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A cure for Wolfram syndrome could lead to a cure for diabetes and blindness.

Friday, January 10, 2014

A potential target for the treatment of Wolfram syndrome

Since I moved to Washington University School of Medicine in July, 2012, the pace of my research on Wolfram syndrome has accelerated. I have a much better understanding of the disorder clinically and at the molecular level. As I mentioned in my blog a year ago, I believe that calcium leakage from the endoplasmic reticulum to the cytosol is one of the major etiologies for beta cell death and neurodegeneration in Wolfram. We have discovered that the calcium leakage results in calpain-2 enzyme activation and cell death. These findings will be published very soon. We are looking for drugs that can prevent this process. If my theory holds true, the drug that can halt the calcium leakage should delay the progression of the disease.