First hemophilia B gene therapy approved: More than two decades in the making.
作者信息
Herzog Roland W, VandenDriessche Thierry, Ozelo Margareth C
机构信息
Herman B Wells Center for Pediatric Research, Indiana University, Indianapolis, IN 46202, USA.
Department of Gene Therapy & Regenerative Medicine, Vrije Universiteit Brussel (VUB), Brussels, Belgium; Department of Cardiovascular Sciences, Center for Molecular & Vascular Biology, University of Leuven, Leuven, Belgium.
出版信息
Mol Ther. 2023 Jan 4;31(1):1-2. doi: 10.1016/j.ymthe.2022.12.001. Epub 2022 Dec 16.
Abstract
摘要
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First conditional marketing authorization approval in the European Union for hemophilia "A" gene therapy.血友病“A”基因疗法首次获得欧盟有条件上市许可批准。
Mol Ther. 2022 Nov 2;30(11):3335-3336. doi: 10.1016/j.ymthe.2022.09.020. Epub 2022 Oct 19.
2
Adeno-Associated Virus Gene Therapy for Hemophilia.腺相关病毒基因治疗血友病。
Annu Rev Med. 2023 Jan 27;74:231-247. doi: 10.1146/annurev-med-043021-033013. Epub 2022 Sep 14.
3
Worldwide use of factor IX Padua for hemophilia B gene therapy.全球范围内使用帕多瓦因子IX进行B型血友病基因治疗。
Mol Ther. 2022 Jul 6;30(7):2394-2396. doi: 10.1016/j.ymthe.2022.06.002. Epub 2022 Jun 14.
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Adeno-associated viral vectors for the treatment of hemophilia.用于治疗血友病的腺相关病毒载体。
Hum Mol Genet. 2016 Apr 15;25(R1):R36-41. doi: 10.1093/hmg/ddv475. Epub 2015 Nov 27.
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X-linked thrombophilia with a mutant factor IX (factor IX Padua).伴有突变型凝血因子IX（帕多瓦凝血因子IX）的X连锁血栓形成倾向
N Engl J Med. 2009 Oct 22;361(17):1671-5. doi: 10.1056/NEJMoa0904377.
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