Ticket to Ride – Targeting Proteins to Exosomes for Brain Delivery


Addressing Proteins to Exosomes for Targeted Delivery

Targeting therapeutically relevant biomolecules to protected sites of the body, such as the brain, is a major challenge in the design of molecular therapies. Exosomes recently emerged as promising vehicles for targeted drug delivery, offering advantages such as good biocompatibility, low immunogenicity, targeting specificity, and the ability to overcome barriers. While, to date, most strategies for linking biomolecules to exosomes have dealt with RNA or small molecules, a specific approach for the active and selective packaging of proteins has so far been lacking. In this issue of Molecular Therapy, Sterzenbach et al. present an elegant strategy for specific sorting of protein cargo to exosomes by engineering an exosome address signal to the protein of interest. The authors further demonstrate that, upon administration via the nasal route, exosomes carry the engineered protein across the blood-brain barrier and target its biological activity to neural cells. The study opens up new perspectives for therapeutic targeting of biologically active proteins to the brain as well as other tissues. (read more…)

Krämer-Albers EM. (2017) Ticket to Ride: Targeting Proteins to Exosomes for Brain Delivery. Mol Ther [Epub ahead of print]. [article]

Leave a Reply

Your email address will not be published. Required fields are marked *