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Article

Expression of full-length and truncated dystrophin mini-genes in transgenic mdx mice.

Human molecular genetics - 1 Aug 1995

Phelps S F, Hauser M A, Cole N M, Rafael J A, Hinkle R T, Faulkner J A, Chamberlain J S

Abstract excerpt

Duchenne and Becker muscular dystrophy are caused by defects in the dystrophin gene, and are candidates for treatment by gene therapy. We have shown previously that overexpression of a full-length dystrophin cDNA prevents the development of dystrophic symptoms in mdx mice. We show here that this functional correction can be achieved by expressing the full-length muscle isoform at a lower level than is present in...

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Source

Identifiers

PMID: 7581361
DOI: 10.1093/hmg/4.8.1251

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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Expression of full-length and truncated dystrophin mini-genes in transgenic mdx mice.PMID 7581361Read ↗

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Lentiviral vectors can be used for full-length dystrophin gene therapySemantic proximity Read ↗

Dystrophin rescue by trans-splicing: a strategy for DMD genotypes not eligible for exon skipping approaches.Semantic proximity Read ↗

Antisense-induced exon skipping and synthesis of dystrophin in the mdx mouseSemantic proximity Read ↗

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