Engineering CRISPR for emerging therapeutic applications  

作　　者：Xiaoqi Sun Zhuang Liu 

机构地区：[1]Department of Pharmaceutical Sciences,University of Michigan,Ann Arbor,MI 48109,USA [2]Institute of Functional Nano&Soft Materials(FUNSOM),Jiangsu Key Laboratory for Carbon-Based Functional Materials&Devices,Soochow University,Suzhou 215123,China

出　　处：《EngMedicine》2024年第3期10-20,共11页医工交叉（英文）

基　　金：supported by the New Cornerstone Science Foundation through the New Cornerstone Investigator Program and the XPLORER PRIZE,the National Research Program of China(2021YFF0701800 and 2020YFA0211100);the National Natural Science Foundation of China(T2321005 and 52032008);the Collaborative Innovation Center of Suzhou Nano Science and Technology,and the 111 Program of the Ministry of Education of China.

摘　　要：Clustered regularly interspaced short palindromic repeats(CRISPRs)were originally identified in bacterial and archaeal genomes as an adaptive immune response against invading viruses and phages.The discovery of the ability of CRISPR–Cas9 to cut targeted DNA sequences at predetermined sites has introduced a new era of precision and flexibility in genome editing.Building on this classic CRISPR–Cas system,the discovery of other diverse CRISPR systems and their further engineering have produced various powerful tools for gene editing and modulation at the genomic,chromatin,and RNA levels.This review summarizes the fundamental knowledge regarding CRISPR–Cas systems and explores the engineering strategies of these versatile systems for novel therapeutic applications.We discuss the evolution from classic CRISPR editing(CRISPR 1.0)to advanced methodologies such as base editing,prime editing,CRISPR-directed integrases,epigenetic editing,and RNA editing.This review focuses on the related principles,therapeutic applications,challenges,and future outlooks.

关 键 词：CRISPR Base editing Prime editing CRISPR-directed integrase Epigenetic editing RNA editing 

分 类 号：Q78[生物学—分子生物学]