Biomedical research including immunology and stem cell biology has developed greatly because of the evolving technology of gene modification and conventional transplantation methods using the most common experimental laboratory animal, the mouse. To translate promising research findings based on mouse research into clinical medicine, however, we need to clarify whether similar events take place in humans. In the study of hematology and immunology, humanized mice provide a unique and efficient experimental system to evaluate differentiation, function, and interaction of human blood cells or immune components. Here we review the latest experimental findings in the fields of immunology, stem cell biology, and regenerative medicine using humanized mice.