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

Saturday, May 31, 2014

Four pillars to provide a cure for Wolfram syndrome and type 1 diabetes

I always carry a card articulating my strategy to provide a cure for Wolfram syndrome. I think there are four pillars to provide a cure for Wolfram syndrome.

1. Stop the progression of the disease.
We are looking for FDA-approved drugs that can delay the progression of Wolfram syndrome. These drugs are currently used for other indications. We are also developing new drugs specifically designed for Wolfram.

2. Raise Awareness
It is important to raise awareness of Wolfram in health care professionals and the general public. We need a list of doctors who have experience in managing patients with Wolfram.

3. Replace damaged tissues
We are creating eye cells, brain cells, and pancreatic beta cells from patients' skin cells (iPS technology). We are also correcting genetic mutations in these cells (genome editing technology).

4. Protect remaining tissues
We are looking for a trophic factor that can protect remaining brain cells and beta cells from dysfunction and death. MANF and MANF-related molecule that we have identified are good candidates.