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Review
. 2019 Sep 1;34(5):341-353.
doi: 10.1152/physiol.00012.2019.

CRISPR for Neuromuscular Disorders: Gene Editing and Beyond

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Free PMC article
Review

CRISPR for Neuromuscular Disorders: Gene Editing and Beyond

Courtney S Young et al. Physiology (Bethesda). .
Free PMC article

Abstract

This is a review describing advances in CRISPR/Cas-mediated therapies for neuromuscular disorders (NMDs). We explore both CRISPR-mediated editing and dead Cas approaches as potential therapeutic strategies for multiple NMDs. Last, therapeutic considerations, including delivery and off-target effects, are also discussed.

Keywords: CRISPR/Cas; gene editing; gene therapy; muscular dystrophy; neuromuscular disorders.

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