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
Xue, F; Li, HY; Wu, X; Liu, W; Zhang, FX; Tang, DY; Chen, YF; Wang, WT; Chi, Y; Zheng, J; Du, ZM; Jiang, W; Zhong, C; Wei, J; Zhu, P; Fu, RF; Liu, XF; Chen, LL; Pei, XL; Sun, JJ; Cheng, T; Yang, RC; Xiao, X; Zhang, L
Xiao, X (通讯作者)，East China Univ Sci & Technol, Shanghai 200237, Peoples R China.;Zhang, L (通讯作者)，Chinese Acad Med Sci & Peking Union Med Coll, State Key Inst Hematol & Blood Dis Hosp, Tianjin 300020, Peoples R China.;Yang, RC (通讯作者)，Chinese Acad Med Sci & Peking Union Med Coll, Inst Hematol, Tianjin 300020, Peoples R China.;Yang, RC (通讯作者)，Chinese Acad Med Sci & Peking Union Med Coll, Blood Dis Hosp, Tianjin 300020, Peoples R China.
LANCET HAEMATOLOGY, 2022; 9 (7): E504
