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X连锁重症联合免疫缺陷病基因治疗后白血病的发生

Occurrence of leukaemia following gene therapy of X-linked SCID.

作者信息

Kohn Donald B, Sadelain Michel, Glorioso Joseph C

机构信息

Division of Research Immunology/BMT, Childrens Hospital Los Angeles, USC Keck School of Medicine, 4650 Sunset Boulevard, Los Angeles, California 90027, USA.

出版信息

Nat Rev Cancer. 2003 Jul;3(7):477-88. doi: 10.1038/nrc1122.

Abstract

Recombinant viral vectors have allowed gene transfer to be developed as a promising approach to the treatment of genetic diseases. Recently, gene therapy of children with X-linked severe combined immune deficiency resulted in impressive levels of immune reconstitution--a triumph that was later overshadowed by the development of leukaemia in two patients. What were the causes of this cancer, and how can the therapeutic benefits of gene therapy be achieved while minimizing risk to the patient?

摘要

重组病毒载体使基因转移得以发展成为一种治疗遗传疾病的有前景的方法。最近,对患有X连锁严重联合免疫缺陷的儿童进行基因治疗,使免疫重建达到了令人瞩目的水平——这一胜利后来因两名患者患白血病而蒙上阴影。这种癌症的病因是什么,如何在将对患者的风险降至最低的同时实现基因治疗的益处?

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