Identification of transcriptional repressors of a-synucelin (SNCA) expression

National Center For Advancing Translational Sciences

Investigators

Abstract

This collaborative project aims to identify small molecule transcriptional repressors of alpha-synuclein (SNCA) for PD indications. During this period, the collaborative team screened an additional 45,000 compounds, bringing the total to 150,000 compounds for the project. After confirmation experiments and refinement using a series of counterscreens, compounds were sent to the University of Utah. These compounds are being characterized in cell based models of Parkinsons disease, towards the goal of selecting a lead compound for further development. The collaborator has characterized qHTS hits in more advanced assays and has confirmed their activity. We have identified confirmed novel hits from the screen as well as known AKT inhibitors. A manuscript is under review on repurposing on a known inhibitor and a medicinal chemistry campaign is underway.