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Updated: Sep 10, 2025

A Precision Medicine Tool for Measurement and Monitoring of Hemoglobin S in Sickle Cell Disease Patients Receiving Transfusion Therapy
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对HbSC疾病和其他复合异合体状血红蛋白病的基因疗法:纳入的时间

Andrew Wilks1, Martin H Steinberg2, Haydar Frangoul3

  • 1Boston University Chobanian & Avedisian School of Medicine, Boston, Massachusetts, United States.

Blood
|August 20, 2025
PubMed
概括

基因疗法为状细胞疾病 (SCD) 提供了潜在的治疗方法. 本综述探讨了它在常见的SCD基因型HbSC疾病中的应用,并讨论了胎儿血红蛋白如何减轻并发症.

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