CRISPR for Neuromuscular Disorders: Gene Editing and Beyond.
CRISPR for Neuromuscular Disorders: Gene Editing and Beyond.
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DOI：
10.1152/physiol.00012.2019
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发表时间：
2019-09
期刊：
影响因子：
8.4
通讯作者：
C. Young;A. Pyle;M. Spencer
中科院分区：
文献类型：
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作者：
C. Young;A. Pyle;M. Spencer
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.
