学术中心血液病学

《NEJM》基因疗法成功治疗镰状细胞贫血

通过病毒载体将目的基因转至造血干细胞,科学家首次成功治愈了一位患有镰状细胞贫血(SCD)的男孩。该疗法的效果最早报道于2015年,2016年研究人员在美国血液学会年会上报告了该患者临床症状的好转。《The New England Journal of Medicine》3月1日发表的研究对该病例进行了详细描述。患者SCD基因型为βSβS,2014年接受治疗时13岁,病程近10年...

Published 2017-3-13
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