新型基因编辑技术CRISPR/Cas9系统研究现状

doi:10.3969/j.issn.1000-1565.2016.01.014

摘要/Abstract

摘要： 规律成簇间隔短回文重复序列及其相关系统(clustered regularly interspaced short palindromic repeats/CRISPR associated,CRISPR/Cas system)是细菌和古细菌防御外来噬菌体、质粒或其他外源DNA侵染的获得性免疫系统.依据Cas蛋白种类和同源性,CRISPR/Cas系统被分为3类,其中,Ⅰ类和Ⅲ类需要多种Cas蛋白参与,而Ⅱ类系统,即CRISPR/Cas9组成简单,仅需Cas9蛋白参与即可.经过遗传工程改造后的CRISPR/Cas9已经作为一种新型的基因编辑工具被用于多种生物的基因组编辑.本文就CRISPR/Cas9系统的发现、结构组成、作用机制、研究现状、面临的困境及应用前景等几方面进行了总结.

关键词: 基因编辑, CRISPR/Cas9系统, CRISPR干扰, 干扰机制, 基因治疗

Abstract: Clustered regularly interspaced short palindromic repeats(CRISPR)and associated proteins(Cas)comprise the CRISPR/Cas system,which confers adaptive immunity against foreign invading phages,plasmids and other mobile elements in many bacteria and most archaea.On the basis of the species and homology of the Cas protein,CRISPR/Cas systems have been classified into three distinct groups(type Ⅰ~Ⅲ).The component of type Ⅱ CRISPR/Cas9 system is the simplest one,and is well-charactered in resent study.CRISPR/Cas9 system modified by genetic engineering has proven to be an efficient gene-targeting tool in various organisms.Here,the auther review the discovery,components,molecular mechanism,applications,challenges and future prospect of CRISPR/Cas9-mediated genome editing.

Key words: gene editing, CRISPR/Cas9, CRISPRi, interference mechanism, gene therapy

中图分类号:

Q789

骞蕾阳,周志军,常岩林. 新型基因编辑技术CRISPR/Cas9系统研究现状[J]. 河北大学学报(自然科学版), 2016, 36(1): 84-93.

QIAN Leiyang,ZHOU Zhijun,CHANG Yanlin. Research status of novel gene editing technology of CRISPR/Cas9 system[J]. Journal of Hebei University (Natural Science Edition), 2016, 36(1): 84-93.

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