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

Saturday, July 12, 2014

Patient-based therapeutics 13: Molecular Surgery 2

We have been trying to replace the disease-causing Wolfram gene segment with a "healthy" Wolfram gene segment in iPS cells from patients with Wolfram syndrome.

http://wolframsyndrome.blogspot.com/2014/05/patient-based-therapeutics-part-10.html

Why do we need to do it? Even if we can successfully make high-quality eye cells and brain cells from iPS cells of Wolfram syndrome patients, these cells may degenerate again because of the disease-causing WFS1 segment (called mutations). 

Using an engineered enzyme and artificial DNA, we are replacing the disease-causing segment with the healthy one. We designed artificial DNA, confirmed its efficacy in vitro, and introduced it into patients' iPS cells. It took a few months to do these because this is a new technology. Currently we are checking if the disease-causing segment has been successfully replaced using DNA sequencing. I will keep you updated about our progress.

Have the best weekend of your life!