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Article

Gentamicin-induced readthrough of stop codons in Duchenne muscular dystrophy.

Annals of neurology - 1 Jun 2010

Malik Vinod, Rodino-Klapac Louise R, Viollet Laurence, Wall Cheryl, King Wendy, Al-Dahhak Roula, Lewis Sarah, Shilling Christopher J, Kota Janaiah, Serrano-Munuera Carmen, Hayes John, Mahan John D, Campbell Katherine J, Banwell Brenda, Dasouki Majed, Watts Victoria, Sivakumar Kumaraswamy, Bien-Willner Ricardo, Flanigan Kevin M, Sahenk Zarife, Barohn Richard J, Walker Christopher M, Mendell Jerry R

Abstract excerpt

OBJECTIVE: The objective of this study was to establish the feasibility of long-term gentamicin dosing to achieve stop codon readthrough and produce full-length dystrophin. Mutation suppression of stop codons, successfully achieved in the mdx mouse using gentamicin, represents an important evolving treatment strategy in Duchenne muscular dystrophy (DMD). METHODS: Two DMD cohorts received 14-day gentamicin...

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Identifiers

PMID: 20517938
DOI: 10.1002/ana.22024

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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