肿瘤细胞治疗研究
英文原题：Current advances of CRISPR-Cas technology in cell therapy.
CRISPR-Cas是一种多功能的基因组编辑技术，已广泛应用于基础研究和转化医学。
CRISPR-Cas是一种多功能的基因组编辑技术，已广泛应用于基础研究和转化医学。自被发现以来，这种细菌来源的核酸内切酶已被改造为一系列稳健的基因组编辑工具，用于在特定位点引入移码突变或碱基转换。自2016年首次人体试验启动以来，CRISPR-Cas已在57项细胞治疗试验中接受测试，其中38项聚焦于工程化CAR-T细胞和TCR-T细胞治疗恶性肿瘤，15项为工程化造血干细胞治疗血红蛋白病、白血病和AIDS，以及4项为工程化iPSCs治疗糖尿病和癌症。在此，我们旨在综述CRISPR技术的最新突破，并重点介绍其在细胞治疗中的应用。
CRISPR-Cas is a versatile genome editing technology that has been broadly applied in both basic research and translation medicine. Ever since its discovery, the bacterial derived endonucleases have been engineered to a collection of robust genome-editing tools for introducing frameshift mutations or base conversions at site-specific loci. Since the initiation of first-in-human trial in 2016, CRISPR-Cas has been tested in 57 cell therapy trials, 38 of which focusing on engineered CAR-T cells and TCR-T cells for cancer malignancies, 15 trials of engineered hematopoietic stem cells treating hemoglobinopathies, leukemia and AIDS, and 4 trials of engineered iPSCs for diabetes and cancer. Here, we aim to review the recent breakthroughs of CRISPR technology and highlight their applications in cell therapy.
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