| Project/Area Number |
22249042
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| Research Category |
Grant-in-Aid for Scientific Research (A)
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| Allocation Type | Single-year Grants |
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| Section | 一般 |
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| Research Field |
Pediatrics
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| Research Institution | Kyoto University |
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Principal Investigator |
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| Co-Investigator(Kenkyū-buntansha) |
SAITO Megumu 京都大学, iPS 細胞研究所, 准教授 (90535486)
NIWA Akira 京都大学, iPS 細胞研究所, 特定助教 (20546999)
OSHIMA Koichi 京都大学, iPS 細胞研究所, 特定研究員 (60525377)
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| Project Period (FY) |
2010 – 2012
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| Project Status |
Completed (Fiscal Year 2012)
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| Budget Amount *help |
¥48,230,000 (Direct Cost: ¥37,100,000、Indirect Cost: ¥11,130,000)
Fiscal Year 2012: ¥11,700,000 (Direct Cost: ¥9,000,000、Indirect Cost: ¥2,700,000)
Fiscal Year 2011: ¥13,910,000 (Direct Cost: ¥10,700,000、Indirect Cost: ¥3,210,000)
Fiscal Year 2010: ¥22,620,000 (Direct Cost: ¥17,400,000、Indirect Cost: ¥5,220,000)
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| Keywords | 遺伝性疾患 / iPS細胞 / 遺伝子改変 / 疾患解析 / NOGマウス / 疾患特異的 / 創薬 |
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| Research Abstract |
We were engaged in research to generate iPS cells from somaticcells of patients with various hereditary diseases, induced these iPS ells to differentiate appropriately into the cells the diseased organs,and thereby elucidate the etiology and the mechanisms of the diseases. We succeeded the generation of disease-specific iPS cells from patients with congenital neutropenia, Duchenne muscular dystrophy, CINCA syndrome, Nakajyo-Nishimura syndrome, spinal muscular atrophy, Chediak-Higashi syndrome, Hirschsprung disease by using conventional retroviral method. We failed to generate iPS cells from patients with Fanconi Anemia and reticular dysgenesis without gene collection at the stage of skin fibroblasts. We have confirmed recapitulation of phenotypes with several disease-specific iPS cells including CINCA syndrome, reticular dysgenesis and Chediak-Higashi syndrome.
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