USA: Stem cell team wins Nobel Prize for medicine

Three researchers who pioneered the creation of "designer mice" to demonstrate the roles of different genes in human development and disease win the 2007 Nobel medicine prize. Three researchers who pioneered the creation of "designer mice" to demonstrate the roles of different genes in human development and disease won the 2007 Nobel medicine prize on Monday (October 8). Mario Capecchi, 70, Martin Evans, 66, and Oliver Smithies, 82, won the prestigious 10 million Swedish crown ($1.54 million) award for helping forge a new and fundamental branch of medicine -- gene targeting. Their work led to the development of mouse "models" of human disease and is widely used to study the function of genes in both disease and in normal biology. It is also a basis for gene therapy -- correcting faulty genes to treat disease. In 2001, the trio took the Albert Lasker Award for Basic Medical Research, seen as the U.S. version of the Nobel since many of its recipients have gone on to become Nobel laureates. Smithies, now of the University of North Carolina and Capecchi of the Howard Hughes Medical Institute and the University of Utah, developed precise methods for changing desired genes one by one. These discoveries led to the development of deleting, or knocking out, genes to discover their function. "What do they do? The knowledge will surely help and no doubt in maybe ten years, maybe five years, maybe in one year, they will improve on the method and it will become old fashioned. But it will have contributed to things in the meantime and I can have my glow for a while," said Smithies speaking at a press conference in Chapel Hill. The Italian-born Capecchi lived as a street urchin and almost starved while his mother was imprisoned in the Nazi concentration camp of Dachau during World War II. He later became a U.S. citizen, as did the British-born Smithies. Evans is British. "In terms of gene content, mouse and human are 99.9% the same so whatever we learn in the mouse is going to be directly applicable to the human," Capecchi explained. Capecchi and Smith's gene-targeting technique that helped scientists create "designer mice" is used by scores of labs across the world and has helped pin down the function of 10,000 different genes. Scientists have used their techniques to genetically modify mice with human conditions such as cancer, heart disease, Alzheimer's disease, cystic fibrosis and high blood pressure. The work has also helped speed the commercial development of drugs. Evans, of Cardiff University, laid the groundwork for making so-called knockout mice when he discovered that days-old embryos are made up of super-powerful cells later dubbed embryonic stem cells. Each one of the cells has the power to give rise to all the cells and tissues in an animal. Speaking in the English university town of Cambridge, Evans explained the significance of their research "The implications are enormous." said Evans "What we can do is - we can change specifically change a designer gene in a mouse which means we can find out what it does. You really don't know what the gene, the genetic inheritance, is doing unless you see it operating in real life, in a mouse in this case. And, all of the information we've had from, for instance, the human genome project, all of the understanding of human genes has now given us a large amount of information and we need to know now what these genes actually do. And the mouse is our model system in which we've been able to find this out in." "One of the things we've worked on is cystic fibrosis and we basically made cystic fibrosis mice. They're not like humans. They don't have the same illness but they have just the same fault in them" explained Evans, "So it's possible therefore to look at means like gene therapy to repair those faults." When asked why he thought he'd won the prize, Evans said it was because he originated the cell research. "The reason I've won is because I was the originator of the cellular side of this work - cell and developmental biology. No, there's a huge amount of work in this in the States. The difference between us and the States at the moment is with the human embryonic stem cells. In this country it is legal to make human stem cells under license from human embryos. In the United States it's not, at the moment." Evans said. President George W. Bush has vetoed two bills that would expand federal funding of such study, although it is actively encouraged in Britain and elsewhere. Medicine is traditionally the first of the Nobel prizes handed out each year.

ITN Source | October 9, 2007

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