EV-based Nanocarriers for NF1 Therapies

This project will seek to develop novel delivery systems for therapeutic payloads to treat NF1. The goal is to develop next generation carriers based on “designer” (i.e., custom made) extracellular vesicles (EVs), capable of targeting neurofibromas to deliver genetic material to reduce tumor burden. EVs are cell-derived natural carriers that play a crucial role in relaying signals between cells under healthy and pathological conditions. The implementation of designer EVs as novel delivery systems for the treatment of NF1 will help to overcome major limitations observed with other status quo delivery methodologies. Currently there are no examples of EV-based carriers for the deployment of gene therapies against NF1. Thus, the work proposed herein is fundamentally innovative and potentially transformative. The advancements derived from this work will allow us to deliver different types of therapeutic cargos against NF1, which will be key to enabling collaborations with the wider NF1 research and clinical care community to help find an effective treatment for this condition.