Identification of modulators of TRAP1

National Center For Advancing Translational Sciences

Investigators

Abstract

During this period, the team evaluate the most advanced molecules is several cellular and in vivo models of lysosomal storage disorders with promising results. Treatment with TRAP1 agonist normalize the phenotype of several LSD and reduce the lipid content in mouse models of Frabry disease. As a branch, the Early Translation Branch (ETB) has fostered and maintained over 80 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 informatics 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.