Department of Neurology, University of California, Los Angeles, California.
Center for Duchenne Muscular Dystrophy at UCLA, University of California, Los Angeles, California.
Physiology (Bethesda). 2019 Sep 1;34(5):341-353. doi: 10.1152/physiol.00012.2019.
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.
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。
