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

Sunday, March 9, 2014

Patient-based therapeutics part 7- Making eye cells

I am on my way to Japan to establish a collaboration with a major Japanese medical center on our Wolfram syndrome project. Why Japan? The reason is very simple. They have a technology to make eye cells from patient-derived induced pluripotent stem cells (iPS cells). If my research group can constantly make eye cells from Wolfram syndrome iPS cells, we can understand the mechanisms of optic atrophy in Wolfram syndrome and test the efficacy of our candidate drugs using these eye cells. This is also another opportunity to raise awareness of Wolfram syndrome.

As I repeatedly mentioned in my previous blogs, Wolfram is a weapon to combat diabetes and blindness. I believe that Wolfram is an accelerated form of diabetes and blindness. Many of us will experience beta cell death and death of eye cells later in our lives. A drug that can delay the progression of Wolfram may be helpful for us when we get old.