一个年轻的成年人与状细胞疾病后意外的复杂性:一个案例报告
Joshua Glass1, HyoJeong Han1,2,3, Jennifer Webb1,2,3
1Division of Pediatric Hematology, Center for Cancer and Blood Disorders, Children's National Hospital, Washington, District of Columbia, USA.
Journal of clinical apheresis
|July 25, 2025
概括
一个4级不良事件,一个通用的强力克隆性发作,发生在状细胞疾病 (SCD) 患者的基因治疗后自主干细胞采集. 这种以前未报告的发作事件突显了SCD基因疗法的潜在风险.
科学领域:
- 血液学 血液学 血液学
- 神经学 神经学
- 基因治疗 基因治疗
背景情况:
- 接受基因治疗的状细胞疾病 (SCD) 患者需要通过非瑞斯收集干细胞.
- 基因疗法旨在纠正SCD的遗传缺陷,但非术程序具有潜在的风险.
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