Development of microRNA AAV Vectors to Improve Angelman Syndrome

This grant focuses on the development of a microRNA approach as a potential therapeutic for the treatment of Angelman syndrome.  In collaboration with the Gene Therapy Program at the University of Pennsylvania, the latest research plan will build on the milestones already achieved with CRISPR-Cas9/sgRNA interference of the UBE3A-antisense transcript (UBE3A-AS) to develop a novel strategy to suppress UBE3A-AS extension utilizing microRNAs as a potential one-and-done treatment option.