Generation of inactivated IL2RG and RAG1 monkeys with severe combined immunodeficiency using base editing  

作　　者：Xiao Zheng Chunhui Huang Yingqi Lin Bofeng Han Yizhi Chen Caijuan Li Jiawei Li Yongyan Ding Xichen Song Wei Wang Weien Liang Jianhao Wu Jiaxi Wu Jiale Gao Chengxi Wei Xudong Zhang Zhuchi Tu Sen Yan 

机构地区：[1]Guangdong Key Laboratory of Non-human Primate Research,Guangdong-Hongkong-Macao Institute of CNS Regeneration,Jinan University,510632 Guangzhou,China [2]Department of Pathophysiology,School of Medicine,Jinan University,510632 Guangzhou,China

出　　处：《Signal Transduction and Targeted Therapy》2023年第10期4825-4837,共13页信号转导与靶向治疗（英文）

基　　金：This work was supported by the National Key Research and Development Program of China(2021YFA0805300,2021YFA0805204);the National Natural Science Foundation of China(82171244,81922026,32170981);Guangzhou Key Research Program on Brain Science(202007030008).

摘　　要：Severe combined immunodeficiency(SCiD)encompasses a range of inherited disorders that lead to a profound deterioration of the immune system.Among the pivotal genes associated with SCID,RAG1 and IL2RG play crucial roles.IL2RG is essential for the development,differentiation,and functioning of T,B,and NK cells,while RAG1 critically contributes to adaptive immunity by facilitating V(D)J recombination during the maturation of lymphocytes.Animal models carrying mutations in these genes exhibit notable deficiencies in their immune systems.

关 键 词：IMMUNODEFICIENCY IMMUNITY RAG1 

分 类 号：R394[医药卫生—医学遗传学]