Humanized mouse models to study human diseases

Purpose of review Update on humanized mouse models and their use in biomedical research. Recent findings The recent description of immunodeficient mice bearing a mutated IL-2 receptor gamma chain (IL2r gamma) facilitated greatly the engraftment and function of human hematolymphoid cells and other cells and tissues. These mice permit the development of human immune systems, including functional T and B cells, following engraftment of hematopoietic stem cells (HSCs). The engrafted functional human immune systems are capable of T and B cell-dependent immune responses, antibody production, antiviral responses, and allograft rejection. Immunodeficient IL2rg null mice also support heightened engraftment of primary human cancers and malignant progenitor cells, permitting in-vivo investigation of pathogenesis and function. In addition, human-specific infectious agents for which animal models were previously unavailable can now be studied in vivo using these new-generation humanized mice. Summary Immunodeficient mice bearing an IL2rg null mutated gene can be engrafted with functional human cells and tissues, including human immune systems, following engraftment with human hematolymphoid cells. These mice are now used as in-vivo models to study human hematopoiesis, immunity, regeneration, stem cell function, cancer, and human-specific infectious agents without putting patients at risk.