qHTS to Identify Inhibitors of DYT1 Inclusion Formation

National Center For Advancing Translational Sciences

Investigators

Linked publications, trials & patents

Abstract

During this period, the NCGC has screened additional targeted small molecule libraries against the DYT1 assay to identify potential repurposing leads. Compounds of interest are currently being tested in advanced cell-based models and secondary assays to further characterize their activity. As a center, the NCGC has fostered and maintained over 110 active collaborations with both NIH and extramural investigators, facilitating drug discovery efforts across the entire spectrum of human disease. These efforts have led to dozens of high-throughput screens and a number of medicinal chemistry campaigns to further improve on screening hits, providing our collaborators and the general research community with publications and a variety of promising small molecule probes and leads. In addition, the NCGC has worked to advance a number of informatic initiatives to make better use of existing drug and disease target information and provide the general public with easily accessible resources, further catalyzing the development of new therapies for human disease.