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Article

Therapeutic Applications of CRISPR/Cas for Duchenne Muscular Dystrophy.

Current gene therapy - 1 Jan 2017

Wong Tatianna Wai Ying, Cohn Ronald D

Abstract excerpt

BACKGROUND: Duchenne muscular dystrophy (DMD) is an X-linked neuromuscular disease caused by the lack of dystrophin due to mutations in the DMD gene. Since dystrophin is essential in maintaining the integrity of the sarcolemmal membrane, the absence of the protein leads to muscle damage and DMD disease manifestation. Currently, there is no cure with only symptomatic management available. OBJECTIVE: The most...

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Identifiers

PMID: 29173172
DOI: 10.2174/1566523217666171121165046

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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