| Budget Amount *help |
¥4,810,000 (Direct Cost: ¥3,700,000、Indirect Cost: ¥1,110,000)
Fiscal Year 2017: ¥1,300,000 (Direct Cost: ¥1,000,000、Indirect Cost: ¥300,000)
Fiscal Year 2016: ¥1,690,000 (Direct Cost: ¥1,300,000、Indirect Cost: ¥390,000)
Fiscal Year 2015: ¥1,820,000 (Direct Cost: ¥1,400,000、Indirect Cost: ¥420,000)
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| Outline of Final Research Achievements |
In our study, we attempted to establish gene and cell therapy for recessive dystrophic epidermolysis bullosa (RDEB) using induced pluripotent stem cell (iPSC) technology. RDEB is an inherited skin disorder caused by the mutation in the type VII collagen gene, an important component for skin integrity between epidermis and dermis, resulting in skin fragility. Therefore, RDEB is characterized by repeated and incurable skin blisters with minor trauma. We first generated iPSC from T cells isolated from the patient with RDEB using Sendai virus vector to avoid viral gene insertion. We next tried to correct pathogenic gene mutation in RDEB-derived iPSCs using homologous recombination-based CRISPR/CAS9 system. In this trial, we modified template DNA plasmid by installing drug-resistance gene flanked by transposon inverted repeat sequence to completely remove drug-resistance gene after gene correction, and confirmed whether our system worked well or not.
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