Targeted Delivery of Biomolecules to the CNS via Extracellular Vesicles

Type of Award: catalyst
Award Number:
Award Period: September 2017 - August 2019
Amount Awarded: $ 250,000.00
PI(s): Ernesto Bongarzone, UIC; Steven Roth, UIC; Joshua Leonard, NU;

Abstract: The complex structure of the central nervous system (CNS) and its privileged circulatory isolation by the blood-brain barrier pose challenges for delivering therapeutic molecules. A novel means to overcome these challenges is to leverage the advantageous properties of extracellular vesicles (EVs). EVs are cellularly-secreted nanoparticles that transfer RNA, lipids, and proteins to recipient cells via endocytosis and membrane fusion. EVs are highly attractive as therapeutic delivery vehicles, since they exhibit favorable stability, non-toxicity, are non-immunogenic, and may cross the blood-brain barrier (BBB). However, efficiently directing EVs to specific cells remains a limitation. In this proposal, we will use our distinct and complementary expertise to develop novel vehicles for delivering therapeutics to the CNS, by engineering EVs to express surface recognition proteins that direct their uptake by key cells involved in most disease processes in the brain - neurons and oligodendrocytes (OLs). The PIs will leverage their years of experience with the retina and optic nerve, and the spinal cord, as target regions for testing this central hypothesis.