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Article

Human dystrophin expression corrects the myopathic phenotype in transgenic mdx mice.

Human molecular genetics - 1 Apr 1992

Wells D J, Wells K E, Walsh F S, Davies K E, Goldspink G, Love D R, Chan-Thomas P, Dunckley M G, Piper T, Dickson G

Abstract excerpt

Duchenne and the less severe Becker form of muscular dystrophy (DMD,BMD) result from genetic deficiency in the level and/or activity of the protein dystrophin. The recent availability of cDNA based minigenes encoding recombinant dystrophin polypeptides has raised the possibility of somatic gene transfer as a therapeutic approach to treat dystrophin deficiency. In this respect, the mdx mouse provides a useful...

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PMID: 1301134
DOI: 10.1093/hmg/1.1.35

Genes

DMD

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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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