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

doi:10.1016/j.gendis.2023.03.017

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CRISPR/Cas9 system and its applications in nervous system diseases

Review article | 更新时间：2024-02-04

- CRISPR/Cas9 system and its applications in nervous system diseases
- Genes & Diseases Vol. 11, Issue 2, (2024)
- 作者机构：
  
  1. Key Laboratory of Perinatal Medicine of Wenzhou,Zhejiang,Wenzhou,China,325027
  2. The Second School of Medicine, Wenzhou Medical University,Zhejiang,Wenzhou,China,325035
  3. Key Laboratory of Structural Malformations in Children of Zhejiang Province,Zhejiang,Wenzhou,China,325000
  4. Department of Pediatrics, The Second School of Medicine, The Second Affiliated Hospital and Yuying Children's Hospital of Wenzhou Medical University,Zhejiang,Wenzhou,China,325027
  5. Zhejiang Provincial Clinical Research Center for Pediatric Disease,Zhejiang,Wenzhou,China,325027
  6. The First School of Medicine, Wenzhou Medical University,Zhejiang,Wenzhou,China,325035
- 作者简介：
- 基金信息：
- DOI：10.1016/j.gendis.2023.03.017
  CLC：
- Published：2024，
  
  Published Online：1 March 2024，
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Haibin Jiang, Mengyan Tang, Zidi Xu, et al. CRISPR/Cas9 system and its applications in nervous system diseases. [J]. Genes & Diseases 11(2).(2024)
DOI：

Haibin Jiang, Mengyan Tang, Zidi Xu, et al. CRISPR/Cas9 system and its applications in nervous system diseases. [J]. Genes & Diseases 11(2).(2024) DOI： 10.1016/j.gendis.2023.03.017.

摘要

Abstract

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.

关键词

Keywords

CRISPR/Cas9Gene editingMutationNeurological diseasesTherapeutics

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