AMPK synthetic activators

National Center For Advancing Translational Sciences

Investigators

Abstract

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.