Reduction of ATXN2 expression as a means to treat Spinocerebellar Ataxia Type 2 (SCA2)

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During this period, the NCGC conducted the primary screen and identified putative inhibitors of ATXN2 expression, which are currently being validated. 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.