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Three Win Nobel for Work on 'Gene Targeting'

MICHELE NORRIS, Host:

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. Transcript provided by NPR, Copyright NPR.

NPR transcripts are created on a rush deadline by an NPR contractor. This text may not be in its final form and may be updated or revised in the future. Accuracy and availability may vary. The authoritative record of NPR’s programming is the audio record.

Since he joined NPR in 2000, Knox has covered a broad range of issues and events in public health, medicine, and science. His reports can be heard on NPR's Morning Edition, All Things Considered, Weekend Edition, Talk of the Nation, and newscasts.