CRISPR/Cas基因组编辑技术及其在神经退行性疾病中的应用研究进展

Genome-editing technology of CRISPR/Cas and research progress of its application in neurodegenerative diseases

CRISPR/Cas系统实质上是一系列由RNA引导的核酸内切酶,其存在于约40%的细菌和90%的古细菌中,是细菌或古细菌为抵抗病毒或质粒而演化出的一种获得性免疫,主要作用是沉默入侵的外源核酸。近年来,CRISPR/Cas系统凭借其简单、高效且可编辑的靶向基因修饰能力,开启了基因编辑的新纪元。神经退行性疾病一直是困扰人类的重大疾病,而CRISPR/Cas系统的出现为治疗神经退行性疾病提供了新的思路和方法,现介绍CRISPR/Cas系统基因编辑的机制及其在神经退行性疾病中的应用。

CRISPR/Cas system,which exists in approximately 40% of the bacteria and 90% of the archaea,is a series of endonuclease guided by RNA.This system is a kind of adaptive immunity in bacteria to degrade the invasion of exogenous nucleic acid.Genome-editing technology of CRISPR/Cas is simple and effective for the targeting and the modification of a desired gene.In recent years,the CRISPR/Cas system has opened a new era in the field of genome-editing.Neurodegenerative diseases,including Huntingtin disease,Parkinson＇s disease,Alzheimer＇s disease and amyotrophic lateral sclerosis,have become one of the major problem on human health.The emergence of the CRISPR-Cas system provides a new thought and a novel method to carry out the researches and treatment on neurodegenerative diseases.This article mainly reviewed the mechanism on genome-editing of the CRISPR/Cas system and the application of CRISPR/Cas system on the research and treatment of neurodegenerative diseases.