SMN Ubiquitin Ligase

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During this period, the NCGC has conducted a medicinal chemistry campaign to define structure-activity relationships of the lead chemotypes and further improve their potencies. A new secondary assay has also been under active development to provide further detail and needed activity data to support ongoing medicinal chemistry efforts and better drive SAR determination. As a center, the NCGC has fostered and maintained over 130 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.