We have created multiple mouse models of Wolfram syndrome to test the efficacy of therapeutic candidates. In addition, we have been developing human cell and tissue models of Wolfram syndrome using induced pluripotent stem cells (iPS cells) derived from patients’ skin cells. These models may enable us to predict more accurately how effective a therapeutic candidate would be in clinical studies. We could successfully create neural progenitor cells from these cells. We are trying to create eye cells and insulin-producing beta cells from Wolfram iPS cells. The dose and safety of therapeutic candidates are currently evaluated in these cells.
Thank you for your continued support and encouragement.