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Development of Humanized Mice in the Age of Genome Editing
Author(s) -
Hosur Vishnu,
Low Benjamin E.,
Avery Cindy,
Shultz Leonard D.,
Wiles Michael V.
Publication year - 2017
Publication title -
journal of cellular biochemistry
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.028
H-Index - 165
eISSN - 1097-4644
pISSN - 0730-2312
DOI - 10.1002/jcb.26002
Subject(s) - humanized mouse , biology , computational biology , genome editing , human genome , human disease , genetically engineered , gene targeting , gene , transgene , transcription activator like effector nuclease , genetically modified organism , immune system , genome , genetics
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.