Bioinspired EV mimetics for the delivery of gene editing compounds

The ability to change genetic information efficiently and precisely using CRISPR/Cas gene editing has transformed the fields of life sciences and precision medicine. The potential of this cutting-edge technology is severely limited by our inability to effectively and specifically transport these molecules to their active site. Extracellular vesicles (EVs) are natural nanoparticles that have an inherent ability to target specific cells – a feature that can be exploited for drug delivery. Yet, EVs are a heterogeneous population of vesicles, and their preparation is ill-defined, motivating the need for mimetic carriers. In this project, we aim to develop bioinspired EV mimetics to deliver gene editing compounds. By mimicking the key molecules of EVs and employing bioengineering, we will generate synthetic nanoparticles with enhanced stability and tissue-targeting capacity. The mimetics will be tested in cell models and evaluated in mice for body-wide distribution and gene editing potency.