Use of Genetically Engineered Mice for Research

Abstract

Constructing a unique animal model that closely mimics a surgical disease of interest is an important step for researchers when embarking on a career in basic science research. The mouse is an excellent experimental model for defining human gene function because of the genetic similarity to humans and ease of manipulation by molecular means. Genomic sequencing has demonstrated that of ~30,000 genes in both mice and humans, only 1 % is unique to either species (Doyle et al., Transgenic Res 21(2):327–349, 2012). Despite this similarity, the creation of a significant number of genetically altered mice with the same phenotype can be a difficult task. Nevertheless, the use of genetically engineered mice has been, and continues to be, a powerful tool for studying human disease and developing therapeutics. The use of transgenic and knockout mice for research allows investigators to study gene function, dissect molecular mechanisms, and define cellular pathways underlying disease states. In order to accomplish this, consideration must be given to the pathophysiology of the disease to be modeled. A specific understanding of how the disease develops will affect which animal model will be best suited for one’s research investigations. By manipulating the mouse genome, researchers are able to accelerate the research process, discover novel targets, and make further contributions to various fields of research. The objective of this chapter is to provide an overview of the creation of common mouse models of human disease and to serve as a resource for identifying specific mouse strains that may be used for research investigations.