Nobel goes to knock out kings

Two US scientists and their UK collaborator have been awarded the Nobel Prize for medicine for their groundbreaking work in gene technology.

Sir Martin J. Evans

Mario Capecchi, Oliver Smithies and UK scientist Sir Martin Evans developed a technique known as gene targeting enabling them to replicate human diseases in mice by introducing genetic changes into the animal's stem cells.

Their discoveries led to the creation of an immensely powerful technology referred to as gene targeting in mice. It is now being applied to virtually all areas of biomedicine – from basic research to the development of new therapies.

Sir Martin, of the University of Cardiff, has specialised on the inherited disease cystic fibrosis. He said: “It’s wonderful - the sort of thing which when you start off as a youngster in science you look up to these fantastic Nobel Prize winners, and think, could that ever be you? No, of course not.”

Gene targeting is often used to inactivate single genes. Such gene “knockout” experiments have elucidated the roles of numerous genes in embryonic development, adult physiology, aging and disease. To date, more than ten thousand mouse genes - approximately half of the genes in the mammalian genome - have been knocked out. Ongoing international efforts will make “knockout mice” for all genes available within the near future.

With gene targeting it is now possible to produce almost any type of DNA modification in the mouse genome, allowing scientists to establish the roles of individual genes in health and disease. Gene targeting has already produced more than five hundred different mouse models of human disorders, including cardiovascular and neuro-degenerative diseases, diabetes and cancer.

All three scientists will share the prestigious $1.54 million award.