Frank J. Schoenen

TL;DR: N2,N4-Dibenzylquinazoline-2,4-diamine (DBeQ) was identified as a selective, potent, reversible, and ATP-competitive p97 inhibitor that blocks multiple processes that have been shown by RNAi to depend on p97, including degradation of ubiquitin fusion degradation and endoplasmic reticulum-associated degradation pathway reporters.

TL;DR: Dopaminergic neurons were generated from iPSC lines derived from patients with Gaucher disease with and without parkinsonism, suggesting that noninhibitory small-molecule chaperones of glucocerebrosidase may prove useful for the treatment of Parkinson disease.

TL;DR: The results nominate ML240 as a promising starting point for the development of a novel agent for the chemotherapy of cancer, and provide a rationale for developing pathway‐specific p97 inhibitors.

TL;DR: How novel small-molecule probes identified through the NIH Molecular Libraries Program are enabling the exploration of biological pathways and therapeutic hypotheses not otherwise testable is described.