Alternative AAV gene therapy for hemophilia A using expression of Bi8, a novel single-chain FVIII-mimetic antibody.

已完结 20 由 Carly Carly 发布于 2025/9/29 13:51:04

DOI:10.1182/blood.2024027709
作者：Vincent Muczynski,Olivier D Christophe,Lewis Tanner,Charlotte Vayssiere,Alice Guérin,Caterina Casari,Jenny Hazel McIntosh,Doyoung Lee,Gavin Ling,Satyen Harish Gohil,Peter J Lenting,Edward G Tuddenham
文献类型：期刊论文
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American Society of Hematology (ASH)

Abstract：The recent approval of adeno-associated virus (AAV)-based gene therapies for haemophilia A (HA) represents a major advancement in the management of this X-linked bleeding disorder, offering

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