$199,000 Several promising treatments are being developed for Angelman syndrome (AS), with the goal of restoring function of UBE3A. Therapies administered early in life are expected to be most beneficial, but AS is often not diagnosed until 1-4 years of […]
Read more
$150,000 UBE3A is a protein that labels other proteins for disposal. It is thought that accumulation of proteins that would normally be “trashed” causes the deficits in AS. Using stem cells generated from Angelman syndrome patients or stem cells genetically […]
Read more
$200,000 – 2-years Dr. Zylka’s lab will expand on the work previously funded by ASF that led to the identification of a family of topoisomerase inhibitors that can unsilence the paternal UBE3A gene. In order to learn more about this […]
Read moreBen Philpot, Ph.D. – University of North Carolina-Chapel Hill Heather Hazlett, Ph.D. – Carolina Institute of Developmental Disabilities at University of North Carolina-Chapel Hill Ron Thibert, M.D. – Massachusetts General Hospital $295,970 (2 years) This unique research project is the […]
Read more