Development of cell therapy for epidermolysis bullosa

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We performed stem cell transplantation including bone marrow transplantation and mesenchymal stem/stromal cell infusion into adult epidermolysis bullosa model mice (type XVII collagen (COL17) knockout mice). 1. Intramedullary infusion/transplantation induced better engraftment in the peripheral blood than conventional intravascular infusion, and the recovery of COL17 was also observed as well. 2. Donor-derived keratinocytes and recipient-derived keratinocytes were respectively collected by flowcytometer, and investigated mRNA expression differences by microarray technique. 57 genes were detected which have different mRNA expression between the two groups. 3. In corporation with Nagoya University, clinical trials of stem cell transplantation for severe epidermolysis bullosa patients are now applying, and it is under consideration.