Scientists have been awarded the Nobel Prize for one of the greatest advances in biological science in recent years, and a technique many scientists now make use of daily. This technique is known as gene targeting or, to give it its common name, gene “knockout”.

This development, pioneered by three researchers, Americans Mario Capecchi and Oliver Smithies and their British colleague, Martin Evans, means they now have the ability to replicate human diseases in mice, enabling them to study ‘mouse models’ of many human diseases, including cardiovascular, neuro-degenerative diseases, diabetes and cancer. These models, together with new light being shed on the ageing process and the development of the embryo in the womb, will have a profound influence on medical research. Sir Martin likened the award to winning the world cup.

This advance will effectively mean that the medical research establishment will have a much better understanding of the function of specific genes in pathways in the whole organism and a greater ability to predict whether drugs acting on those pathways are likely to have beneficial effects on the disease.

The Nobel Committee praised the technique as “an immensely powerful technology, which is now being used in virtually all areas of biomedical research”. They also said that as a result of this technique, “Science has gained a greater understanding of how disease can strike otherwise healthy people” and that “Its impact on the understanding of gene function and its benefits to mankind will continue to increase over many years to come”.

One of the most important aspects of the development of this particular procedure means research into genetic conditions such as muscular dystrophy and cystic fibrosis will benefit hugely, as it will enable responsible genetic defects to be identified and corrected in animals, prior to trialling potential treatments on humans.