一名接受镰状细胞病基因治疗的患者出现与慢病毒载体无关的骨髓增生异常综合征。
Myelodysplastic syndrome unrelated to lentiviral vector in a patient treated with gene therapy for sickle cell disease.
作者信息
Hsieh Matthew M, Bonner Melissa, Pierciey Francis John, Uchida Naoya, Rottman James, Demopoulos Laura, Schmidt Manfred, Kanter Julie, Walters Mark C, Thompson Alexis A, Asmal Mohammed, Tisdale John F
机构信息
Cellular and Molecular Therapeutics Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD.
bluebird bio, Inc, Cambridge, MA.
出版信息
Blood Adv. 2020 May 12;4(9):2058-2063. doi: 10.1182/bloodadvances.2019001330.
Abstract
Ability to accurately attribute adverse events post–gene therapy is required to describe the benefit-risk of these novel treatments. A SCD patient developed myelodysplastic syndrome post-LentiGlobin treatment; we show how insertional oncogenesis was excluded as the cause.
摘要
准确归因基因治疗后不良事件的能力对于描述这些新型治疗的获益风险至关重要。一名镰状细胞病（SCD）患者在接受LentiGlobin治疗后发生了骨髓增生异常综合征；我们展示了如何排除插入性致癌作用作为病因。
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