Developing inhibitors for PIP4Kgamma for Huntington's disease

National Center For Advancing Translational Sciences

Investigators

Linked publications & trials

Abstract

The main goal of this project is to develop potent and selective inhibitors of phosphatidylinositol-5-phosphate 4-kinase, type II, gamma (PIP4Kgamma). During this period, the project team has further characterized the previously developed lead molecules using assays to 1) measure huntington protein levels, 2) assess cytotoxicity induced by mutant huntington protein, 3) examine target engagement using biophysical approaches. The team has also investigated the cellular effects of PIP4K inhibition/silencing using transcriptomic methods.