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2014 Fiscal Year Final Research Report

Establishment of a novel disease model using disease specific iPS cells of congenital neutropenia

Research Project

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Project/Area Number 24591548
Research Category

Grant-in-Aid for Scientific Research (C)

Allocation TypeMulti-year Fund
Section一般
Research Field Pediatrics
Research InstitutionKyoto University

Principal Investigator

WATANABE Kenichiro  京都大学, 医学(系)研究科(研究院), 臨床教授 (20324634)

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)
Project Period (FY) 2012-04-01 – 2015-03-31
Keywords疾患特異的iPS細胞 / 骨髄不全 / 細胞死 / 疾患モデル
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.

Free Research Field

血液学

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Published: 2016-06-03  

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