Intra-Amniotic rAAV-Mediated Microdystrophin Gene Transfer Improves Canine X-Linked Muscular Dystrophy and May Induce Immune Tolerance

Duchenne muscular dystrophy (DMD) is a severe congenital disease due to mutations in the dystrophin gene. Supplementation of dystrophin using recombinant adenoassociated virus vector has promise as a treatment of DMD, although therapeutic benefit of the truncated dystrophin still remains to be eluci...

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Publicado en:Mol Ther
Autores principales: Hayashita-Kinoh, Hiromi, Yugeta, Naoko, Okada, Hironori, Nitahara-Kasahara, Yuko, Chiyo, Tomoko, Okada, Takashi, Takeda, Shin'ichi
Formato: Artigo
Lenguaje:Inglês
Publicado: Nature Publishing Group 2015
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Original Article
Acceso en línea:https://ncbi.nlm.nih.gov/pmc/articles/PMC4395797/
https://ncbi.nlm.nih.gov/pubmed/25586688
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2015.5
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