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Article

In Vivo Genome Editing Restores Dystrophin Expression and Cardiac Function in Dystrophic Mice.

Circulation research - 29 Sept 2017

El Refaey Mona, Xu Li, Gao Yandi, Canan Benjamin D, Adesanya T M Ayodele, Warner Sarah C, Akagi Keiko, Symer David E, Mohler Peter J, Ma Jianjie, Janssen Paul M L, Han Renzhi

Abstract excerpt

RATIONALE: Duchenne muscular dystrophy is a severe inherited form of muscular dystrophy caused by mutations in the reading frame of the dystrophin gene disrupting its protein expression. Dystrophic cardiomyopathy is a leading cause of death in Duchenne muscular dystrophy patients, and currently no effective treatment exists to halt its progression. Recent advancement in genome editing technologies offers a...

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Identifiers

PMID: 28790199
DOI: 10.1161/CIRCRESAHA.117.310996
PMCID: PMC5623072

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 5 Sept 2026. Manifest publication-graph-cohort-2026-09-05.1.

Source receipt: pubmed, retrieved 5 Sept 2026.

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