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CRISPR/Cas9 system and its applications in nervous system diseases

REVIEW ARTICLE

CRISPR/Cas9 system and its applications in nervous system diseases

Haibin Jiang
Mengyan Tang
Zidi Xu
Yanan Wang
Mopu Li
Shuyin Zheng
Jianghu Zhu
Zhenlang Lin
Min Zhang
Genes & Diseases第11卷, 第2期pp.675-686纸质出版 2024-03-01在线发表 2023-04-13
197305

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 CRISPR loci, Cas genes, and its associated proteins. This system can recognize exogenous DNA and utilize the Cas9 protein's nuclease activity to break DNA doublestrand 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.

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CRISPR/Cas9Gene editingMutationNeurological diseasesTherapeutics