清脆的
嵌合抗原受体
Cas9
向性
基因组编辑
细胞外小泡
遗传增强
生物
电穿孔
计算生物学
癌症研究
病毒学
基因传递
癌症
免疫疗法
基因
细胞生物学
遗传学
病毒
作者
Qian Xu,Zheng Zhang,Lei Zhao,Yun Qin,Haodong Cai,Zhe Geng,Xiaojian Zhu,Wei Zhang,Yuanyuan Zhang,Jiaqi Tan,Jue Wang,Jianfeng Zhou
标识
DOI:10.1016/j.jconrel.2020.07.033
摘要
The CRISPR/Cas9 system is an efficient genome-editing system that has been successfully applied in the field of gene therapy. However, clinical applications of the CRISPR/Cas9 system are limited by the delivery method and safety concerns. Extracellular Vesicles (EVs) can be released from almost every type of cell, and they act as shuttles to convey molecules between cells. Here, we used EVs derived from epithelial cells as a biosafety delivery platform for the CRISPR/Cas9 system and modified the EVs with a chimeric-antigen receptor (CAR) to give them selective tropism to tumors. Compared to normal EVs, CAR-EVs accumulated in cancer tumors rapidly and released the CRISPR/Cas9 system targeting the MYC oncogene efficiently, both in vitro and in vivo. Taken together, the combination of EV and CAR was confirmed to be a novel strategy facilitating the use of natural gene therapy platforms in cancer treatment in this proof-of-concept research.
科研通智能强力驱动
Strongly Powered by AbleSci AI