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Updated: Jun 18, 2025

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状细胞疾病的基因疗法在地平线上:临床医生的观点

Henna Butt1,2, John F Tisdale1

  • 1Cellular and Molecular Therapeutics Branch, National Heart, Lung and Blood Institute, National Institutes of Health, Bethesda, MD, USA.

Expert review of hematology
|July 30, 2024
PubMed
概括

基因疗法为状细胞病 (SCD) 提供了有前途的治疗方法,这是一种使人衰弱的遗传疾病,影响了数百万人. 技术的进步使得这些潜在的拯救生命的治疗方法对患有SCD的患者更容易获得.

关键词:
状细胞疾病是一种状细胞疾病.临床试验是指临床试验中的临床试验.基因添加 基因添加基因编辑 基因编辑基因调节 基因调节 基因调节基因治疗的基因疗法

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