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Article

The Future of Exon Skipping for Duchenne Muscular Dystrophy.

Human gene therapy - 1 May 2023

Aartsma-Rus Annemieke

Abstract excerpt

Antisense oligonucleotide (ASO)-mediated exon skipping can restore the open reading frame of dystrophin transcripts for Duchenne muscular dystrophy (DMD) patients. This allows production of internally deleted dystrophin proteins as found in the later onset, less severely progressive Becker muscular dystrophy. At present, ASOs that induce exon skipping and dystrophin restoration are approved for the treatment of...

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Share to Dystrophin
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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Share to Muscular Dystrophy, Duchenne

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Share to Oligonucleotides, Antisense

Share to Telomeres, Telomerase, and Senescence

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Identifiers

PMID: 36924282
DOI: 10.1089/hum.2023.026

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 1 Sept 2026.

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