Novel therapeutics for Angelman syndrome by manipulating Ube3a expression
$199,972 (2-year award)
Funding for this grant is an attempt to promote research targeted to identifying agents or druggable compounds that may increase expression of UBE3A in brain neurons. Using multidisciplinary facilities, advanced genetic engineering and robotic drug analysis, this grant attempts to screen thousands of chemicals and compounds through an experimental protocol that will enable identification of UBE3A protein expression. This research has exciting promise of identifying potential new therapeutic agents or identifying new protein or molecular pathways that may improve UBE3A expression.
This study proved that the paternal copy of UBE3A could be activated.