Villanova University, Department of Chemical & Biological Engineering, Villanova, Pennsylvania, USA.
Biotechnol Prog. 2021 Jan;37(1):e3066. doi: 10.1002/btpr.3066. Epub 2020 Sep 1.
CAR-T therapy is a particularly effective treatment for some types of cancer that uses retroviruses to deliver the gene for a chimeric antigen receptor (CAR) to a patient's T cells ex vivo. The CAR enables the T cells to bind and eradicate cells with a specific surface marker (e.g., CD19 B cells) after they are transfused back into the patient. This treatment was proven to be particularly effective in treating non-Hodgkin's lymphoma (NHL) and acute lymphoblastic leukemia (ALL), but the current CAR-T cell manufacturing process has a few significant drawbacks. For example, while lentiviral and gammaretroviral transduction are both relatively effective, the process of producing viral vectors is time-consuming and costly. Additionally, patients must undergo follow up appointments for several years to monitor them for any unanticipated side effects associated with the virus. Therefore, several studies have endeavored to find alternative non-viral gene delivery methods that are less expensive, more precise, simple, and safe. This review focuses on the current state of the most promising non-viral gene delivery techniques, including electroporation and transfection with cationic polymers or lipids.
嵌合抗原受体 (CAR) -T 细胞疗法是一种特别有效的癌症治疗方法，它使用逆转录病毒将嵌合抗原受体 (CAR) 的基因传递到患者的 T 细胞中。CAR 使 T 细胞能够在回输到患者体内后结合并清除具有特定表面标志物的细胞（例如 CD19 B 细胞）。这种治疗方法已被证明对治疗非霍奇金淋巴瘤 (NHL) 和急性淋巴细胞白血病 (ALL) 特别有效，但目前的 CAR-T 细胞制造工艺存在一些明显的缺点。例如，虽然慢病毒和γ逆转录病毒转导都相对有效，但生产病毒载体的过程既耗时又昂贵。此外，患者必须接受数年的后续预约，以监测与病毒相关的任何意外副作用。因此，一些研究致力于寻找更廉价、更精确、更简单和更安全的替代非病毒基因传递方法。这篇综述重点介绍了最有前途的非病毒基因传递技术的现状，包括电穿孔和用阳离子聚合物或脂质转染。
