Establishment of a basis for therapeutic treatment of chronic granulomatous disease using human induced pluripotent stem cells

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We tried to model chronic granulomatous disease using disease-specific induced pluripotent stem cells (iPS cells). At first, we generated patient specific iPS cells from T cells in 4 patients' peripheral blood and CYBB gene knockout iPS cells by using genome editing technology. The CYBB knockout iPS cells could be differentiated into neutrophil that cannot generate reactive oxygen species. However, macrophage or dendritic cells differentiated from CYBB gene knockout iPS cells in this study did not exhibit any significant abnormality. With consideration for the possibility that the origin of iPS cells can affect results of disease modeling, a system for generating iPS cells from various blood cells was established. Finally, a system for homologous recombination in iPS cells using adeno-associated virus was also established. These experimental systems could be a platform for a therapeutic treatment of chronic granulomatous disease.