Our group as well as a few other groups around the world showed that Wolfram syndrome is associated with endoplasmic reticulum. Endoplasmic reticulum is a cellular compartment executing multiple functions, including the quality control of proteins destined for secretion and calcium storage and release.
Our recent discovery strongly suggests that dysregulated calcium storage and release in the endoplasmic reticulum plays a role in the pathogenesis of Wolfram syndrome. Thus, drugs that target endoplasmic reticulum may be beneficial for patients with Wolfram syndrome. There are not many drugs that can modulate endoplasmic reticulum functions. There are at least there different drug targets in the endoplasmic reticulum. These are SERCA, IP3 receptor, and Ryanodine Receptor. We need to identify and test more drugs that can modulate these targets.
Thank you for reading this blog. I hope that 2015 will be a great year for us.