| Budget Amount *help |
¥18,460,000 (Direct Cost: ¥14,200,000、Indirect Cost: ¥4,260,000)
Fiscal Year 2019: ¥5,330,000 (Direct Cost: ¥4,100,000、Indirect Cost: ¥1,230,000)
Fiscal Year 2018: ¥5,720,000 (Direct Cost: ¥4,400,000、Indirect Cost: ¥1,320,000)
Fiscal Year 2017: ¥7,410,000 (Direct Cost: ¥5,700,000、Indirect Cost: ¥1,710,000)
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| Outline of Final Research Achievements |
Recessive dystrophic epidermolysis bullosa is an inherited disorder characterized by blisters and erosions on the whole body. It was considered that a radical treatment could be developed by combining a local treatment in which fibroblasts and epidermal keratinocytes differentiated from iPS cells genetically treated using gene editing technology such as CRISPR/Cas9 are directly administered to the affected area and a systemic treatment by systemic administration of hematopoietic/mesenchymal stem cells. In this study, we established iPS cells from epidermal keratinocytes derived from patients with epidermolysis bullosa, and confirmed the possibility of differentiation into mesenchymal stem cells. Furthermore, we have established a technology to specifically and efficiently repair gene mutations that are frequently found in Japanese patients.
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