong>s That Can<br />
Differentiate To Form Myogenic <strong>Cell</strong>s Is Enhanced<br />
by Expressing IL-10 in Dog with Duchenne Muscular<br />
Dystrophy<br />
Yuko N. Kasahara, Hiromi H. Kinoh, Tomoko Chiyo, Hironori<br />
Okada, Takashi Okada, Shin’ichi Takeda.<br />
177<br />
Modulating <strong>Cell</strong> Cycle Inhibitor Expression To Enhance<br />
Skeletal Myoblast Engraftment<br />
Pratiksha I. Thakore, David G. Ousterout, Charles A. Gersbach.<br />
178<br />
Human Muscle Derived Stem <strong>Cell</strong>s Exhibit Bone<br />
Regeneration Capacity Similar to Bone Marrow<br />
Mesenchymal Stem <strong>Cell</strong>s When <strong>Gene</strong>tically Modiied To<br />
Express BMP2 Both In Vitro and In Vivo<br />
Xueqin Gao, Arvydas Usas, Ying Tang, Aiping Lu, Jian Tan, Johannes<br />
Schneppendahl, Nick Oyster, Bing Wang, Rocky Tuan, Johnny<br />
Huard.<br />
179<br />
Rapid Progression <strong>of</strong> Muscular Dystrophy in<br />
Dystrophin/Utrophin-/- Mice Is Associated with Muscle<br />
Progenitor <strong>Cell</strong> Exhaustion<br />
Aiping Lu, Minakshi Poddar, Ying Tang, Jonathan D. Proto,<br />
Xiaodong Mu, Bing Wang, Johnny Huard.<br />
180<br />
A New Mouse Model <strong>of</strong> DMD: A Tool for Therapeutic<br />
Development Directed at Exon Duplications<br />
Adeline Vulin, Nicolas Wein, Yuuki J. Kaminoh, Andrew R. Findlay,<br />
Kevin M. Flanigan.<br />
181<br />
Investigation <strong>of</strong> Dystromirs in the mdx Mouse<br />
Thomas C. Roberts.<br />
182<br />
Dose-Dependent Toxicity <strong>of</strong> Humanized Renilla<br />
Reniformis GFP (hrGFP) Limits Its Utility as a Reporter<br />
<strong>Gene</strong> in Mouse Muscle<br />
Lindsay M. Wallace, Andrew Moreo, K. Reed Clark, Scott Q. Harper.<br />
174<br />
<strong>Gene</strong>rating Dystrophin+ Myogenic <strong>Cell</strong>s from mdx<br />
Fibroblasts by Using a Triple-Recombinase iPS <strong>Cell</strong>-<br />
Based Strategy<br />
Jonathan M. Geisinger, Chunli Zhao, Alfonso P. Farrugio,<br />
Christopher L. Chavez, Christopher R. Bjornson, Tawny L. Neal,<br />
Michele P. Calos.<br />
175<br />
Alpha7 Integrin Partially Preserves the Mechanical<br />
Function <strong>of</strong> the Extensor Digitorum Longus Muscle in<br />
Dystrophin-Deicient Mice<br />
Chady H. Hakim, Dean J. Burkin, Dongsheng Duan.<br />
Infectious Diseases and Vaccines<br />
183<br />
Electroporation <strong>of</strong> ZFN mRNA Enables Eficient CCR5<br />
<strong>Gene</strong> Disruption in Mobilized Blood Hematopoietic<br />
Stem <strong>Cell</strong>s at Clinical Scale<br />
Paula M. Cannon, Jill E. Henley, Travis Wood, Lynn Truong, Jianbin<br />
Wang, Kenneth Kim, Jenny Yan, Philip D. Gregory, Gary Lee,<br />
Michael C. Holmes.<br />
100<br />
<strong>Final</strong> <strong>Program</strong> SALT LAKE CITY, UTAH May 15–18, 2013
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