Two Americans and a Briton won the 2007 Nobel Prize in medicine yesterday for developing the immensely powerful “knockout” technology, which allows scientists to create animal models of human disease in mice.
The winners, who will share the $1.54 million prize, are Mario R. Capecchi, 70, of the University of Utah in Salt Lake City; Oliver Smithies, 82, of the University of North Carolina in Chapel Hill; and Sir Martin J. Evans, 66, of Cardiff University in Wales.
Other scientists are applying their technology, also known as gene targeting, in a variety of ways, from basic research to the development of new therapies, said the Nobel Committee from the Karolinska Institute in Stockholm that selected the winners.
The knockout technique provided researchers with a superb new tool for finding out what any given gene does. It allows them to genetically engineer a strain of mice with the gene missing, or knocked out, then watch to see what the mice can no longer do.
After the first decoding of the mouse and human genomes in 2001 yielded thousands of new genes of unknown function, knockout mice became a prime source of information for making sense of these novel genes.
Most human genes can also be studied in this way through their counterpart genes in the mouse. Mice have been likened to pocket-size humans, because they have the same organs and their genes are about 95 percent identical in sequence. Scientists have developed more than 500 mouse models of human ailments, including those affecting the heart and central nervous system, as well as diabetes, cancer and cystic fibrosis.
Knockout mice are so important in medical research that thousands of strains are kept available in institutions like the Jackson Laboratory in Bar Harbor, Me.
“The technique is revolutionary and has completely changed the way we use the mouse to study the function of genes,” said Dr. Richard P. Woychik, the lab”s director. “When people come across a novel human gene, one of the first things they think about is knocking it out in a mouse.”
The three laureates, who are friends but work independently, also shared a Lasker Award in 2001. They began their work in the 1980s, and the first reports that the technology could generate gene-targeted mice were published in 1989. The reports involved a rare inherited human disease, the Lesch-Nyhan syndrome, in which lack of an enzyme causes fits of self-mutilation.
