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临床观点:通过基因治疗方法推进血友病治疗

Courtney D Thornburg1, Steve W Pipe2, Alessio Cantore3

  • 1National Institutes of Health, National Heart, Lung, and Blood Institute, Division of Blood Diseases and Resources, Bethesda, MD, USA.

Molecular therapy : the journal of the American Society of Gene Therapy
|April 23, 2025
PubMed
概括

基因疗法为血友病 (一种先天性出血障碍) 提供了新的希望,已获得批准的治疗方法和基因编辑方面的有希望的进展. 这些创新方法旨在提供更持久,更有效的血友病护理,改善患者的生活质量.

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