规律间隔成簇短回文重复序列(CRISPR)-CRISPR相关蛋白(CAS)系统已在基因编辑邻域掀起一波热潮,相比已研究成熟CRISPR/cas9系统,CRISPR/cas13系统作为一种新型的RNA编辑工具,开启了RNA水平研究及诊疗的新时代.CRISPR/cas13系统在CRISPR RNA(crRNA)的引导下可以特异性切割靶序列,同时能对周边RNA进行非特异性的"附带切割",基于该CRISPR/cas13系统的特性,通过优化与改造,CRISPR/cas13系统已成为分子诊断及疾病治疗的新工具.本文对CRISPR/cas13系统的特点及分子机制进行总结,并对其在分子诊断、疾病治疗等方面的研究进展进行综合论述.
The Clustered regularly interspaced short Palindromic repeats(CRISPR)-CRISPR-relat-ed protein(CAS)systems have made waves in the gene-editing neighborhood.Compared with the mature CRISPR/CAS9 system,CRISPR/CAS13 system,as a novel RNA editing tool,opens a new era of RNA level research and treatment.Under the guidance of CRISPR RNA(crRNA),the CRISPR/CAS13 system can spe-cifically cut target sequences,and at the same time can perform non-specific"incidental cutting"of peripheral RNA.Based on the characteristics of the CRISPR/CAS13 system,through optimization and modification,The CRISPR/CAS13 system has become a new tool for molecular diagnosis and disease treatment.This article sum-marizes the characteristics and molecular mechanisms of the CRISPR/cas13 system,and comprehensively dis-cusses its research progress in molecular diagnosis and disease treatment.