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

作　　者：陶鹏飞 孙雅煊[1] 赵卓 黄汉昌[1] TAO Peng-Fei;SUN Ya-Xuan;ZHAO Zhuo;HUANG Han-Chang(Beijing Key Laboratory of Bioactive Substances and Functional Food, Beijing Union University, Beijing 100191, China)

机构地区：[1]北京联合大学生物活性物质与功能食品北京市重点实验室,北京100191

出　　处：《生命科学》2018年第3期333-341,共9页Chinese Bulletin of Life Sciences

基　　金：国家自然科学基金项目(31471587);北京市属高等学校高层次人才引进与培养计划项目(CIT&TCD 201504034);北京联合大学"启明星"大学生科技创新项目(201711417011)

摘　　要：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.

关 键 词：CRISPR/Cas系统 基因组编辑 神经退行性疾病 

分 类 号：R741.05[医药卫生—神经病学与精神病学]