Epigenetic regulation of Ube3a by a candidate Angelman syndrome drug (UNCilencer1)

$110,000

The ASF Joseph Wagstaff Postdoctoral Research Fellowship was granted to its first recipient.  Dr. King will do his postdoctoral research on a new drug UNCilencer1, which turns on the silenced paternal UBE3A gene in mouse neurons.  UNCilencer1 is a member of a class of agents known as topoisomerase inhibitors.  His work will explore the extent to which this drug and other related agents change UBE3A expression in the brain and will explore mechanisms whereby the topoisomerase inhibitors interact/affect expression of UBE3A antisense.

Screening will be done to make sure the drug is specific for UBE3A and thus not able to up-regulate or turn on any other important silenced genes.  It is hoped that the results of these experiments will advance a pharmaceutical treatment for those with Angelman syndrome.