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CRISPR/Cas9系统及其在神经系统疾病中的应用。

CRISPR/Cas9 system and its applications in nervous system diseases.

作者信息

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

机构信息

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

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

出版信息

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

DOI:10.1016/j.gendis.2023.03.017
PMID:37692518
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC10491921/
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 loci, 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.

摘要

成簇规律间隔短回文重复序列（CRISPR）/CRISPR相关蛋白9（Cas9）系统是许多细菌和古生菌的一种后天免疫系统，由基因座、基因及其相关蛋白组成。该系统能够识别外源DNA，并利用Cas9蛋白的核酸酶活性切割DNA双链，随后通过DNA修复实现碱基插入或缺失。近年来，多项实验室研究和临床研究揭示了CRISPR/Cas9系统在神经疾病中的治疗作用。本文综述了CRISPR/Cas9介导的基因编辑技术及其在神经疾病临床应用中的潜力。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/7998/10491921/45e3ea3e58c3/gr1.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/7998/10491921/45e3ea3e58c3/gr1.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/7998/10491921/45e3ea3e58c3/gr1.jpg

相似文献

[1]
CRISPR/Cas9 system and its applications in nervous system diseases.

Genes Dis. 2023-4-13

[2]
Clustered Regularly Interspaced Short Palindromic Repeats and Clustered Regularly Interspaced Short Palindromic Repeats-Associated Protein 9 System: Factors Affecting Precision Gene Editing Efficiency and Optimization Strategies.

Hum Gene Ther. 2023-12

[3]
Applications of Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) as a Genetic Scalpel for the Treatment of Cancer: A Translational Narrative Review.

Cureus. 2023-12-6

[4]
Design of a generic CRISPR-Cas9 approach using the same sgRNA to perform gene editing at distinct loci.

BMC Biotechnol. 2019-3-20

[5]
Advances in therapeutic application of CRISPR-Cas9.

Brief Funct Genomics. 2020-5-20

[6]
[Recent developments in enhancing the efficiency of CRISPR/Cas9- mediated knock-in in animals].

Yi Chuan. 2020-7-20

[7]
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Trends Biotechnol. 2023-8

[8]
The application and progression of CRISPR/Cas9 technology in ophthalmological diseases.

Eye (Lond). 2023-3

[9]
CRISPR/Cas9 for genome editing: progress, implications and challenges.

Hum Mol Genet. 2014-9-15

[10]
CRISPR/Cas9-based Genome Editing of Pseudomonas aeruginosa.

Methods Mol Biol. 2024

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Int J Mol Sci. 2025-6-4

[2]
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Curr Pharm Des. 2025-4-21

[3]
CRISPR/Cas9-Based therapeutics as a promising strategy for management of Alzheimer's disease: progress and prospects.

Front Cell Neurosci. 2025-4-7

[4]
Non-Drug and Non-Invasive Therapeutic Options in Alzheimer's Disease.

Biomedicines. 2025-1-1

[5]
CRISPR/Cas9 System as a Promising Therapy in Thalassemia and Sickle Cell Disease: A Systematic Review of Clinical Trials.

Mol Biotechnol. 2025-1-10

[6]
Behavioral analysis of motor and non-motor impairment in rodent models of Parkinson's disease.

Front Aging Neurosci. 2024-12-23

[7]
Developmental and Epileptic Encephalopathies: Need for Bridging the Gaps Between Clinical Syndromes and Underlying Genetic Etiologies.

Indian J Pediatr. 2025-1

[8]
Emerging Gene Therapies for Alzheimer's and Parkinson's Diseases: An Overview of Clinical Trials and Promising Candidates.

Cureus. 2024-8-16

[9]
Regulates Retina Development in Zebrafish.

Int J Mol Sci. 2024-9-5

[10]
Advancement of animal and poultry nutrition: Harnessing the power of CRISPR-Cas genome editing technology.

J Adv Vet Anim Res. 2024-6-21

本文引用的文献

[1]
Locus-Specific Isolation of the Chromatin Identifies Regulators Relevant to Pluripotency of Mouse Embryonic Stem Cells and Reprogramming of Somatic Cells.

Int J Mol Sci. 2022-12-3

[2]
CAPTURE of the Human U2 snRNA Genes Expands the Repertoire of Associated Factors.

Biomolecules. 2022-5-14

[3]
CRISPR-Based Genome-Editing Tools for Huntington's Disease Research and Therapy.

Neurosci Bull. 2022-11

[4]
CAR T cell killing requires the IFNγR pathway in solid but not liquid tumours.

Nature. 2022-4

[5]
CRISPR-Cas9 Gene Editing Protects from the A53T-SNCA Overexpression-Induced Pathology of Parkinson's Disease .

CRISPR J. 2022-2

[6]
S-Nitrosylation of p62 Inhibits Autophagic Flux to Promote α-Synuclein Secretion and Spread in Parkinson's Disease and Lewy Body Dementia.

J Neurosci. 2022-4-6

[7]
Application of CRISPR/Cas9 in Alzheimer's Disease.

Front Neurosci. 2021-12-21

[8]
Applications of CRISPR-Cas9 Technology to Genome Editing in Glioblastoma Multiforme.

Cells. 2021-9-7

[9]
Lewy Body-like Inclusions in Human Midbrain Organoids Carrying Glucocerebrosidase and α-Synuclein Mutations.

Ann Neurol. 2021-9

[10]
Gene Editing and Modulation: the Holy Grail for the Genetic Epilepsies?

Neurotherapeutics. 2021-7

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