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

Monday, May 19, 2014

Wolfram syndrome is a prototype of human endoplasmic reticulum disease

It has been established that endoplasmic reticulum (ER) dysfunction is the root cause of Wolfram syndrome. It would be possible to identify biomarkers reflecting ER health and develop treatments targeting the ER in mechanistically homogeneous Wolfram syndrome patients, which may lead to a breakthrough for treatments of common diseases, such as type 1 and type 2 diabetes and neurodegeneration, in which ER dysfunction is involved.