503. Minimally Effective Dose of Systemic AAV8-MTM1 Needed To Prolong Survival and Correct Severe Muscle Pathology in a Canine Model of X-Linked Myotubular Myopathy
David L. Mack(California Institute for Regenerative Medicine), Martin K. Childers(University of Washington), M. W. O'CALLAGHAN, Robert W. Grange(Virginia Tech), Michael W. Lawlor(Medical College of Wisconsin), Virginie Latournerie(Inserm), Karine Poulard(Genethon (France)), Laurine Buscara(Inserm), Hui Meng(Medical College of Wisconsin), Alan H. Beggs(Boston Children's Hospital), Jon Doering(Virginia Tech), Jessica M. Snyder(University of Washington), Melissa Goddard(Forest Institute), P. Véron(Genethon (France)), Jennifer L. Strande(Amgen (United States)), Fujun Liu(University of Florida), Lin Yang(Northwest University), Samia Martin(Genethon (France)), Federico Mingozzi(Spark Therapeutics (United States)), Stefan Czerniecki(California Institute for Regenerative Medicine), Anna Buj‐Bello(Pediatrics and Genetics), Philippe Moullier(Inserm), Christine Le Bec(Genethon (France)), Fulvio Mavilio(The San Raffaele Telethon Institute for Gene Therapy)
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