2014 Fiscal Year Final Research Report
Analysis of occurrence of leukemia from congenital bone marrow failure syndrome and establishment of its treatment using human iPS cells
| Project/Area Number |
26670511
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| Research Category |
Grant-in-Aid for Challenging Exploratory Research
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| Allocation Type | Multi-year Fund |
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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) |
OTSU Makoto 東京大学, 医科学研究所, 准教授 (30361330)
MOCHIZUKI Shinji 東京大学, 医科学研究所, 特任助教 (90349473)
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| Project Period (FY) |
2014-04-01 – 2015-03-31
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| Keywords | 先天異常学 |
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| Outline of Final Research Achievements |
Severe congenital neutropenia (SCN) and cyclic neutropenia (CyN) are belonged to congenital bone marrow failure syndrome (CBMFS). Acute myelogenous leukemia (AML) was sometimes developed in the patients with some CBMFS including SCN. No AML occurred in the patients with CyN. To investigate the mechanism of AML occurrence, we established iPS cells from the patients (both had mutation in ELANE gene region) with SCN (SCN-iPS cells) or CyN (CyN-iPS cells), respectively. Myeloid cells derived from CyN-iPS cells revealed that the capability to differentiate into hematopoietic cells including neutrophils was almost similar to control iPS cells. SCN-iPS cells showed decreased capability into neutrophils, reflecting the disease status of SCN patients. To compare the mechanism of hematopoietic differentiation from SCN- and CyN-iPS cells was expected to be useful to analyze the mechanism of the onset of AML and treatment of these AML.
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| Free Research Field |
小児血液
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