We are creating different types of eye cells using iPS cells derived from skin cells of patients with Wolfram syndrome. We plan to use these cells to understand the mechanisms of eye manifestations in Wolfram and test potential drugs. In the future, these cells could be used to replace damaged tissues after the abnormal gene segment is replaced with the normal one.
http://wolframsyndrome.blogspot.com/2014/05/patient-based-therapeutics-part-9.html
Yesterday, we got the first type of eye cells! I got so excited. It took more than two months to do it, and one of my scientists came to the lab every day including holidays and Sundays in the past few months. I really appreciate his efforts and support from others, including the funding support from the Snow Foundation and the Internal Medicine of Washington University Medical Center. Now we can start testing MANF and other drugs. We will make the best use of these cells!