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The Philpot lab seeks to understand the pathophysiology underlying monogenic neurodevelopmental disorders, and we use this information to develop small molecule and gene therapies to treat these disorders. Our research focuses on early-stage development of treatments for Pitt-Hopkins, Dup15q, and Angelman syndromes.  Our lab has made key therapeutic discoveries, including developing an approach to unsilence the epigenetically-repressed paternal UBE3A allele as a novel treatment strategy for Angelman syndrome.

Philpot, Ben