Ube3a STUb gene therapy for the treatment of Angelman syndrome.

Primary Investigator: K. Nash

This project funded Agilis Biotherapeutics to work with University of South Florida to establish a novel gene therapy approach to examine the unique delivery methods of AAV in conjunction with a uniquely engineered E6-AP protein that was designed to allow secretion followed by uptake in neurons. This is an opportunity to bypass the limitation of limited neuronal transfection of AAV and distribute more Ube3a into neurons of the brain.