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Improved extracellular vesicle-based mRNA delivery for familial hypercholesterolemia treatment

Theranostics. 2023-06; 
Zheng Yang, Panpan Ji, Zhelong Li, Rongxin Zhang, Mengying Wei, Yang Yang, Lijun Yuan, Yan Han, Guodong Yang
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Recombinant Antibody Expression … , or CD9-MCP recombinant plasmid were used, and the EVs … the CD9-MCP recombinant plasmid compared with the None or … were also synthesized by Genscript Biotech Corporation. … Get A Quote

摘要

Extracellular vesicle (EV)-based low-density lipoprotein receptor () mRNA delivery showed excellent therapeutic effects in treating familial hypercholesterolemia (FH). Nevertheless, the loading inefficiency of EV-based mRNA delivery presents a significant challenge. Recently, RNA-binding proteins (RBPs) have been fused to EV membrane proteins for selectively encapsulating targeted RNAs to promote loading efficiency. However, the strong interaction between therapeutic RNAs and RBPs prevents RNA release from endosomes to the cytosol in the recipient cells. In this study, an improved strategy was developed for efficient encapsulation of mRNA into EVs in donor cells and controllable release in recipient cells. Th... More

关键词

Extracellular vesicles, RNA aptamer, endosome escape, loading efficiency, low-density lipoprotein receptor, therapeutic mRNA
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