Morgan Richard A, Boyerinas Benjamin
Bluebird bio, 150 Second Street, Cambridge, MA 02141, USA.
Biomedicines. 2016 Apr 20;4(2):9. doi: 10.3390/biomedicines4020009.
Gene transfer technology and its application to human gene therapy greatly expanded in the last decade. One area of investigation that appears particularly promising is the transfer of new genetic material into T cells for the potential treatment of cancer. Herein, we describe several core technologies that now yield high-efficiency gene transfer into primary human T cells. These gene transfer techniques include viral-based gene transfer methods based on modified and non-viral methods such as DNA-based transposons and direct transfer of mRNA by electroporation. Where specific examples are cited, we emphasize the transfer of chimeric antigen receptors (CARs) to T cells, which permits engineered T cells to recognize potential tumor antigens.
在过去十年中,基因转移技术及其在人类基因治疗中的应用得到了极大的扩展。一个特别有前景的研究领域是将新的遗传物质导入T细胞以用于癌症的潜在治疗。在此,我们描述了几种目前能高效地将基因导入原代人T细胞的核心技术。这些基因转移技术包括基于病毒的基因转移方法(基于修饰的病毒)以及非病毒方法,如基于DNA的转座子和通过电穿孔直接转移mRNA。在引用具体例子时,我们强调了嵌合抗原受体(CAR)向T细胞的转移,这使得工程化T细胞能够识别潜在的肿瘤抗原。