Article
Long-term effects of systemic gene therapy in a canine model of myotubular myopathy.
Muscle & nerve - 1 Nov 2017
Elverman Matthew, Goddard Melissa A, Mack David, Snyder Jessica M, Lawlor Michael W, Meng Hui, Beggs Alan H, Buj-Bello Ana, Poulard Karine, Marsh Anthony P, Grange Robert W, Kelly Valerie E, Childers Martin K
Abstract excerpt
INTRODUCTION: X-linked myotubular myopathy (XLMTM), a devastating pediatric disease caused by the absence of the protein myotubularin, results from mutations in the MTM1 gene. While there is no cure for XLMTM, we previously reported effects of MTM1 gene therapy using adeno-associated virus (AAV) vector on muscle weakness and pathology in MTM1-mutant dogs. Here, we followed 2 AAV-infused dogs over 4 years....
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