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Article

Rescue of dystrophic muscle through U7 snRNA-mediated exon skipping.

Science (New York, N.Y.) - 3 Dec 2004

Goyenvalle Aurélie, Vulin Adeline, Fougerousse Françoise, Leturcq France, Kaplan Jean-Claude, Garcia Luis, Danos Olivier

Abstract excerpt

Most mutations in the dystrophin gene create a frameshift or a stop in the mRNA and are associated with severe Duchenne muscular dystrophy. Exon skipping that naturally occurs at low frequency sometimes eliminates the mutation and leads to the production of a rescued protein. We have achieved per...

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Share to Mice, Inbred mdx
Share to Muscle Contraction

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Share to Muscle Fibers, Skeletal

Share to Muscular Dystrophy, Animal

Share to Muscular Dystrophy, Duchenne

Share to Oligonucleotides, Antisense

Share to RNA, Small Nuclear

Source

Identifiers

PMID: 15528407
DOI: 10.1126/science.1104297

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 1 Sept 2026. Prepared record publication-15528407-2026-09-01.1. Manifest publication-graph-cohort-2026-09-01.1.

Source receipt: PubMed via Helena Literature Corpus, retrieved 1 Sept 2026.

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