嵌合抗原受体T细胞技术及其临床应用

Chimeric antigen receptor-engineered T cells and its clinical application

嵌合抗原受体 T 细胞（CAR-T）疗法是利用基因改造技术，使 T 细胞表达肿瘤特异性嵌合抗原受体，以抗原依赖、非主要组织相容性复合体（MHC）限制的方式结合肿瘤抗原，肿瘤相关抗原（TAA）的单链抗体（scFv）和 T 细胞的活化序列在体外进行基因重组，形成重组质粒。在体外通过转染技术，转染经纯化与大规模扩增后的 T 细胞，启动并活化特异性杀伤肿瘤反应，其临床细胞治疗的应用显示出高效性，在白血病、淋巴瘤、黑色素瘤等恶性肿瘤治疗中均显示出良好的抗肿瘤效应，使得 CAR-T 技术成为主流细胞治疗手段。

Chimeric antigen receptor T-cell （CAR-T） technology is based on genetic modification technology to express T-cell expression tumor specific chimeric antigen receptor bind tumor antigen in an antigen-dependent anti-MCH way. Single chain antibody fragment （scFv） of tumor-associated antigen （TAA） combines with up-stream activating sequence of T-cell in vitro. The forming recombinant plasmid transfects the purified and large scale proliferating T-cell in vitro by transfection technique. This process starts and activates specific killing reaction of tumor. The clinical application of cell therapy shows high efficiency and good anti-tumor effect in treatment of malignant neoplasm, such as leukemia, lymphoma, melanoma, which made CAR-T become the mainstream method of cell therapy.