CAR-T(CAR-T)细胞疗法在非肿瘤性疾病中的应用
Chimeric antigen receptor T (CAR-T) cell therapy in non-oncological diseases.
CAR-T(CAR-T)细胞在血液系统恶性肿瘤中的应用推动了这种免疫治疗形式的显著进展。
CELL INTELLIGENCE · 肿瘤细胞治疗研究
肿瘤细胞治疗研究
英文原题:Advancing CART therapy for acute myeloid leukemia: recent breakthroughs and strategies for future development.
Advancing CART therapy for acute myeloid leukemia: recent breakthroughs and strategies for future development.
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基于其他血液系统恶性肿瘤的治疗结果,以及复发/难治性急性髓系白血病(AML)对新疗法的需求,研究者正在开发用于AML的嵌合抗原受体(CAR)T疗法。CAR-T 治疗AML面临的最大挑战是找到特异性靶抗原,因为AML细胞表达的抗原通常也存在于健康造血干细胞(HSC)上。肿瘤和HSC同时表达靶抗原可能导致靶向肿瘤外毒性。本综述指导研究人员设计、开发并推动CAR-T 疗法进入临床,用于治疗AML。具体而言,我们介绍设计此类疗法时需考虑的问题、可用于证明疗效和安全性的体内外检测方法,以及医院治疗和管理患者所需的专业知识与设施。
Chimeric antigen receptor (CAR) T therapies are being developed for acute myeloid leukemia (AML) on the basis of the results obtained for other haematological malignancies and the need of new treatments for relapsed and refractory AML. The biggest challenge of CART therapy for AML is to identify a specific target antigen, since antigens expressed in AML cells are usually shared with healthy haematopoietic stem cells (HSC).
The concomitant expression of the target antigen on both tumour and HSC may lead to on-target/off-tumour toxicity. In this review, we guide researchers to design, develop, and translate to the clinic CART therapies for the treatment of AML. Specifically, we describe what issues have to be considered to design these therapies; what in vitro and in vivo assays can be used to prove their efficacy and safety; and what expertise and facilities are needed to treat and manage patients at the hospital.
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