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-Reference-Cited by-同舟云学术

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

Published:2022-07 Issue:7 Volume:9 Page:e504-e513
ISSN:2352-3026
Container-title:The Lancet Haematology
language:en
Short-container-title:The Lancet Haematology

Author:

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

Publisher

Elsevier BV

Subject

Hematology

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3. Long-term safety and efficacy of factor IX gene therapy in hemophilia B;Nathwani;N Engl J Med,2014

4. Hemophilia B gene therapy with a high-specific-activity factor IX variant;George;N Engl J Med,2017

5. Gene therapy with adeno-associated virus vector 5-human factor IX in adults with hemophilia B;Miesbach;Blood,2018

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