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

CRISPR/Cas9 system and its applications in nervous system diseases

Genes Dis. 2023 Apr 13;11(2):675-686. doi: 10.1016/j.gendis.2023.03.017. eCollection 2024 Mar.

Authors

Haibin Jiang¹, Mengyan Tang², Zidi Xu¹, Yanan Wang¹, Mopu Li¹, Shuyin Zheng¹, Jianghu Zhu^{3

4

5

6}, Zhenlang Lin^{3

4

5

6}, Min Zhang^{3

4

5

6}

Affiliations

¹ The Second School of Medicine, Wenzhou Medical University, Wenzhou, Zhejiang 325035, China.
² The First School of Medicine, Wenzhou Medical University, Wenzhou, Zhejiang 325035, China.
³ 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.
⁴ Key Laboratory of Perinatal Medicine of Wenzhou, Wenzhou, Zhejiang 325027, China.
⁵ Key Laboratory of Structural Malformations in Children of Zhejiang Province, Wenzhou, Zhejiang 325000, China.
⁶ Zhejiang Provincial Clinical Research Center for Pediatric Disease, Wenzhou, Zhejiang 325027, China.

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.

Keywords: CRISPR/Cas9; Gene editing; Mutation; Neurological diseases; Therapeutics.

Publication types

Review