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Article

Emerging genetic therapies to treat Duchenne muscular dystrophy.

Current opinion in neurology - 1 Oct 2009

Nelson Stanley F, Crosbie Rachelle H, Miceli M Carrie, Spencer Melissa J

Abstract excerpt

PURPOSE OF REVIEW: Duchenne muscular dystrophy is a progressive muscle degenerative disease caused by dystrophin mutations. The purpose of this review is to highlight two emerging therapies designed to repair the primary genetic defect, called 'exon skipping' and 'nonsense codon suppression'. RECENT FINDINGS: A drug, PTC124, was identified that suppresses nonsense codon translation termination. PTC124 can lead to...

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Share to Dystrophin
Share to Exons
Share to Codon, Terminator

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Share to Muscular Dystrophy, Duchenne

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Source

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Identifiers

PMID: 19745732
DOI: 10.1097/WCO.0b013e32832fd487
PMCID: PMC2856442

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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