免疫疾病的基因治疗：好坏参半的消息。

Gene therapy for immune disorders: good news tempered by bad news.

作者信息

Puck Jennifer M, Malech Harry L

机构信息

Genetics and Molecular Biology Branch, National Human Genome Research Institute, National Institutes of Health, Bethesda, MD 20892, USA.

出版信息

J Allergy Clin Immunol. 2006 Apr;117(4):865-9. doi: 10.1016/j.jaci.2006.01.041.

DOI:10.1016/j.jaci.2006.01.041

PMID:16630946

Abstract

After a dozen years of human gene therapy trials characterized by minimal gene correction and disappointing clinical impact, the field of gene therapy received some good news in 2000. Infants with X-linked severe combined immunodeficiency who received retroviral gene addition to cells from their bone marrow developed impressive immune reconstitution. During the following 2 years, additional patients were treated and the news was even better-babies receiving gene therapy had sustained T-cell production and in several cases developed better cell function than most patients treated with standard bone marrow transplants. Unfortunately, bad news followed. Three of the patients experienced leukemic T-cell expansions, found to be associated with retroviral insertions into genomic DNA. Where does the field stand today?

摘要

在经历了十几年以基因校正极少、临床效果令人失望为特征的人类基因治疗试验后，基因治疗领域在2000年迎来了一些好消息。患有X连锁严重联合免疫缺陷的婴儿接受了来自其骨髓细胞的逆转录病毒基因添加，免疫重建效果显著。在接下来的两年里，更多患者接受了治疗，结果甚至更好——接受基因治疗的婴儿T细胞持续产生，在一些病例中，其细胞功能比大多数接受标准骨髓移植治疗的患者更好。不幸的是，随后传来了坏消息。三名患者出现白血病性T细胞扩增，发现这与逆转录病毒插入基因组DNA有关。该领域如今处于什么状况呢？