| Project/Area Number |
26860733
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| Research Category |
Grant-in-Aid for Young Scientists (B)
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| Allocation Type | Multi-year Fund |
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| Research Field |
Hematology
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| Research Institution | Tottori University |
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Principal Investigator |
Kurosaki Hajime 鳥取大学, 医学(系)研究科(研究院), 助教 (70464295)
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| Project Period (FY) |
2014-04-01 – 2017-03-31
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| Project Status |
Completed (Fiscal Year 2016)
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| Budget Amount *help |
¥3,900,000 (Direct Cost: ¥3,000,000、Indirect Cost: ¥900,000)
Fiscal Year 2015: ¥1,950,000 (Direct Cost: ¥1,500,000、Indirect Cost: ¥450,000)
Fiscal Year 2014: ¥1,950,000 (Direct Cost: ¥1,500,000、Indirect Cost: ¥450,000)
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| Keywords | 血友病 / 幹細胞 / 再生医療 / 人工染色体ベクター / iPS細胞 / 細胞補充 |
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| Outline of Final Research Achievements |
Artificial chromosome (AC) vectors have some unique characteristics as compared with conventional vectors, carrying large transgenes without size limitation, showing persistent expression of transgenes, and existing independently from host genome in cells. Hemophilia A (HA) is X chromosome-linked hemorrhagic disorder caused by defects in the coagulation factor VIII (FVIII) gene. Induced pluripotent stem (iPS) cells, which can be generated from an individual’s own tissues and contribute to any tissues, have a great potential for gene therapy. Induced-PS cells were generated from HA mouse embryonic fibroblasts by introducing AC vector with four reprogramming factors. The iPS cells were corrected by transferring AC vector with FVIII gene. The AC vector system, which contains the combination of reprogramming factors for making iPS cells and complement genes, may be a promising tool for safer gene- and cell-therapy of HA.
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