CRISPR-Cas基因编辑技术的研究进展

Research Advances on CRISPR-Cas Genome Editing Technology

对于研究基因功能和靶向修饰,基因靶向编辑技术已经成为最重要的基因工具。成簇规律间隔短回文重复序列-成簇规律间隔短回文重复序列关联蛋白(clustered regularly interspaced short palindromic repeats-CRSPR-associated proteins, CRISPR-Cas)系统是继锌指核酸内切酶(zinc finger nucleases, ZFNs)和转录激活因子样效应物核酸酶(transcription activator-like effector nucleases,TALENs)的第三代基因定点编辑技术。CRISPR-Cas系统广泛存在于古生菌及细菌中,是机体长期进化形成的以RNA引导的降解入侵病毒或噬菌体DNA的适应性免疫系统。通过对原核生物CRISPR-Cas系统进行改造,形成了以RNA引导的对靶细胞中特定的基因序列进行定点修饰的新一代基因编辑技术。本文就该系统的发现、分型、结构、作用机制和应用等展开讨论。

For the study of gene function and targeted modifications, gene-targeted editing technology has become the most important genetic tool. The clustered regularly interspaced short palindromic repeats-CRSPR-associated proteins（CRISPR-Cas）system is a third-generation genome site-specific editing technology for zinc finger nucleases（ZFNs） and transcription activator-like effector nucleases（TALENs）. The CRISPR-Cas system is widely found in the archaea and bacteria, it is a long-term evolution of body＇s adaptive immune system by RNA-directed degradation of invasive virus or phage DNA. By modifying the prokaryotic CRISPR-Cas system, a new generation of gene editing technology that is guided by RNA and target for specific gene sequences in target cells was formed. Here, we discusse the discovery, classification, structure, mechanism and application of the system.