2019 Fiscal Year Final Research Report
Investigation of pathogenesis of leukemia in congenital bone marrow failure syndrome using human iPS cells
| Project/Area Number |
17K19600
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| Research Category |
Grant-in-Aid for Challenging Research (Exploratory)
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| Allocation Type | Multi-year Fund |
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| Research Field |
Tumor biology and related fields
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| Research Institution | Hiroshima University |
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Principal Investigator |
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| Co-Investigator(Kenkyū-buntansha) |
小林 正夫 広島大学, 医系科学研究科(医), 名誉教授 (00162016)
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| Project Period (FY) |
2017-06-30 – 2020-03-31
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| Keywords | 小児血液学 / iPS細胞 |
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| Outline of Final Research Achievements |
We established iPS cells from congenital bone marrow failure syndrome. Myeloid cells derived from severe congenital neutropenia (SCN)-iPS cells revealed the maturation arrest at a promyelocyte stage, reflecting the symptom of SCN patients. One from cyclic neutropenia (CyN) didn’t show that. We also knocked-in and -out some genes which are associated with leukemogenesis in SCN by genome-editing technologies and compared the process of the differentiation into blood cells. In addition, SCN-iPS cells were expected to be useful for the investigation into the mechanisms causing the occurrence of leukemia and the establishment of the methods to prevent it in SCN.
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| Free Research Field |
医歯薬学
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| Academic Significance and Societal Importance of the Research Achievements |
小児にみられる先天性骨髄不全症候群には重症先天性好中球減少症など高率に白血病化することが知られているものが多い。重症先天性好中球減少症と近接した遺伝子変異によるとされる周期性好中球減少症では白血病化は起こらず、また臨床経過も異なる。これら疾患の患者由来iPS細胞の比較・解析は白血病化のメカニズムの解明に有効であると考えられる。特に血液に分化していく過程で白血病化を時間経過ごとに追跡することが可能であるiPS細胞を用いることの意義は大きい。今回これらの解析系の確立、安定化により様々な疾患の解析が可能となった。
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