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Article

[Gene therapy for muscular dystrophy].

No to hattatsu = Brain and development - 1 Mar 2004

Takeda Shin'ichi

Abstract excerpt

Duchenne muscular dystrophy (DMD) is an X-linked, lethal muscle disorder caused by mutations in the dystrophin gene. Although an adeno-associated virus (AAV) vector-mediated gene transfer provides an attractive approach to the treatment of DMD, limitation in insertion size up to 4.9 kb excludes incorporation of a full-length dystrophin cDNA (14 kb) into an AAV vector. We previously generated micro-dystrophin...

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Share to Mice, Transgenic
Share to Muscular Dystrophy, Animal
Share to Muscular Dystrophy, Duchenne

Methodology Data sources Editorial policy Privacy Delete account & data About Contact Helena Bioinformatics

Share to Genetic Vectors

Share to Dependovirus

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Source

Identifiers

PMID: 15031985

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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