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CRISPR for Neuromuscular Disorders: Gene Editing and Beyond
Published Web Location
https://doi.org/10.1152/physiol.00012.2019Abstract
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.
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