Calpain inhibitor and ibudilast rescue β cell functions in a cellular model of Wolfram syndrome

• DOI: 10.1073/pnas.2007136117
• Publication Date: 2020-07-07T00:27:53Z
• Authors: Lien D. Nguyen; Tom T. Fischer; Damien Abreu; Alfredo Arroyo; Fumihiko Urano; Barbara E. Ehrlich
• Keywords: Wolfram syndrome

Significance Wolfram syndrome is a rare multisystem disease characterized by diabetes insipidus, mellitus, optic nerve atrophy, and deafness (DIDMOAD). It primarily caused mutations in the 1 gene, WFS1 . As monogenetic disorder, model for neurodegeneration. There no effective treatment this invariably fatal disease. Here we characterize as regulator of calcium homeostasis subsequently target signaling to reverse deficits cellular syndrome.