《生命科学》 2018, 30(3): 333-341

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

陶鹏飞，孙雅煊，赵　卓，黄汉昌*

(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)

摘　要：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。

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

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)

Abstract: Abstract: 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.