Identification of inhibitors of the polyamine transporter ATP13A3

National Center For Advancing Translational Sciences

Investigators

Abstract

During this reporting period, the project team has worked with collaborators to adopt high-throughput assays of ATP13A3 function, both biochemical and cell-based. The project team has completed screening of NCATS diversity and bioactive collections (>32,000) in an optimized high-throughput polyamine cellular uptake and transport assay. Cherry picking, follow-up and counter screening yielded several ATP13A3 phenotypic inhibitors that were sent to collaborators for confirmation, and validation in orthogonal assays. Analog expansion is currently underway. Structural modeling and virtual screening are in progress.