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Article

Dystrophin Gene-Editing Stability Is Dependent on Dystrophin Levels in Skeletal but Not Cardiac Muscles.

Molecular therapy : the journal of the American Society of Gene Therapy - 3 Mar 2021

Bengtsson Niclas E, Tasfaout Hichem, Hauschka Stephen D, Chamberlain Jeffrey S

Abstract excerpt

Gene editing is often touted as a permanent method for correcting mutations, but its long-term benefits in Duchenne muscular dystrophy (DMD) may depend on sufficiently high editing efficiencies to halt muscle degeneration. Here, we explored the persistence of dystrophin expression following recombinant adeno-associated virus serotype 6 (rAAV6):CRISPR-Cas9-mediated multi-exon deletion/reframing in systemically...

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Source

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Identifiers

PMID: 33160075
DOI: 10.1016/j.ymthe.2020.11.003
PMCID: PMC7934576

Genes

DMD

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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