This collaborative team aims to identify and, where possible, repurpose compounds that are capable of correcting ER dysfunction in Wolfram syndrome. This includes the pursuit of small molecules that modulate ER calcium dysfunction or restore Wfs1 function by correcting protein misfolding. During this period, the collaborative team worked to develop a high-throughput amenable assay to identify small molecules that improve folding of mutant Wfs1. Additionally, the repurposing of small molecules with ER calcium activity was advanced by synthesizing compounds and characterizing them in Wolfram-relevant assays.
