CRISPR/Cas9 system and its applications in nervous system diseases

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 pro...

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Published inGenes & diseases Vol. 11; no. 2; pp. 675 - 686
Main Authors Jiang, Haibin, Tang, Mengyan, Xu, Zidi, Wang, Yanan, Li, Mopu, Zheng, Shuyin, Zhu, Jianghu, Lin, Zhenlang, Zhang, Min
Format Journal Article
LanguageEnglish
Published Elsevier B.V 01.03.2024
Chongqing Medical University
KeAi Communications Co., Ltd
Subjects
CRISPR/Cas9
Gene editing
Mutation
Neurological diseases
Review
Therapeutics
Neurological diseases
Mutation
CRISPR/Cas9
Gene editing
Therapeutics
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Summary: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 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.
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ISSN:2352-3042
2352-4820
2352-3042
DOI:10.1016/j.gendis.2023.03.017