Worldwide use of factor IX Padua for hemophilia B gene therapy
Author:
Publisher
Elsevier BV
Subject
Drug Discovery,Pharmacology,Genetics,Molecular Biology,Molecular Medicine
Reference13 articles.
1. 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;Lancet Haematol.,2022
2. X-linked thrombophilia with a mutant factor IX (factor IX Padua);Simioni;N. Engl. J. Med.,2009
3. Hemophilia B gene therapy with a high-specific-activity factor IX variant;George;N. Engl. J. Med.,2017
4. Etranacogene dezaparvovec (AMT-061 phase 2b): normal/near normal FIX activity and bleed cessation in hemophilia B;Von Drygalski;Blood Adv.,2019
5. WFH: closing the global gap--achieving optimal care;Skinner;Haemophilia,2012
Cited by 3 articles. 订阅此论文施引文献 订阅此论文施引文献，注册后可以免费订阅5篇论文的施引文献，订阅后可以查看论文全部施引文献
1. Production of therapeutic levels of human FIX-R338L by engineered B cells using GMP-compatible medium;Molecular Therapy - Methods & Clinical Development;2023-12
2. Humanization and functional characterization of enhanced coagulation factor IX variants identified through ancestral sequence reconstruction;Journal of Thrombosis and Haemostasis;2023-11
3. First hemophilia B gene therapy approved: More than two decades in the making;Molecular Therapy;2023-01
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