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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

Research Project

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Project/Area Number 26670511
Research Category

Grant-in-Aid for Challenging Exploratory Research

Allocation TypeMulti-year Fund
Research Field Embryonic/Neonatal medicine
Research InstitutionThe University of Tokyo

Principal Investigator

EBIHARA Yasuhiro  東京大学, 医科学研究所, 特任准教授 (40302608)

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)
Project Period (FY) 2014-04-01 – 2015-03-31
Keywords先天異常学
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.

Free Research Field

小児血液

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Published: 2016-06-03  

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