| Budget Amount *help |
¥4,420,000 (Direct Cost: ¥3,400,000、Indirect Cost: ¥1,020,000)
Fiscal Year 2011: ¥1,170,000 (Direct Cost: ¥900,000、Indirect Cost: ¥270,000)
Fiscal Year 2010: ¥1,560,000 (Direct Cost: ¥1,200,000、Indirect Cost: ¥360,000)
Fiscal Year 2009: ¥1,690,000 (Direct Cost: ¥1,300,000、Indirect Cost: ¥390,000)
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| Research Abstract |
In an attempt to elucidate the pathogenesis of congenital marrow failure syndromes, we established a neutrophil differentiation system from human iPS cells. To evaluate this system as a tool to investigate pathogenesis of Shwachman-Diamond syndrome, we generate iPS cells from a patient with severe congenital neutropenia caused by HAX-1 gene mutation, and differentiate neutrophils from these iPS cells. The proportion of mature neutrophils was remarkably decreased by differentiation from HAX1-iPS cells compared to iPS cells derived from a healthy donor. Our differentiation system using patient-derived iPS cells recapitulates the phenotype of the patient with severe congenital neutropenia. These results suggest our model is useful to investigate the pathogenesis of bone marrow failure syndromes with neutropenia including Shwachman-Diamond syndrome.
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