CRISPR 用于神经肌肉疾病：基因编辑及其他。
CRISPR for Neuromuscular Disorders: Gene Editing and Beyond.
机构信息
Department of Neurology, University of California, Los Angeles, California.
Center for Duchenne Muscular Dystrophy at UCLA, University of California, Los Angeles, California.
出版信息
Physiology (Bethesda). 2019 Sep 1;34(5):341-353. doi: 10.1152/physiol.00012.2019.
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.
摘要
这是一篇综述，描述了 CRISPR/Cas 介导的神经肌肉疾病 (NMD) 疗法的进展。我们探讨了 CRISPR 介导的编辑和失活 Cas 方法作为多种 NMD 的潜在治疗策略。最后，还讨论了治疗方面的考虑因素，包括递送和脱靶效应。
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