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对于状细胞疾病的基因治疗

Alexis Leonard1, John F Tisdale2

  • 1St. Jude Children's Research Hospital, Memphis, TN.

Hematology. American Society of Hematology. Education Program
|December 9, 2023
PubMed
概括

基因疗法通过使用自身造血干细胞移植 (HSCT) 提供了状细胞疾病 (SCD) 的潜在治疗方法. 目前的基因添加和编辑策略在SCD治疗中显示出有希望的结果.

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