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Article

Micro-dystrophin cDNA ameliorates dystrophic phenotypes when introduced into mdx mice as a transgene.

Biochemical and biophysical research communications - 17 May 2002

Sakamoto Miki, Yuasa Katsutoshi, Yoshimura Madoka, Yokota Toshifumi, Ikemoto Takaaki, Suzuki Misao, Dickson George, Miyagoe-Suzuki Yuko, Takeda Shin'ichi

Abstract excerpt

The adeno-associated virus vector is a good tool for gene transfer into skeletal muscle, but the length of a gene that can be incorporated is limited. To develop a gene therapy for Duchenne muscular dystrophy, we generated a series of rod-truncated micro-dystrophin cDNAs: M3 (one rod repeat, 3.9 kb), AX11 (three rod repeats, 4.4 kb), and CS1 (four rod repeats, 4.9 kb). These micro-dystrophins, driven by a CAG...

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Identifiers

PMID: 12054513
DOI: 10.1016/S0006-291X(02)00362-5

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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Micro-dystrophin cDNA ameliorates dystrophic phenotypes when introduced into mdx mice as a transgene.PMID 12054513Read ↗

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