CRISPR for Neuromuscular Disorders: Gene Editing and Beyond

Physiology
Courtney S YoungMelissa J Spencer

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.

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Citations

Aug 8, 2019·Physiology·Gary C Sieck
Oct 15, 2020·Antioxidants·Judith Cantó-SantosGlòria Garrabou
Jul 15, 2021·Nature Reviews. Molecular Cell Biology·James J DowlingMelissa J Spencer

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Methods Mentioned

BETA
exome sequencing

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