| Budget Amount *help |
¥5,070,000 (Direct Cost: ¥3,900,000、Indirect Cost: ¥1,170,000)
Fiscal Year 2013: ¥1,690,000 (Direct Cost: ¥1,300,000、Indirect Cost: ¥390,000)
Fiscal Year 2012: ¥1,170,000 (Direct Cost: ¥900,000、Indirect Cost: ¥270,000)
Fiscal Year 2011: ¥2,210,000 (Direct Cost: ¥1,700,000、Indirect Cost: ¥510,000)
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| Research Abstract |
We focused on the use of iPSCs for cell-based therapy of hemophilia. We generated iPSCs from mesenchymal stem cells isolated from C57BL/6 mice. The mouse iPSCs were generated through the induction of four Yamanaka transcription factor genes. The iPSCs released functional coagulation factor VIII (FVIII) following transduction with a lentiviral vector. The subcutaneous transplantation of iPSCs expressing FVIII into nude mice resulted in teratoma formation, and significantly increased plasma levels of FVIII. The plasma concentration of FVIII was at levels appropriate for human therapy at 2–4 weeks post-transplantation. We next examined which promoter was suitable for the production of coagulation factor from iPSCs, and found that EF1alpha promoter stably drove transgene in iPSCs. We also developed iPSCs from FIX-deficient mouse fibroblasts by sendai virus vector. The established iPSCs had a potency to produce chimeric mouse, and could produce functional FIX after the transduction.
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