CRISPR/Cas9 system and its applications in nervous system diseases

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外文摘要：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 recog-nize 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.

外文关键词：

CRISPR/Cas9Gene editingMutationNeurological diseasesTherapeutics

作者：

Haibin Jiang、Mengyan Tang、Zidi Xu、Yanan Wang、Mopu Li、Shuyin Zheng、Jianghu Zhu、Zhenlang Lin、Min Zhang

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作者单位：

The Second School of Medicine,Wenzhou Medical University,Wenzhou,Zhejiang 325035,China

The First School of Medicine,Wenzhou Medical University,Wenzhou,Zhejiang 325035,China

Department of Pediatrics,The Second School of Medicine,The Second Affiliated Hospital and Yuying Children's Hospital of Wenzhou Medical University,Wenzhou,Zhejiang 325027,China

Key Laboratory of Perinatal Medicine of Wenzhou,Wenzhou,Zhejiang 325027,China

Key Laboratory of Structural Malformations in Children of Zhejiang Province,Wenzhou,Zhejiang 325000,China

Zhejiang Provincial Clinical Research Center for Pediatric Disease,Wenzhou,Zhejiang 325027,China

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基金：

国家自然科学基金

项目编号：

82271747

出版年：

2024

DOI：

10.1016/j.gendis.2023.03.017

基因与疾病(英文)

ISSN：

年,卷(期)：2024.11(2)

参考文献量149