Open AccessCRISPR for Neuromuscular Disorders: Gene Editing and Beyond
Author(s) -
Courtney S. Young,
April D. Pyle,
Melissa J. Spencer
Publication year - 2019
Publication title -
physiology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 3.14
H-Index - 125
eISSN - 1548-9213
pISSN - 1548-9221
DOI - 10.1152/physiol.00012.2019
Subject(s) - crispr , genome editing , biology , computational biology , medicine , gene , bioinformatics , genetics
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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