Wolfram Syndrome iPSCs Today I would like to discuss how we use induced pluripotent stem cells (iPS cells) derived from patients with Wolfram syndrome for developing treatment. Our group as well as a group in Columbia University have created iPS cells from patients with Wolfram syndrome.What are induced pluripotent stem cells (iPS cells)? iPS cells […]
Patient-Based Therapeutics Part 4 – Drug Screening Progress Based on the data obtained from our patients, animal models, and cell models of Wolfram syndrome, we found that calcium depletion of the endoplasmic reticulum (ER) plays a role in the pathogenesis of Wolfram syndrome. So we have been looking for drugs that can prevent ER calcium-depletion-mediated […]
A Few weeks ago, a young woman who had been battling Wolfram syndrome for many years passed away. Everyone knew, loved and supported her. Below is a touching blog post from Washington University School of Medicine’s Dr. Fumihiko Urano about our friend, Ms. K. In Memory of K Yesterday I was heartbroken because I learned […]
Why do I study such a rare disease as Wolfram Syndrome? I am often asked, “Why do you study such a rare disease, Wolfram?” My answer is, “It is the weapon to combat common diseases.” My secret answer is, “I want to help Wolfram patients and their families.” Why it is so important to study […]
Research Update from Dr. Fumihiko Urano Patient-Based Therapeutics Part 3 Instead of introducing my research activities, I would like to introduce my colleagues today. I have a lot of colleagues who have been helping me develop diagnostics and therapeutics for Wolfram at the Washington University Medical Center. Without their help, I cannot accomplish anything. I […]
Research Update from Dr. Fumihiko Urano Patient-Based Therapeutics Part 2 We are taking an unconventional approach to develop therapeutics for Wolfram syndrome. I would call it “patient-based therapeutics.” This implies a few things. One of these is the “mechanism-based treatment.” How can we achieve this component of “patient-based therapeutics” for Wolfram syndrome? Here are our […]