Establishment of a novel disease model using disease specific iPS cells of congenital neutropenia
| Project/Area Number |
24591548
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| Research Category |
Grant-in-Aid for Scientific Research (C)
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| Allocation Type | Multi-year Fund |
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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 |
WATANABE Kenichiro 京都大学, 医学(系)研究科(研究院), 臨床教授 (20324634)
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| Co-Investigator(Kenkyū-buntansha) |
梅田 雄嗣 京都大学, 大学院医学研究科 (80397538)
平家 俊男 京都大学, 大学院医学研究科 (90190173)
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| Project Period (FY) |
2012-04-01 – 2015-03-31
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| Project Status |
Completed (Fiscal Year 2014)
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| Budget Amount *help |
¥5,460,000 (Direct Cost: ¥4,200,000、Indirect Cost: ¥1,260,000)
Fiscal Year 2014: ¥1,560,000 (Direct Cost: ¥1,200,000、Indirect Cost: ¥360,000)
Fiscal Year 2013: ¥1,950,000 (Direct Cost: ¥1,500,000、Indirect Cost: ¥450,000)
Fiscal Year 2012: ¥1,950,000 (Direct Cost: ¥1,500,000、Indirect Cost: ¥450,000)
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| Keywords | 疾患特異的iPS細胞 / 骨髄不全 / 細胞死 / 疾患モデル / iPS細胞 / 好中球減少 / 好中球 |
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| Outline of Final Research Achievements |
We generated induced pluripotent stem cell lines from a patient presenting for severe congenital neutropenia with HAX1 gene deficiency and analyzed their in vitro neutrophil differentiation potential. Cytostaining and flow cytometric analyses of myeloid cells differentiated from patient-derived induced pluripotent stem cells showed arrest at the myeloid progenitor stage and apoptotic predisposition, both of which replicated abnormal granulopoiesis. Moreover, lentiviral transduction of the HAX1 cDNA into patient-derived induced pluripotent stem cells reversed disease-related abnormal granulopoiesis. This in vitro neutrophil differentiation system, which uses patient-derived induced pluripotent stem cells for disease investigation, may serve as a novel experimental model and a platform for high-throughput screening of drugs for various congenital neutrophil disorders in the future.
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Report
(4 results)
Research Products
(5 results)
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[Journal Article] HAX1 in induced pluripotent stem cells from a patient with severe congenital neutropenia improves defective granulopoiesis.2014
Author(s)
Morishima T, Watanabe K, Niwa A, Hirai H, Saida S, Tanaka T, Kato I, Umeda K, Hiramatsu H, Saito MK, Matsubara K, Adachi S, Kobayashi M, Nakahata T, Heike T
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Journal Title
Haematologica
Volume: 99(1)
Issue: 1
Pages: 19-27
DOI
Related Report
Peer Reviewed / Open Access
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