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.

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