Humanized Mouse Models of Clinical Disease

UMMS Affiliation

Program in Molecular Medicine; Diabetes Center of Excellence

Publication Date


Document Type



Animal Experimentation and Research | Immunology and Infectious Disease | Immunopathology | Investigative Techniques | Pathology


Immunodeficient mice engrafted with functional human cells and tissues, that is, humanized mice, have become increasingly important as small, preclinical animal models for the study of human diseases. Since the description of immunodeficient mice bearing mutations in the IL2 receptor common gamma chain (IL2rgnull) in the early 2000s, investigators have been able to engraft murine recipients with human hematopoietic stem cells that develop into functional human immune systems. These mice can also be engrafted with human tissues such as islets, liver, skin, and most solid and hematologic cancers. Humanized mice are permitting significant progress in studies of human infectious disease, cancer, regenerative medicine, graft-versus-host disease, allergies, and immunity. Ultimately, use of humanized mice may lead to the implementation of truly personalized medicine in the clinic. This review discusses recent progress in the development and use of humanized mice and highlights their utility for the study of human diseases.


allergy, autoimmunity, cancer, humanized mice, immunodeficient mice, infectious disease, regenerative medicine

DOI of Published Version



Annu Rev Pathol. 2017 Jan 24;12:187-215. 10.1146/annurev-pathol-052016-100332. Epub 2016 Dec 5. Link to article on publisher's site

Journal/Book/Conference Title

Annual review of pathology

Related Resources

Link to Article in PubMed

PubMed ID