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Article

Morpholino oligomer-mediated exon skipping averts the onset of dystrophic pathology in the mdx mouse.

Molecular therapy : the journal of the American Society of Gene Therapy - 1 Sept 2007

Fletcher Sue, Honeyman Kaite, Fall Abbie M, Harding Penny L, Johnsen Russell D, Steinhaus Joshua P, Moulton Hong M, Iversen Patrick L, Wilton Stephen D

Abstract excerpt

Duchenne and Becker muscular dystrophies are allelic disorders arising from mutations in the dystrophin gene. Duchenne muscular dystrophy is characterized by an absence of functional protein, whereas Becker muscular dystrophy, commonly caused by in-frame deletions, shows synthesis of partially functional protein. Anti-sense oligonucleotides can induce specific exon removal during processing of the dystrophin...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Share to Muscular Dystrophy, Duchenne

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Source

Identifiers

PMID: 17579573
DOI: 10.1038/sj.mt.6300245

Provenance

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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Morpholino oligomer-mediated exon skipping averts the onset of dystrophic pathology in the mdx mouse.PMID 17579573Read ↗

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