Development of cell therapy for epidermolysis bullosa

我们进行了干细胞移植，包括骨髓移植和间充质干细胞/基质细胞输注到成年大疱性表皮病模型小鼠（XVII型胶原（COL 17）基因敲除小鼠）。1.骨髓内灌注/移植诱导的外周血中的植入比常规血管内灌注更好，并且还观察到COL 17的恢复。2.采用流式细胞仪分别收集供者和受者角质形成细胞，采用基因芯片技术检测mRNA表达差异。共检测到57个基因在两组间表达差异。3.与名古屋大学合作，目前正在申请对严重的大疱性表皮病患者进行干细胞移植的临床试验，并正在考虑中。

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.