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Updated: Sep 14, 2025

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为什么对血友病的基因治疗的接受率低于预期?

Glenn F Pierce1, Mark Skinner1,2,3, Brian O'Mahony1,4

  • 1World Federation of Hemophilia, Montreal, Quebec, Canada.

Research and practice in thrombosis and haemostasis
|July 25, 2025
PubMed
概括

血友病的基因疗法显示出希望,在因子IX治疗中取得了一些成功,实现了功能治愈. 然而,基因相关病毒载体的挑战和高成本阻碍了因子VIII和FIX基因疗法的广泛采用.

关键词:
在 AAV AAV AAV 中.治疗 治疗 治疗 治疗有效性 有效性 有效性.第九因子XIX因子第八个因子的因素.卫生公平性健康公平性健康不平等 在健康不平等.单源性疾病是一个单源性疾病.预防 预防 预防 预防罕见的出血障碍 罕见的出血障碍在安全方面,安全是安全的.

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