CRISPR/Cas9 system and its applications in nervous system diseases

被引：16

作者：

Jiang, Haibin

Tang, Mengyan ^{[1
]}

Xu, Zidi

Wang, Yanan

Li, Mopu

Zheng, Shuyin

Zhu, Jianghu ^{[2
,3
,4
,5
,6
]}

Lin, Zhenlang ^{[6
]}

Zhang, Min ^{[2
,3
,4
,5
,6
]}

机构：

[1] Wenzhou Med Univ, Sch Med 2, Wenzhou 325035, Zhejiang, Peoples R China

[2] Wenzhou Med Univ, Sch Clin Med 1, Wenzhou 325035, Zhejiang, Peoples R China

[3] Wenzhou Med Univ, Yuying Childrens Hosp, Sch Med 2, Wenzhou 325027, Zhejiang, Peoples R China

[4] Key Lab Perinatal Med Wenzhou, Wenzhou 325027, Zhejiang, Peoples R China

[5] Key Lab Struct Malformat Children Zhejiang Prov, Wenzhou 325000, Zhejiang, Peoples R China

[6] Wenzhou Med Univ, Affiliated Hosp 2, Basic Med Res Ctr, 109 Xueyuan West Rd, Wenzhou 325027, Zhejiang, Peoples R China

来源：

GENES & DISEASES | 2024年 / 11卷 / 02期

基金：

中国国家自然科学基金;

关键词：

CRISPR/Cas9; Gene editing; Mutation; Neurological diseases; Therapeutics; PROVIDES ACQUIRED-RESISTANCE; PARKINSONS-DISEASE; ALZHEIMERS-DISEASE; MOUSE MODELS; HOMOLOGOUS RECOMBINATION; HUNTINGTONS-DISEASE; DRAVET SYNDROME; GENOMIC DNA; A-BETA; GENE;

D O I：

10.1016/j.gendis.2023.03.017

中图分类号：

Q5 [生物化学]; Q7 [分子生物学];

学科分类号：

071010 ; 081704 ;

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 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. & COPY; 2023 The Authors. Publishing services by Elsevier B.V. on behalf of KeAi Communications Co., Ltd. This is an open access article under the CC BY-NC-ND license (http://creativecommons. org/licenses/by-nc-nd/4.0/).

引用

页码：675 / 686

页数：12