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Article

Therapeutic antisense-induced exon skipping in cultured muscle cells from six different DMD patients.

Human molecular genetics - 15 Apr 2003

Aartsma-Rus Annemieke, Janson Anneke A M, Kaman Wendy E, Bremmer-Bout Mattie, den Dunnen Johan T, Baas Frank, van Ommen Gert-Jan B, van Deutekom Judith C T

Abstract excerpt

The dystrophin deficiency leading to the severely progressing muscle degeneration in Duchenne muscular dystrophy (DMD) patients is caused by frame-shifting mutations in the DMD gene. We are developing a reading frame correction therapy aimed at the antisense-induced skipping of targeted exons from the pre-mRNA. Despite introducing a (larger) deletion, an in-frame transcript is generated that allows the synthesis...

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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: 12668614
DOI: 10.1093/hmg/ddg100

Genes

DMD

Provenance

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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