Modified lentiviral globin gene therapy for pediatric β0/β0 transfusion-dependent β-thalassemia: A single-center, single-arm pilot trial
Shiqi LiSikai LingKey Laboratory of Systems Biomedicine (Ministry of Education), Shanghai Center for Systems Biomedicine, Shanghai Jiao Tong University, Shanghai 200240, China; BDgene Therapeutics, Shanghai 200240, ChinaDawei WangShanghai Institute of Hematology, State Key Laboratory of Medical Genomics, National Research Center for Translational Medicine at Shanghai, Ruijin Hospital Affiliated to Shanghai Jiao Tong University School of Medicine, Shanghai 200025, ChinaXiaoyuan WangBDgene Therapeutics, Shanghai 200240, ChinaFangyuan HaoBDgene Therapeutics, Shanghai 200240, ChinaLiufan YinZhongtao YuanLin LiuLin Zhang920th Hospital of Joint Logistics Support Force of People's Liberation Army of China, Kunming, Yunnan 650100, ChinaYu LiYingnian ChenLe LuoYing DaiLihua Zhang920th Hospital of Joint Logistics Support Force of People's Liberation Army of China, Kunming, Yunnan 650100, ChinaLvzhe ChenDongjie DengWei TangShanghai Institute of Hematology, State Key Laboratory of Medical Genomics, National Research Center for Translational Medicine at Shanghai, Ruijin Hospital Affiliated to Shanghai Jiao Tong University School of Medicine, Shanghai 200025, ChinaSujiang ZhangShanghai Institute of Hematology, State Key Laboratory of Medical Genomics, National Research Center for Translational Medicine at Shanghai, Ruijin Hospital Affiliated to Shanghai Jiao Tong University School of Medicine, Shanghai 200025, ChinaSanbin Wang920th Hospital of Joint Logistics Support Force of People's Liberation Army of China, Kunming, Yunnan 650100, China. Electronic address: [email protected]Yujia CaiKey Laboratory of Systems Biomedicine (Ministry of Education), Shanghai Center for Systems Biomedicine, Shanghai Jiao Tong University, Shanghai 200240, China. Electronic address: [email protected]
2024en
ABI
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