CRISPR/Cas9:基因编辑的新时代

CRISPR/Cas9:a New Era of Gene Editing

基因编辑技术是通过核酸内切酶对基因组DNA进行定向改造的技术,可以实现对特定DNA碱基的缺失、替换等,常用的四种基因编辑工具分别是:巨型核酸酶、锌指核酸酶、转录激活因子样效应物核酸酶以及CRISPR/Cas9系统。其中CRISPR/Cas9系统作为一种新型的基因组编辑技术具有组成简单、特异性好、切割效率高的优点。该文对CRISPR/Cas9系统的结构组成和功能机制,动植物基因靶向编辑和人类在遗传性疾病、病毒感染性疾病以及肿瘤方面进行综述,旨在对CRISPR/Cas9系统的现状和发展进行总结和展望。

Gene editing techniques use endonucleases to make desired changes within genomic DNA by insertion,deletion or substitution of DNA sequences.Four types of endonucleases are commonly used as gene editing tools:meganucleases,zinc finger nucleases,transcription activator-like effector nucleases,and CRISPR/Cas9 systems.Among these tools,the CRISPR/Cas9 system is a new gene editing technology remarked for simplicity,good targeting specificity and high cutting efficiency.This article reviews structural and functional characteristics of CRISPR/cas9 systems,as well as their current or potential applications in editing of animal and plant genes and treatment of human genetic diseases,viral diseases,and tumors.Current progress in research and applications of CRISPR/Cas9 systems is summarized and future perspectives are discussed.