Urano, Fumihiko (Washington University School of Medicine)
Mahadevan, Jana (Washington University School of Medicine)
Jadhav, Ajit (NCATS)
Yang, Shyh-Ming (NCATS)
Maloney, David (NCATS)
Henderson, Mark (NIDA)
Harvey, Brandon (NIDA)
This technology includes the use of JTV-519 and oxidized form of JTV-519, as a novel treatment for Wolfram syndrome and other diseases associated with endoplasmic reticulum (ER). JTV-519 can prevent the leakage of ER calcium to the cytosol and abnormal activation of a pro-apoptotic enzyme, calpain 2, in cell models of Wolfram syndrome. Further, these compounds can prevent cell death in beta cell models of these diseases.
Wolfram syndrome and other diseases associated with endoplasmic reticulum.
Novel treatment for diseases currently lacking therapeutic options.