DOI: 10.1515/mr-2022-0029
Site-specific genome editing in treatment of inherited diseases:possibility,progress,and perspectives
Chao Huang1
Qing Li2
Jinsong Li3
1.Key Laboratory of Systems Health Science of Zhejiang Province,School of Life Science,Hangzhou Institute for Advanced Study,University of Chinese Academy of Sciences,No.1 Sub-Lane Xiangshan,Hangzhou 310024,Zhejiang,China2.State Key Laboratory of Cell Biology,Shanghai Key Laboratory of Molecular Andrology,CAS Center for Excellence in Molecular Cell Science,Shanghai Institute of Biochemistry and Cell Biology,Chinese Academy of Sciences,University of Chinese Academy of Sciences,No.320 Yueyang Road,Shanghai 200031,China3.Key Laboratory of Systems Health Science of Zhejiang Province,School of Life Science,Hangzhou Institute for Advanced Study,University of Chinese Academy of Sciences,Hangzhou,Zhejiang,China
摘要：Advancements in genome editing enable per-manent changes of DNA sequences in a site-specific manner,providing promising approaches for treating human genetic disorders caused by gene mutations.Recently,genome editing has been applied and achieved significant progress in treating inherited genetic disorders that remain incurable by conventional therapy.Here,we present a review of various programmable genome editing systems with their principles,advantages,and limitations.We introduce their recent applications for treating inherited diseases in the clinic,including sickle cell disease(SCD),β-thalassemia,Leber congenital amaurosis(LCA),heterozygous familial hypercholester-olemia(HeFH),etc.We also discuss the paradigm of ex vivo and in vivo editing and highlight the promise of somatic editing and the challenge of germline editing.Finally,we propose future directions in delivery,cut-ting,and repairing to improve the scope of clinical applications.
机标关键词：genomediseaseseditinginheritedperspectivespossibilityprogresssite-specific
论文发表日期：2022-10-28
在线出版日期：2025-08-15（本平台首次上网日期，不代表文献的发表时间）
页数：30( 471-500 )
英文信息