Our laboratory is working to develop mouse models of human disease. Experimental mice have been the major in vivo system used to investigate the molecular, cellular and genetic basis of health and disease for over 100 years.
One of the major advances over the last 30 years was the ability to make specific alterations to the mouse genome and produce “genetically modified” (GM) mice. Two new technologies, TALEN and CRISPR (1,2) are revolutionising this process such that creating a single point mutation or multiple gene modifications in mice can be performed in a rapid, cost effective manner.
Genome Wide Association studies and large scale genome sequencing are beginning to identify many genetic alterations associated with human disease. Smaller scale familial studies of heritable diseases are another valuable path to identifying genetic alterations associated with various human conditions. These databases are being mined to isolate potential disease causing genetic alterations.
The advances in our ability to create genetically modified mice and our increase understanding of the genetic basis of disease is being utilised to create novel mouse models of human disease. These animal models will be useful to further understand the aetiology of disease and also to develop therapeutics for the treatment of disease.
Wang,H., Yang,H., Shivalila,C., Dawlaty,M., Cheng,A., Zhang,F. and Jaenisch,R. (2013) One-step generation of mice carrying mutations in multiple genes by CRISPR/Cas-mediated genome engineering. Cell, 2013; 153:910–8.
Sung,Y., Baek,I.-J., Kim,D., Jeon,J., Lee,J., Lee,K., Jeong,D., Kim,J.-S. and Lee,H.-W. (2013) Knockout mice created by TALEN-mediated gene targeting. Nature biotechnology, 2013; 31:23–4.
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