Inhibitors of the mitochondrial calcium uniporter for the treatment of disease.

The mitochondrial calcium uniporter (MCU) is a protein located in the inner mitochondrial membrane that is responsible for mitochondrial Ca2+ uptake. Under certain pathological conditions, dysregulation of Ca2+ uptake through the MCU results in cellular dysfunction and apoptotic cell death. Given the role of the MCU in human disease, researchers have developed compounds capable of inhibiting mitochondrial calcium uptake as tools for understanding the role of this protein in cell death. In this article, we describe recent findings on the role of the MCU in mediating pathological conditions and the search for small-molecule inhibitors of this protein for potential therapeutic applications.