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Mankai Ju

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Articles

Factor IX Padua AAV gene therapy in adolescents with hemophilia B: a phase 1 trial

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By Feng Xue, Ai Zhang, Mankai Ju, Xia Wu, Bingqi Xu, Xiaolei Pei, Yuhua Wang, Wei Liu, Aiguo Liu, Wei Jiang, Xinyue Dai, Yunfei Chen, Xiaofan Liu, Rongfeng Fu, Ting Sun, wenjing gu, Jing Zheng, Zengmin Du, Caifeng Yang, Xiao Xiao, Renchi Yang, Qun Hu, Lei Zhang
| Nature Verified
Abstract Adeno-associated virus (AAV)-mediated factor IX Padua gene therapy has demonstrated good safety and efficacy in adult patients with hemophilia B, but its safety and efficacy in adolescent patients with hemophilia B remain unknown. Here we report a multicenter, single-arm, phase 1 study involving 11 adolescent participants (aged 12–18 years) with severe or moderately severe hemophilia B (factor IX coagulant activity (FIX:C) ≤2 IU dl−1) in China.
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