What are knockout mice?

Knockout mice are animal models that have been genetically modified to delete or inactivate a specific gene. The term ‘knockout’ refers to knocking out the functionality of the target gene. These mouse models allow researchers to gain insight into how the loss of the gene affects physiology, behavior and disease development. Observing the characteristics of a knockout phenotype can also help researchers understand human gene expression and genetic diseases. 

Study of gene function

Loss of function is a valuable scientific method to study gene function by comparing the knockout animal model to a wild-type mouse. Sometimes a gene of interest needs to be inactivated at a certain developmental stage or cell type for a defined phenotype of interest to be observed. Conditional knockout models enable the study of gene inactivation in a specific cell type, tissue of interest, or in a temporally controlled manner, such as a defined developmental stage from pups to adult mice.

Human disease research

Knockout mouse models have been used extensively to research human diseases such as cancer, heart disease, diabetes, obesity, arthritis and neurodegenerative diseases, and have formed the basis of many of the advances in human medicine. By creating a knockout mouse model for one of the many genes suspected of contributing to a particular human disease, scientists can investigate what effect knocking out this gene might have on the development of disease over time.