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

Friday, May 23, 2014

Endoplasmic reticulum disease theory

I feel that we are hitting the tipping point. Our "Endoplasmic reticulum (ER) dysfunction" theory for Wolfram and diabetes is getting attention, which is good news.

Accumulating evidence in my research team indicates that restoring ER calcium homeostasis is beneficial for Wolfram syndrome patients. We are testing this theory using three animal models and cells derived from patients. There are two ways to restore ER calcium homeostasis.

1. Increase calcium influx to the endoplasmic reticulum.
2. Decrease calcium efflux from the endoplasmic reticulum.

At this point, the second strategy seems to be working, but we are trying both. My hope is to test the second strategy in healthy individuals and patients soon.