Advances and applications of genome-edited animal models for severe combined immunodeficiency  

作　　者：Xiao Zheng Chun-Hui Huang Sen Yan Ming-Deng Rong 

机构地区：[1]Stomatological Hospital,School of Stomatology,Southern Medical University,Guangzhou,Guangdong 510280,China [2]Guangdong Key Laboratory of Non-Human Primate Models,Guangdong-Hongkong-Macao Institute of CNS Regeneration,Jinan University,Guangzhou,Guangdong 510632,China [3]School of medicine,Jinan University,Guangzhou,Guangdong 510632,China [4]Department of Neurology,Guangzhou Red Cross Hospital,Faculty of Medical Science,Jinan University,Guangzhou,Guangdong 510220,China

出　　处：《Zoological Research》2025年第1期249-260,共12页动物学研究(英文)

基　　金：supported by the Postdoctoral Fellowship Program of CPSF (GZC20231064);China Postdoctoral Science Foundation (2024M761345);Guangzhou Basic and Applied Basic Research Foundation (2024A04J6615);Scientific Research Project of Southern Medical University Stomatological Hospital (PY2023004);National Key Research and Development Program of China (2021YFA0805300);National Natural Science Foundation of China (82171244,32470564)。

摘　　要：Severe combined immunodeficiency disease(SCID),characterized by profound immune system dysfunction,can lead to life-threatening infections and death.Animal models play a pivotal role in elucidating biological processes and advancing therapeutic strategies.Recent advances in gene-editing technologies,including zincfinger nucleases(ZFNs),transcription activator-like effector nucleases(TALENs),CRISPR/Cas9,and base editing,have significantly enhanced the generation of SCID models.These models have not only deepened our understanding of disease pathophysiology but have also driven progress in cancer therapy,stem cell transplantation,organ transplantation,and infectious diseasemanagement.Thisreviewprovidesa comprehensive overview of current SCID models generated using novel gene-editing approaches,highlighting their potential applications in translational medicine and their role in advancing biomedical research.

关 键 词：Gene-editing technology Animal model Translational biomedicine Severe combined immunodeficiency disease 

分 类 号：R-332[医药卫生] R593.2