英国HCDO基因疗法工作组:关于将血友病基因疗法纳入成人的常规临床实践的指导

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Pratima Chowdary1,2, Beatriz Duran3, Paul Batty1,2

  • 1Katharine Dormandy Haemophilia and Thrombosis Centre, Royal Free Hospital, London, UK.

Haemophilia : the official journal of the World Federation of Hemophilia
|November 20, 2024
PubMed
概括

新的指导方针为英国的腺相关病毒载体 (AAV) 基因治疗提供了一个框架. 这确保了对血友病患者的安全和有效治疗,利用一个中心和分支模型获得最佳结果.

关键词:
英国HCDOHCDO临床实践指南 临床实践指南基因治疗的基因疗法血友症A是一种血友病.血友病B型 血友病B型 血友病B型这是一个多学科的多学科教育.患者途径 患者途径

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