Development of stem cell-based gene therapy adapted for the regeneration-inducing medicine

The goal of our project is to develop a gene therapy targeting stem cells as a curative treatment for inherited diseases based on the collaborative research of “regeneration-inducing medicine” with StemRIM. We aim to provide non-invasive and effective gene therapy through the ex vivo approach, in which stem cells are genetically modified outside the body and then transplanted back again to the patients who have been suffering from intractable inherited diseases, such as epidermolysis bullosa, hemophilia, metabolic disorders, etc.