Safety and activity of an engineered, liver-tropic adeno-associated virus vector expressing a hyperactive Padua factor IX administered with prophylactic glucocorticoids in patients with haemophilia B: a single-centre, single-arm, phase 1, pilot trial

Authors: Feng Xue, Huiyuan Li, Xia Wu, Wei Liu, Feixu Zhang, Dingyue Tang, Yunfei Chen, Wentian Wang, Ying Chi, Jing Zheng, Zengmin Du, Wei Jiang, Chen Zhong, Jun Wei, Ping Zhu, Rongfeng Fu, Xiaofan Liu, Lingling Chen, Xiaolei Pei, Junjiang Sun, Tao Cheng, Renchi Yang, Xiao Xiao, Lei Zhang

Published: 2022-05-19

DOI: 10.1016/s2352-3026(22)00113-2

Source: Full article

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Abstract

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