Long-term effects of systemic gene therapy in a canine model of myotubular myopathy

Abstract : 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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https://hal-univ-evry.archives-ouvertes.fr/hal-02179358
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Submitted on : Wednesday, July 10, 2019 - 4:40:55 PM
Last modification on : Wednesday, August 21, 2019 - 11:02:06 AM

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Matthew Elverman, Melissa Goddard, David Mack, Jessica Snyder, Michael Lawlor, et al.. Long-term effects of systemic gene therapy in a canine model of myotubular myopathy. Muscle & nerve. Supplement., 2017, 56 (5), pp.943-953. ⟨10.1002/mus.25658⟩. ⟨hal-02179358⟩

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