医学。基因治疗——前方新挑战。

Medicine. Gene therapy--new challenges ahead.

作者信息

Williams David A, Baum Christopher

机构信息

Division of Experimental Hematology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH 45229, USA.

出版信息

Science. 2003 Oct 17;302(5644):400-1. doi: 10.1126/science.1091258.

DOI:10.1126/science.1091258

PMID:14563994

Abstract

The successful use of retroviral gene transfer to treat 10 patients with X-linked severe combined immunodeficiency (SCID-X1) has been welcomed as evidence of the therapeutic potential of gene therapy. However, as Williams and Baum suggest in their Perspective, the discovery that 2 of the 10 patients developed leukemia within 3 years of gene therapy (Hacein-Bey-Abina et al.) reinforces the need to develop even more specific gene therapy interventions.

摘要

逆转录病毒基因转移成功用于治疗10名X连锁重症联合免疫缺陷病（SCID-X1）患者，这一成果受到广泛欢迎，被视为基因治疗具有治疗潜力的证据。然而，正如威廉姆斯和鲍姆在他们的《观点》文章中所指出的，10名患者中有2名在基因治疗后3年内患上白血病（哈辛-贝-阿比纳等人的研究），这一发现凸显了开发更具针对性的基因治疗干预措施的必要性。