Surgery Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD 20892, USA.
Gene Ther. 2009 Aug;16(8):1042-9. doi: 10.1038/gt.2009.54. Epub 2009 Jun 4.
Genetically engineered lymphocytes hold promise for the treatment of genetic disease, viral infections and cancer. However, current methods for genetic transduction of peripheral blood lymphocytes rely on viral vectors, which are hindered by production and safety-related problems. In this study, we demonstrated an efficient novel nonviral platform for gene transfer to lymphocytes. The Sleeping Beauty transposon-mediated approach allowed for long-term stable expression of transgenes at approximately 50% efficiency. Utilizing transposon constructs expressing tumor antigen-specific T-cell receptor genes targeting p53 and MART-1, we demonstrated sustained expression and functional reactivity of transposon-engineered lymphocytes on encountering target antigen presented on tumor cells. We found that transposon- and retroviral-modified lymphocytes had comparable transgene expression and phenotypic function. These results demonstrate the promise of nonviral ex vivo genetic modification of autologous lymphocytes for the treatment of cancer and immunologic disease.
基因工程淋巴细胞有望用于治疗遗传疾病、病毒感染和癌症。然而,目前外周血淋巴细胞的基因转导方法依赖于病毒载体,这受到生产和安全相关问题的限制。在这项研究中,我们展示了一种高效的新型非病毒基因转移平台用于淋巴细胞。睡眠美人转座子介导的方法允许大约 50%的效率进行长期稳定的转基因表达。利用表达针对 p53 和 MART-1 的肿瘤抗原特异性 T 细胞受体基因的转座子构建体,我们证明了转座子工程化淋巴细胞在遇到肿瘤细胞上呈现的靶抗原时的持续表达和功能反应性。我们发现转座子和逆转录病毒修饰的淋巴细胞具有可比的转基因表达和表型功能。这些结果表明,非病毒体外遗传修饰自体淋巴细胞用于治疗癌症和免疫性疾病具有广阔的前景。