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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Detaylı Bibliyografya
Yayımlandı:Mol Ther
Asıl Yazarlar: Hayashita-Kinoh, Hiromi, Yugeta, Naoko, Okada, Hironori, Nitahara-Kasahara, Yuko, Chiyo, Tomoko, Okada, Takashi, Takeda, Shin'ichi
Materyal Türü: Artigo
Dil:Inglês
Baskı/Yayın Bilgisi: Nature Publishing Group 2015
Konular:
Original Article
Online Erişim: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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