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新出现的数据影响了使用复合腺相关病毒进行血友病基因治疗的风险/益处评估

John Puetz1

  • 1Department of Pediatrics, Division of Pediatric Hematology/Oncology, Saint Louis University School of Medicine, St. Louis, MO, United States.

Frontiers in medicine
|November 29, 2023
PubMed
概括

血友病 (一种出血障碍) 的基因疗法现在已获得批准,但可能并不像最初预期的那样有效或安全. 由于新的治疗选择和新出现的数据,需要对其风险与益处进行重新评估.

关键词:
腺相关病毒的病毒.第九因子XIX因子第八个因子的因素.基因治疗的基因疗法血友病 (hemophilia) 是一种血友病.肝细胞癌是肝细胞癌.血栓性微血管病变 - - 血栓性微血管病变

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