Three Win Nobel for Work on 'Gene Targeting' Three scientists will share the Nobel Prize in Physiology or Medicine for their pioneering work in the fields of "gene targeting" and embryonic stem-cell research. The discoveries, made over the past three decades, laid the groundwork for understanding how genes work.

Three Win Nobel for Work on 'Gene Targeting'

Three Win Nobel for Work on 'Gene Targeting'

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Three scientists will share the Nobel Prize in Physiology or Medicine for their pioneering work in the fields of "gene targeting" and embryonic stem-cell research. The discoveries, made over the past three decades, laid the groundwork for understanding how genes work.


From NPR News, it's ALL THINGS CONSIDERED. I'm Michele Norris.

And today marks the start of the Nobel announcements. And as always, we begin with the Nobel Prize in physiology or medicine. This year, the honors go to Oliver Smithies of the University of North Carolina, Mario Capecchi of the University of Utah and Sir Martin Evans of Cardiff University in Wales. Their work has taught scientists how to tell what a gene is doing and how it might be fixed if it's broken.

NPR's Richard Knox reports.

RICHARD KNOX: Researcher Rick Woychik wonders why it took the Nobel Prize committee so long to reward the work of Smithies, Capecchi and Evans.

RICK WOYCHIK: Their work has absolutely revolutionized the ability of the biomedic research community to understand what genes do.

KNOX: Woychik is director of Jackson Laboratories in Maine, which built its reputation on breeding mice with odd genetic defects. In the old days, before 1989, researchers relied on random mutations to produce a mouse with diabetes or an immune disorder that resembled a human disease. Such accidents of nature are golden for researchers trying to understand the genetics of human diseases. Smithies' big insight was that scientists don't have to wait for nature to create mutations. Now, Woychik says, they can target and control each gene.

WOYCHIK: You can completely turn it off, in which case you've created what is commonly referred to as a knockout.

KNOX: A knockout mouse that's missing only a certain gene.

WOYCHIK: Or you can target the gene in such a way that you can introduce more subtle changes. So it allows us to understand what would happen if - what we call knocked down a gene. But we don't knock it out.

KNOX: This kind of control over genes is especially critical at a time when the human genome has been decoded, along with organisms from yeast to worms and dogs to horses. Smithies, the son of a British insurance salesman and a teacher, says all this new information is useless if you don't know what the genes do.

OLIVER SMITHIES: Just imagine that you don't know what the parts of an automobile are. You know it's got lots of parts. And you start to inactivate one part or another, you can find out what they do. If you happen to knock out the bulb that illuminates the inside of a car, all you've lost is the ability to read your newspaper at night. It's not very important. But if you knock out a wheel, you find that the car will crash.

KNOX: Knock out a crucial gene and the result may be Alzheimer's disease. Scientists all over the world are wielding the gene-targeting tools of Smithies and Capecchi to make drugs that work in new, smarter ways against diseases as diverse as cystic fibrosis, cancer and diabetes. Others are taking up the innovations of today's third Nobelist, Martin Evans. He joined gene targeting to the power of embryonic stem cells. That scales up gene targeting from test tube to living mice. Nobel winner Mario Capecchi thinks the benefits will be profound.

MARIO CAPECCHI: For example, we'll learn how the brain works. And we'll really understand what it means to get information, where do you store it, how do you retrieve it. And once you understand that process, then you'll be able to treat neuropsychiatric diseases much more effectively. Right now, we simply randomly give drugs and see which ones work and which ones don't, and have no idea of what they're doing.

KNOX: Capecchi, who's 70, says he's involved in a project that will take him another 20 years. Smithies, at 82, the most senior of the trio, plans to keep going to the lab every day, including weekends. Whatever he does next, he says the prize is a satisfying capstone.

SMITHIES: It's rather peaceful feeling. It's a feeling of a culmination of a life of science. And actually, my feeling when I heard was a rather sense of peace rather than the excitement.

KNOX: Rather like the peace he feels while piloting his glider through the Carolina skies as he did last Sunday.

Richard Knox, NPR News.

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Americans, Briton Share Nobel for Gene Manipulation

Americans, Briton Share Nobel for Gene Manipulation

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Annika Rohl/Nobel Committee for Physiology or Medicine

Two American scientists and a British researcher share this year's Nobel Prize in Medicine or Physiology for devising the tools to figure out what individual genes do and how to fix them.

Capecchi, 70, who was born in Italy, is at the University of Utah in Salt Lake City. Smithies, 82, born in Britain, is at the University of North Carolina in Chapel Hill. Evans, 66, works at Cardiff University in Wales.

The Nobel Committee says these scientists laid the groundwork for the main work in biology today: figuring out what each of the 30,000 human genes does.

Capecchi invented a way to target single genes and turn them off to see what they do. Smithies used gene targeting to correct defective genes. Evans showed that embryonic stem cells can integrate corrected genes into living animals.

Together, these techniques are a major reason why scientists are so excited about the potential for embryonic stem cell research to cure a wide range of human ailments.

"Gene targeting has pervaded all fields of biomedicine. Its impact on the understanding of gene function and its benefits to mankind will continue to increase over many years to come," said the citation for the $1.54 million prize.

Gene Targeting in Mice

The widely used process has helped scientists use mice to study heart disease, diabetes, cancer, cystic fibrosis and other diseases.

The first mice with genes manipulated in this way were announced in 1989. More than 10,000 different genes in mice have been studied with the technique, the Nobel committee said. That's about half the genes the rodents have.

Steve Brown, director of the mammalian genetics unit at the Medical Research Council in London, said the three researchers have "given us the toolkit to understand how genes function" in mice and so, by extension, in humans. As a result, of their work, he said, "we're on the cusp of having a much better understanding of the relationship between genes and disease."

An Early Morning Call

In a telephone interview from Salt Lake City, Capecchi called the award "a fantastic surprise."

He said he was deep asleep when he got the phone call from the Nobel committee at 3 a.m. local time. "He sounded very serious," Capecchi said, "so the first reaction was, `This must be real."'

Smithies told The Associated Press getting award was "very gratifying." After working on the research for more than 20 years, he said it's "rather enjoyable being recognized at this level."

Smithies said he hopes winning the prize will make it easier to secure funding for other work.

Although gene targeting uses embryonic stem cells from mice, it is different from how stem cells would be used to treat disease in humans. In people, stem cells would be prodded to become replacement tissue like nerve cells for transplant into patients.

Capecchi's work has uncovered the roles of genes involved in organ development in mammals, the committee said. Evans has developed strains of gene-altered mice to study cystic fibrosis, and Smithies has created strains to study such conditions as high blood pressure and heart disease.

The medicine prize was the first of the six prestigious awards to be announced this year. The others are chemistry, physics, literature, peace and economics.

The prizes are handed out every year on Dec. 10, the anniversary of award founder Alfred Nobel's death in 1896.

Last year, the Nobel Prize in medicine went to Americans Andrew Z. Fire and Craig C. Mello for discovering RNA interference, a process that can silence specific genes.

Associated Press contributed to this report.