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

Wednesday, May 28, 2014

Our current efforts May 28, 2014

I would like to briefly outline our current efforts.

1. Develop drugs that can delay the progression of Wolfram syndrome.
As of today, we are focusing on one FDA-approved drug. We are testing the efficacy of this drug in iPS cells from patients and two animal models of Wolfram syndrome. We are also testing two new types of drugs.

2. Making eye cells using iPS cells derived from Wolfram syndrome patients.
As of today, we started seeing eye-like cells in our dishes. I am excited by this. We will keep on differentiating these cells and test the efficacy of our candidate drugs to develop treatments for eye manifestations in Wolfram syndrome.

3. Measuring the levels of disease-progression biomarkers
We have identified two potential disease-progression biomarkers. As of today, we are measuring the levels of these markers in blood samples from patients and control subjects.

4. Correcting genetic mutations using CRISPR technology in cells from Wolfram syndrome patients.
In the future, we need to correct genetic mutations in pancreatic beta cells, brain cells, and eye cells derived from patients' skin cells before they are transplanted. We are performing a "molecular surgery" to replace a mutated segment of WFS1 gene with a normal segment of WFS1 gene in cells from patients.