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!
