CRISPR/Cas9系统及其在神经系统疾病中的应用。
CRISPR/Cas9 system and its applications in nervous system diseases.
作者信息
Jiang Haibin, Tang Mengyan, Xu Zidi, Wang Yanan, Li Mopu, Zheng Shuyin, Zhu Jianghu, Lin Zhenlang, Zhang Min
机构信息
The Second School of Medicine, Wenzhou Medical University, Wenzhou, Zhejiang 325035, China.
The First School of Medicine, Wenzhou Medical University, Wenzhou, Zhejiang 325035, China.
出版信息
Genes Dis. 2023 Apr 13;11(2):675-686. doi: 10.1016/j.gendis.2023.03.017. eCollection 2024 Mar.
The clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated protein 9 (Cas9) system is an acquired immune system of many bacteria and archaea, comprising loci, genes, and its associated proteins. This system can recognize exogenous DNA and utilize the Cas9 protein's nuclease activity to break DNA double-strand and to achieve base insertion or deletion by subsequent DNA repair. In recent years, multiple laboratory and clinical studies have revealed the therapeutic role of the CRISPR/Cas9 system in neurological diseases. This article reviews the CRISPR/Cas9-mediated gene editing technology and its potential for clinical application against neurological diseases.
成簇规律间隔短回文重复序列（CRISPR）/CRISPR相关蛋白9（Cas9）系统是许多细菌和古生菌的一种后天免疫系统，由基因座、基因及其相关蛋白组成。该系统能够识别外源DNA，并利用Cas9蛋白的核酸酶活性切割DNA双链，随后通过DNA修复实现碱基插入或缺失。近年来，多项实验室研究和临床研究揭示了CRISPR/Cas9系统在神经疾病中的治疗作用。本文综述了CRISPR/Cas9介导的基因编辑技术及其在神经疾病临床应用中的潜力。
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