Precise genome editing with base editors  

作　　者：Hongcai Liu Yao Zhu Minjie Li Zhimin Gu 

机构地区：[1]Institute of Systems Medicine,Chinese Academy of Medical Sciences&Peking Union Medical College,Beijing,China [2]Suzhou Institute of Systems Medicine,Suzhou,Jiangsu Province,China [3]Institute of Systems Medicine,Chinese Academy of Medical Sciences&Peking Union Medical College,Beijing 100005,China [4]Suzhou Institute of Systems Medicine,Suzhou 215123,Jiangsu Province,China

出　　处：《Medical Review》2023年第1期75-84,共10页医学评论（英文）

基　　金：supported by CAMS Innovation Fund for Medical Sciences(CIFMS,2022-I2M-1-024,2022-RC180-02 to Z.G.).

摘　　要：Single-nucleotide variants account for about half of known pathogenic genetic variants in human.Genome editing strategies by reversing pathogenic point mutations with minimum side effects have great therapeutic potential and are now being actively pursued.The emerge of precise and effcient genome editing strategies such as base editing and prime editing provide powerful tools for nucleotide conversion without inducing double-stranded DNA breaks(DSBs),which have shown great potential for curing genetic disorders.A diverse toolkit of base editors has been devel-oped to improve the editing effciency and accuracy in different context of application.Here,we summarized the evolving of base editors(BEs),their limitations and future perspective of base editing-based therapeutic strategies.

关 键 词：adenine base editor base editing clustered regularly interspaced short palindromic repeats(CRISPR)/CRISPR-associated protein cytosine base editor 

分 类 号：H31[语言文字—英语]