Utilization of extracellular vesicles as carriers/ vehicle of biological substances to cells and organs

Purpose and goal

The specific aim of this project was to explore the possibility of using human exosomes, as biological drug-delivery-vehicles to transport functional RNA molecules to cells and tissues in order to treat or cure diseases. In this project, we have succeeded for the first time to use human exosome vesicles for the delivery of exogenous mRNA to cells both in vitro and in vivo. We have invented a method to introduce functional mRNA molecules into various exosomes and use them as a biological transport vehicle in vitro and in vivo.

Expected results and effects

We recently discovered that there is a link (transport of molecules) between cell endocytosis and exocytosis (secretion of exosomes) (Maugeri M. et al. Nature Communication, 2019). Our results show for the first time that a fraction of cell-endocytosed mRNA can be detected in secreted exosomes. Thus, we used this cellular process to introduce functional mRNA molecules into various human exosomes. We discovered that these exosomes could transport the administered mRNA molecules to target cells and cause the production of a new protein both in vitro and in vivo.

Planned approach and implementation

The development of a safe drug-delivery vehicle is expected to have a major impact on the treatment of several different diseases. Since human exosomes naturally transport biological material (a cargo of RNAs and proteins) between cells and organs, we hypothesized that these vesicles might be an ideal candidate as biological vehicle (the body’s own vehicle/ carrier), to deliver therapeutic agents (e.g. mRNAs) to cells in order to treat or cure diseases. In this project, we have succeeded to introduce foreign mRNA molecules into exosomes and use them for in vivo mRNA delivery.