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Journal articles
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.
https://hal-univ-evry.archives-ouvertes.fr/hal-02179358
Contributor : Melissa Goddard Connect in order to contact the contributor
Submitted on : Wednesday, July 10, 2019 - 4:40:55 PM
Last modification on : Wednesday, November 17, 2021 - 12:33:16 PM
Contributor : Melissa Goddard Connect in order to contact the contributor
Submitted on : Wednesday, July 10, 2019 - 4:40:55 PM
Last modification on : Wednesday, November 17, 2021 - 12:33:16 PM
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