Identification of modulators of TRAP1

National Center For Advancing Translational Sciences

Investigators

Abstract

During this period, the collaborative team evaluated a panel of advanced TRAP1 agonist leads in several cellular and in vivo models of lysosomal storage disorders (LSDs). Treatment with TRAP1 agonists normalizes the phenotype of several LSDs and reduces lipid content in mouse models of Fabry disease. The team has synthesized analogs for use in cross-coupling studies to characterize potential compound binding sites. The team is also developing recombinant TRAP1 protein to enable cryo-EM studies and DNA-encoded library screening.