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Humanized mice: A brief overview on their diverse applications in biomedical research

Journal of Cellular Physiology

Published online on


Model animals naturally differ from humans in various respects and results from the former are not directly translatable to the latter. One approach to address this issue is humanized mice that are defined as mice engrafted with functional human cells or tissues. In humanized mice, we can investigate the development and function of human cells or tissues (including their products encoded by human genes) in the in vivo context of a small animal. As such, humanized mouse models have played important roles that cannot be substituted by other animal models in various areas of biomedical research. Although there are obvious limitations in humanized mice and we may need some caution in interpreting the results obtained from them, it is reasonably expected that they will be utilized in increasingly diverse areas of biomedical research, as the technology for preparing humanized mice are rapidly improved. In this review, I will describe the methodology for generating humanized mice and overview their recent applications in various disciplines including immunology, infectious diseases, drug metabolism, and neuroscience. Humanized mice, defined as mice engrafted with functional human cells or tissues, provide an excellent small animal model in which development and function of human cells/tissues can be directly analyzed in vivo. Here I describe the methodology of generating humanized mice and their recent applications in diverse areas of biomedical research.