Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
. 2017 Oct;118(10):3043-3048.
doi: 10.1002/jcb.26002. Epub 2017 May 15.

Development of Humanized Mice in the Age of Genome Editing

Vishnu Hosur  1 , Benjamin E Low  1 , Cindy Avery  1 , Leonard D Shultz  1 , Michael V Wiles  1
Affiliations

Development of Humanized Mice in the Age of Genome Editing

Vishnu Hosur et al. J Cell Biochem. 2017 Oct.

Abstract

Mice are the most commonly used model organisms to study human disease. Many genetic human diseases can be recapitulated by modifying the mouse genome allowing the testing of existing and novel therapeutics, including combinatorial therapeutics, without putting humans at risk. Specifically, the development of "humanized" mice, that is, severely immunodeficient mice engrafted with functional human hematopoietic and immune cells and tissues, has revolutionized our ability to study and model human diseases in preclinical in vivo systems. Until recently it has been challenging to develop strains of humanized mice with targeted mutations or that transgenically express human genes with site-specific mutations, and can permit optimal growth of functional human cells and tissues. However, recent advances in targeted nuclease-based genetic engineering have enabled precise modification and development of humanized mouse models at an unprecedented pace. These modifications permit optimal growth of functional human cells and tissues and can be used to replicate human genetically determined diseases. J. Cell. Biochem. 118: 3043-3048, 2017. © 2017 Wiley Periodicals, Inc.

Keywords: CRISPR/Cas9; HUMANIZED; IMMUNODEFICIENT; NSG; SITE-SPECIFIC ENDONUCLEASES; TRANSGENIC MICE.

PubMed Disclaimer

Conflict of interest statement

Conflict of interest: There is no conflict of interest to declare.

References

    1. Billerbeck E, Barry WT, Mu K, Dorner M, Rice CM, Ploss A. Development of human CD4+FoxP3+ regulatory T cells in human stem cell factor-, granulocyte-macrophage colony-stimulating factor-, and interleukin-3-expressing NOD-SCID IL2Rgamma(null) humanized mice. Blood. 2011;117:3076–3086. - PMC - PubMed
    1. Bosma GC, Custer RP, Bosma MJ. A severe combined immunodeficiency mutation in the mouse. Nature. 1983;301:527–530. - PubMed
    1. Brehm MA, Racki WJ, Leif J, Burzenski L, Hosur V, Wetmore A, Gott B, Herlihy M, Ignotz R, Dunn R, Shultz LD, Greiner DL. Engraftment of human HSCs in nonirradiated newborn NOD-scid IL2rgamma null mice is enhanced by transgenic expression of membrane-bound human SCF. Blood. 2012;119:2778–2788. - PMC - PubMed
    1. Brehm MA, Wiles MV, Greiner DL, Shultz LD. Generation of improved humanized mouse models for human infectious diseases. Journal of immunological methods. 2014;410:3–17. - PMC - PubMed
    1. Cao X, Shores EW, Hu-Li J, Anver MR, Kelsall BL, Russell SM, Drago J, Noguchi M, Grinberg A, Bloom ET, et al. Defective lymphoid development in mice lacking expression of the common cytokine receptor gamma chain. Immunity. 1995;2:223–238. - PubMed

Publication types

LinkOut - more resources