In Wolfram syndrome, some cells are sensitive to the loss of function of Wolfram gene. These are insulin-producing pancreatic beta cells, some brain cells, and some eye cells. Why are these cells are sensitive? This is the key to provide a cure for Wolfram syndrome. If we know why these cells are sensitive, we can design a therapy for protecting these cells. In the past 24 months, we discovered that loss of function of Wolfram gene causes dysregulated calcium homeostasis in our cells. So we are trying to manipulate calcium homeostasis by drugs to delay the progression. We still don’t know why these cells are sensitive to dysregulated calcium homeostasis. I keep on thinking.
Thank you for reading this blog. I hope that I will receive the key soon.