CRISPR for Neuromuscular Disorders: Gene Editing and Beyond.
Physiology (Bethesda, Md.), 34(5): 341-353 PMID 31389773 DOI 10.1152/physiol.00012.2019
Аннотация
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.