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

Matthew Elverman Melissa Goddard 1 David Mack Jessica Snyder Michael Lawlor Hui Meng Alan Beggs 2 Ana Buj-Bello 3 Karine Poulard 1 Anthony Marsh Robert Grange 4 Valerie Kelly Martin Childers 5
1 INTEGRARE - Approches génétiques intégrées et nouvelles thérapies pour les maladies rares
2 Genomics Program and Division of Genetics
3 Généthon
4 LAM - Laboratoire d'Astrophysique de Marseille
5 Wake Forest University
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.
Keywords : adeno-associated virus canine gait gene therapy muscle myotubular myopathy neuromuscular disease
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Journal articles
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https://hal-univ-evry.archives-ouvertes.fr/hal-02179358
Contributor : Melissa Goddard <>
Submitted on : Wednesday, July 10, 2019 - 4:40:55 PM
Last modification on : Thursday, March 4, 2021 - 3:50:24 PM

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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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