Abstract

Gene Editing and Targeted Delivery Using Engineered Extracellular Vesicles

Samir EL-Andaloussi, Associate Professor, Karolinska Institute

Extracellular vesicles (EVs) have emerged as important mediators of intercellular communication due to their ability to transfer bioactive lipids, proteins and different species of RNA into cells. As such, EVs can be harnessed for the delivery of macromolecular drugs. Adapting EVs for drug delivery requires cellular engineering that allows for selective loading of biotherapeutics inside or on the surface of EVs. This presentation will cover our recent developments in EV engineering, with focus on active loading and functional delivery of gene editing modalities. In addition to addressing PK, PD and functional delivery of EVs in normal and diseased mice, strategies to enhance tissue targeting and extending plasma half-life of circulating EVs will be discussed.