The Interface of Gene Editing with Regenerative Medicine  

作　　者：Veronica E.Farag Elsie A.Devey Kam W.Leong 

机构地区：[1]Department of Biomedical Engineering,Columbia University,New York City,NY 10027,USA [2]Department of Systems Biology,Columbia University,New York City,NY 10032,USA

出　　处：《Engineering》2025年第3期73-100,共28页工程(英文)

基　　金：supported by the National Institutes of Health(UH3NS115598).

摘　　要：The potential of regenerative medicine in the clinical space is vast,given its ability to repair and replace damaged tissues,restore lost functions due to age or disease,and transform personalized therapy.Traditional regenerative medicine and tissue engineering strategies have created specialized tissues using progenitor cells and various biological stimuli.To date,there are many US Food and Drug Administration(FDA)-approved regenerative medicine therapies,such as those for wound healing and orthopedic injuries.Nonetheless,these therapies face challenges,including off-target effects,a lack of precision,and failure to target the disease or injury at its origin.In search of novel,precise,and efficient alternatives,the regenerative medicine landscape is shifting towards genome engineering technologies,particularly gene editing.Clustered regularly interspaced short palindromic repeats(CRISPR)-based gene editing systems enable precise knock-ins,knockouts,transcriptional activation and repression,as well as specific base conversions.This advancement has allowed researchers to treat genetic and degenerative diseases,control cell fate for highly regulated tissue repair,and enhance tissue functions.In this review,we explore the progress and future prospects of CRISPR technologies in regenerative medicine,focusing on how gene editing has led to advanced therapeutic applications and served as a versatile research tool for understanding tissue development and disease progression.

关 键 词：CRISPR/Cas9 Tissue engineering Regenerative medicine Gene editing Stem cell transplantation Disease models 

分 类 号：R318[医药卫生—生物医学工程]