Mice changing medicine
Professor Steve Brown
Advancing medicine and knowledge through the discovery and investigation of mouse models of human disease
Most human diseases have a significant genetic component - that is changes in the DNA in our genes contribute to the development of the disease. This is true for example for birth defects, diseases of childhood and chronic diseases such as diabetes, obesity, heart disease, stroke, respiratory and psychiatric conditions that affect people of all ages, nationalities and classes. Diseases of ageing such as Alzheimers and other neurodegenerative diseases are also a significant genetic burden on the human population.
In 2001, the human genome project was completed. This project identified all of the genes in the human genome and provided the tools for studying the contribution of genes to disease. The grand challenge that lies ahead for medicine is to identify the function of each and every gene in the human genome, and to determine what diseases arise when a gene goes wrong. Finding the genetic causes for disease has the potential to offer significant advances in medicine from diagnosis to new therapies and treatment.
Shortly after the human genome project was completed, the sequence of the mouse genome was obtained. Although outwardly a mouse looks very different to a human, its development, physiology and biochemistry are remarkably similar to human. Moreover, sequencing of the mouse genome showed that it shares 99% of its genes with human. The mouse has an extensive genetic toolkit that allows us to alter its genes and to determine the consequences in terms of disease. For all these reasons, the mouse has become the most important organism for modelling and studying the genetics of human disease.
MRC Harwell is at the international forefront of the use of mouse genetics to study the relationship between gene and disease. The models we create and study are used to understand the disease processes that occur when a gene goes wrong. The disease models can also be employed for the pre-clinical assessment of new drugs and other therapeutic approaches, before therapies are tried on humans. Eventually, we and others aim to have information about the relationship between gene and disease for every gene in the mouse genome, and by extension every gene in the human genome. This will provide a wealth of data and opportunities for advances in medical understanding and treatment. The national centre for mouse genetics at Harwell is in the vanguard of these developments, providing the research programmes and expertise, allied to state-of-the-art facilities, to advance medicine and knowledge through the discovery and investigation of mouse models of human disease.