新型基因编辑技术CRISPR/Cas9系统研究现状

Research status of novel gene editing technology of CRISPR/Cas9 system

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.

Clustered regularly interspaced short palindromic repeats（CRISPR） and associated proteins （Cas） comprise the CRISPR/Cas system,which confers adaptive immunity against foreign invading pha- ges,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 lI CRISPR/Cas9 system is the simplest one,and is well-charactered in re- sent study. CRISPR/Cas9 system modified by genetic engineering has proven to be an efficient gene-targe- ting tool in various organisms. Here, the auther review the discovery, components, molecular mechanism, applications,challenges and future prospect of CRISPR/Cas9-mediated genome editing.