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Article

Intra-amniotic rAAV-mediated microdystrophin gene transfer improves canine X-linked muscular dystrophy and may induce immune tolerance.

Molecular therapy : the journal of the American Society of Gene Therapy - 1 Apr 2015

Hayashita-Kinoh Hiromi, Yugeta Naoko, Okada Hironori, Nitahara-Kasahara Yuko, Chiyo Tomoko, Okada Takashi, Takeda Shin'ichi

Abstract excerpt

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 elucidated. Besides, host immune responses against the vector as well as transgene products have been denoted in the...

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Source

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Identifiers

PMID: 25586688
DOI: 10.1038/mt.2015.5
PMCID: PMC4395797

Genes

DMD

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-08.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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Intra-amniotic rAAV-mediated microdystrophin gene transfer improves canine X-linked muscular dystrophy and may induce immune tolerance.PMID 25586688Read ↗

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