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.

PubMed
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.

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