摘要：神经肌肉单基因病是目前最常见也最严重的单基因遗传病之一。基因编辑技术,具有治疗包括神经肌肉单基因病在内的许多单基因病的临床潜力。本文主要总结已发表的基因编辑技术在神经肌肉单基因遗传病治疗中的文章,并对其安全性进行简要分析,以期为今后的进一步实验提供参考。
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