Investigation into the molecular mechanisms for the occurrence of leukemia using iPS cells derived from congenital bone marrow failure syndrome and establishment of the methods to prevent its occurrence

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We established iPS cells from somatic cells of a patient with severe congenital neutropenia (SCN), one of congenital bone marrow failure syndrome (SCN-iPS cells). Myeloid cells derived from SCN-iPS cells revealed the maturation arrest at a promyelocyte stage, reflecting the symptom of SCN patients. The gene expression analysis-catenin pathway-related genes, and exogenous Wnt3a induced the improvement in the maturation arrest of SCN-iPS cell-derived myeloid cells. These results indicated the involvement of -catenin pathway in the maturation arrest of myeloid cells in SCN patients and the possibility of novel therapies by activating the pathway. 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.