嵌合抗原受体T细胞治疗恶性血液病的研究进展

Research progress of chimeric antigen receptor T cell therapy for malignant hematologic diseases

嵌合抗原受体（CAR）-T细胞治疗是一种新的免疫疗法。该方法是将识别肿瘤细胞相关抗原的受体与T细胞胞内信号域在体外进行基因重组，再将这些质粒转染至T细胞中，而这类表达CAR的T细胞可特异性识别具有靶抗原的肿瘤细胞并发挥杀伤作用。目前，CAR-T细胞在肿瘤治疗，尤其是在恶性血液病治疗上获得良好疗效，但其疗效、安全性、不良反应、质量控制方面均需进一步验证。笔者拟就CART细胞治疗中的CAR设计、细胞制备、临床试验及不良反应进行综述。

Chimeric antigen receptor （CAR） -T cell therapy is an emerging immunotherapy. Genes encoding fusion proteins of tumor cells antigen recognizing receptors and intracellular signaling domains of T cells recombined ex vivo, subsequently these plasmids were transfected into the T cells. Thus the T cells engineered to express a CAR could recognize special antigen on tumor cells and attack these cells. Currently CAR-T cell therapy showing striking effective in malignant tumor, especially in hematological malignancies, but the efficiency, safety, side effect, quality control of CAR-T cell therapy still need to be confirmed. In this review, we will introduce and discuss the CAR design, CAR-T cell manufacture, clinical trials and side effects in CAR-T cell therapy.