CRISPR-Cas9基因编辑技术在肿瘤基因治疗中的研究进展

The Research Progress of CRISPR-Cas9 Genome Editing Technology for Tumor Gene Therapy

规律成簇间隔短回文重复CRISPR(clustered regularly interspaced short palindromic repeat)/CRISPR相关酶Cas(CRISPR-associated nuclease)系统是古细菌和细菌在与噬菌体抗争的进化过程中产生的抵御外源DNA入侵的一种适应性免疫机制,能有效识别并切割外源DNA.CRISPR-Cas9系统被开发成为新一代的基因编辑技术,相较于传统的锌指核酸酶(zinc-finger nucleases,ZFN)、转录激活因子样效应因子核酸酶(transcription activator-like effector nucleases,TALEN)和胚胎干细胞(embryonic stem cell,ES)打靶技术,该技术操作简单,效率高,成本低,有着极其广阔的应用前景.目前,CRISPR-Cas9技术已经被广泛应用到恶性肿瘤的相关研究中.整理了近几年来有关CRISPR-Cas9基因编辑技术在恶性肿瘤基因治疗中的最新文献报道,并对其研究现状及进展作一综述.

The Clustered Regularly Interspaced Short Palindromic Repeat/CRISPR-associated nuclease 9(CRISPR-Cas9)system is the part of the prokaryotic immune system,which could recognize and delete the exogenous DNA sequences originated from virus or plasmid.Compared to the existed technologies such as ZEN,TALEN or ES targeting,CRISPR-Cas9 system is more efficient,economical and promising approach to manipulate the genome.In this paper,the research status about CRISPR-Cas9 technology are summarized,especially the latest applications in gene therapy studies of different tumors.