逆转录病毒整合与人类基因治疗。
Retroviral integration and human gene therapy.
作者信息
Bushman Frederic D
机构信息
Department of Microbiology, University of Pennsylvania School of Medicine, Philadelphia, Pennsylvania 19104-6076, USA.
出版信息
J Clin Invest. 2007 Aug;117(8):2083-6. doi: 10.1172/JCI32949.
Abstract
Long-term correction of genetic diseases requires permanent integration of therapeutic genes into chromosomes of affected cells. Retroviral vectors are the most widely used delivery vehicles because of their efficiency and precision of integration. However, retroviral integration can take place at a variety of chromosomal sites, and examples have been reported of integration of therapeutic vectors activating oncogenes and causing cancer in patients. This issue of the JCI presents three articles that update successful human gene therapy trials and furthermore evaluate the sites of integration in cells from treated patients, including samples from individuals experiencing serious adverse events following therapy (see the related articles beginning on pages 2225, 2233, and 2241).
摘要
遗传性疾病的长期矫正需要将治疗性基因永久整合到受影响细胞的染色体中。逆转录病毒载体因其整合效率和精准性，是目前应用最为广泛的递送工具。然而，逆转录病毒可整合到染色体的多个位点，已有报道称治疗性载体的整合激活了癌基因并导致患者罹患癌症。本期《临床研究杂志》发表了三篇文章，介绍了成功的人类基因治疗试验，并进一步评估了接受治疗患者细胞中的整合位点，包括来自治疗后出现严重不良事件个体的样本（见第2225页、2233页和2241页开始的相关文章）。
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