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血友病B的AAV介导基因疗法:从早期尝试到现代试验

Vincent Muczynski1, Amit C Nathwani2

  • 1Department of Haematology, University College London - Cancer Institute, United Kingdom of Great Britain and Northern Ireland.

Thrombosis research
|February 21, 2024
PubMed
概括

使用腺相关病毒 (AAVs) 的基因疗法在B型血友病患者中显示出稳定,长期表达凝血因子IX (FIX),减少出血. 目前正在进行的研究解决了诸如先前存在的免疫力等挑战,以向功能性治疗迈进.

关键词:
腺相关病毒的病毒.临床试验临床试验是指临床试验的临床试验.基因治疗是一种基因疗法.血友病B型 血友病B型 血友病B型

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