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Scientific Articles

CRISPR for Neuromuscular Disorders: Gene Editing and Beyond

key information

source: Physiology

year: 2019

authors: Young CS, Pyle AD, Spencer MJ


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.

organization: University of California, USA

DOI: 10.1152/physiol.00012.2019

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