We are actively using induced pluripotent stem cells (iPS cells) to develop therapeutics for Wolfram syndrome. What are iPS cells? These are a type of stem cells derived from our skin cells. iPS cells can be differentiated into any types of cells. So we can use these cells to replace damaged tissues in patients with Wolfram syndrome and other disorders.
In addition, we can use iPS cells to model our disease. Professor Doug Melton at Harvard, a prominent scientist who has developed a method to create insulin-producing cells from stem cells, talked about iPS cells in his interview. In short, we can model our disease using iPS cells to understand the mechanisms and test potential therapies.
Professor Melton said, “If you wanted to design a thought experiment to get at the cause of type 1 diabetes, it would go as follows. We would take a person with diabetes and clone them 100 times. We would put the clones in different environments, feed them different foods, give them different viruses and every five minutes take blood samples, do pancreatectomies and try to figure out the primary cause. Obviously we are not going to do this experiment, but iPS cells give us an alternative, which is why I am so excited.”
Thank you for reading this blog. I am hopeful.