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During this period the team has pursued small molecules to activate 5' adenosine monophosphate-activated protein kinase (AMPK). The team has optimizing selected chemotype via medicinal chemistry efforts. AMPK activators are of interest for Niemann Pick C, a lysosomal storage disease characterized by cholesterol accumulation. The project team has developed a novel high-throughput mass spectrometry-based assay that allows direct measurement of cholesterol in relevant cells. The set of candidate AMPK activators are being assessed using the mass spectrometry assay.
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A Novel Cell-Based Assay to Identify Small Molecules for Galactocerebrosidase (GALC)
Identifying small molecules with selective toxicity towards muscle invasive prostate cancer cells
Evaluation of CD206 agonists in diabetic retinopathy
Identification of small molecules that improve trafficking of NLGN4X/Y for autism
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