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Article

Moving towards successful exon-skipping therapy for Duchenne muscular dystrophy.

Journal of human genetics - 1 Oct 2017

Nakamura Akinori

Abstract excerpt

Duchenne muscular dystrophy (DMD) is an X chromosome-linked lethal muscular disorder with progressing muscle wasting and weakness caused by mutations in the gene encoding a subsarcolemmal protein dystrophin. For a long time, there was no effective cure; however, advances in molecular biology have allowed the development of radical treatment approaches. Among them, exon-skipping therapy using antisense...

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Source

Identifiers

PMID: 28566768
DOI: 10.1038/jhg.2017.57

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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