Base editing: a novel cure for severe combined immunodeficiency  

作　　者：Teng-Cheong Ha Michael Morgan Axel Schambach 

机构地区：[1]Institute of Experimental Hematology,Hannover Medical School,Hannover,30625,Germany [2]REBIRTH Research Center for Translational Regenerative Medicine,Hannover Medical School,Hannover,30625,Germany [3]Division of Hematology/Oncology,Boston Children’s Hospital,Harvard Medical School,Boston,MA,02115,USA

出　　处：《Signal Transduction and Targeted Therapy》2023年第10期4433-4435,共3页信号转导与靶向治疗（英文）

摘　　要：In a recent Cell article,McAuley,Kohn et al.showcased proof-of-principle of a promising gene therapy(GT)approach to correct the underlying genetic defect responsible for CD3δ-deficient severe combined immunodeficiency(SCID)using a CRISPR-Cas9-derived adenine-base-editor(ABE).1 This ground-breaking and important study could impact future GTs for monogenic diseases by providing novel tailored therapeutic options(Fig.1).

关 键 词：IMMUNODEFICIENCY al. DISEASES 

分 类 号：R730.51[医药卫生—肿瘤]