Exosome, a promising vector in gene therapy

Sara Rahsepar,1,* Fatemeh soltani ,2

1. School of Pharmacy, Mashhad University of Medical Sciences
2. Biotechnology Research Center, Mashhad University of Medical Sciences



Exosomes are cup-shaped nanovesicles derived from endosome with a size variation of 30–90 nm, which can be secreted by most biological cells. studies demonstrate that exosomes contain mrnas and non-coding rnas such as mirnas which can be transported to recipient cells, as a result exosomes play a significant role in intercellular communication. this natural ability draws attention to exosomes as potential vectors in targeted gene therapy.


Google scholar, pubmed and scopus databases were searched with no filters, using “ exosomes “ , “ gene therapy ” ,“gene delivery “ and “ vector “ as keywords. 52 papers were related and this review includes 21 of them.


Existing gene delivery vectors have plenty of limitations such as immunogenicity, infection and toxicity. on the other hand, exosomes are manufactured from recipient’s own cells and can overcome the immunogenicity problem. they do not have the ability of self-replication, which prevents the infection probability. in addition, exosomes have a small and flexible bi- layer structure and transfect into cells with the same mechanism of viruses, hence have a high bioavailability and a considerable potential for gene delivery. this potential can be enhanced by using targeting peptides on the surface of exosomes.


According to these evidence, it can be concluded that exosomes have a unique potency as gene therapy vectors and require more consideration.


Exosomes, gene therapy,gene delivery, vector