AAV-Mediated Gene Transfer Restores a Normal Muscle Transcriptome in a Canine Model of X-Linked Myotubular Myopathy

Multiple clinical trials employing recombinant adeno-associated viral (rAAV) vectors have been initiated for neuromuscular disorders, including Duchenne and limb-girdle muscular dystrophies, spinal muscular atrophy, and recently X-linked myotubular myopathy (XLMTM). Our previous work on a canine mod...

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Detalles Bibliográficos
Publicado en:Mol Ther
Main Authors: Dupont, Jean-Baptiste, Guo, Jianjun, Renaud-Gabardos, Edith, Poulard, Karine, Latournerie, Virginie, Lawlor, Michael W., Grange, Robert W., Gray, John T., Buj-Bello, Ana, Childers, Martin K., Mack, David L.
Formato: Artigo
Idioma:Inglês
Publicado: American Society of Gene & Cell Therapy 2020
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Original Article
Acceso en liña:https://ncbi.nlm.nih.gov/pmc/articles/PMC7000997/
https://ncbi.nlm.nih.gov/pubmed/31784415
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2019.10.018
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