2011 Fiscal Year Final Research Report
Analysis of pathogenesis of congenital bone marrow failure syndrome and establishment of its treatment using human iPS cells
| Project/Area Number |
21390321
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| Research Category |
Grant-in-Aid for Scientific Research (B)
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| Allocation Type | Single-year Grants |
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| Section | 一般 |
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| Research Field |
Embryonic/Neonatal medicine
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| Research Institution | The University of Tokyo |
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Principal Investigator |
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| Co-Investigator(Kenkyū-buntansha) |
TSUJI Kohichiro 東京大学, 医科学研究所, 准教授 (50179991)
ETO Kouji 京都大学, iPS細胞研究所, 教授 (50286986)
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| Project Period (FY) |
2009 – 2011
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| Keywords | iPS細胞 / 先天性骨髄不全症候群 / 重症先天性好中球減少症 / ダウン症候群 / RUNX1 / WNT3a |
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| Research Abstract |
1) Analysis of severe congenital neutropenia-derived iPS cells(SCN-iPS cells) Clonal hematopoietic assay indicated that SCN-iPS cells had a significantly decreased capability to generate neutrophil colonies, which contained few mature neutrophils, reflecting the feature of SCN. Since the expression of WNT/b-Catenin pathway-related genes were decreased, we added WNT3a to hematopoiesis-indunction culture. WNT3a stimulated the maturation of SCN-iPS cell-derived neutrophils. These results suggested the possibility of treatment of SCN using WNT3a.
2) Analysis of Down syndrome-derived iPS cells(DS-iPS cells) Clonal hematopoietic assay indicated that DS-iPS cells had increased capabilities to generate all types of definitive hematopoietic/blood cells. Microarray analysis also demonstrated an increased expression of RUNX1 on chromosome 21, which was confirmed by RT-PCR. These results indicated that hematopoietic abnormalities in DS patients might relate with the increased expression of RUNX1, which could be a target gene for the treatment of hematopoietic disorders in patients with Down syndrome.
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[Remarks] 東京大学医科学研究所
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[Remarks] 幹細胞治療研究センター
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[Remarks] 幹細胞プロセシング分野
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