DEBORAH AMOS, host:
It's Nobel season, and the prize in physiology or medicine - yes, that's what it's official called - has been awarded to developers of a technique known gene targeting.
With us now is NPR's health correspondent, Richard Knox. Good morning.
RICHARD KNOX: Good morning.
AMOS: Tell us about the winners. Who are they?
KNOX: They are three guys: Mario Capecchi - who's 70 of University of Utah -Oliver Smithies - 82-year-old researcher at the University of North Carolina -and Sir Martin Evans, 66, who's at Cardiff University in Wales. And they will…
AMOS: They're babies.
KNOX: Yeah. They will share the $1.5 million Nobel Prize this year.
AMOS: Explain what exactly is gene targeting.
KNOX: Gene targeting is trying to find out what a particular gene does. There are, say, about 30,000 genes in the human genome at the heart of every human cell, and we know very little. We're only just beginning to find out what particular genes do. And that's sort of the main agenda item of the scientific, biological enterprise these days, is what do all these genes do and how do they work together in health and disease?
AMOS: And how exactly does that affect medical research? Are there practical implications for this?
KNOX: The practical implications are already beginning to be very broad, and everybody expects of just get - go on and on in many different fields of biology. For instance, these fellows, by showing that you can target genes, you can knock one of the genes out and see what happens to the animal and thereby understand much more about the disease - the contribution that that gene makes.
They've also figured out how to make models of human diseases, diseases that, say, laboratory rats don't generally get, but humans do. And things like everything from mad cow disease to Alzheimer's disease to cystic fibrosis and muscular dystrophy and diabetes. There are many, many models, now, in animals of diseases that are resulting from these gene knockout and gene targeting technology.
AMOS: Talk a little bit, if you would, about the specific human diseases that gene targeting has actually been able to be applied to.
KNOX: Well, I think, that probably the clearest one so far is cystic fibrosis, which was the first model that one of these researcher's devised. Because now we know an awful lot - scientists know an awful lot about what exactly a missing gene does to cause the various aspects of that terrible disease. And doctors are putting that understanding to work in devising drugs to counter the effects of the genes and working on - although not yet succeeding - in doing gene therapy to correct the underlying gene disease.
AMOS: And, if you would, get into the heads of the Nobel Prize Committee. Why this? Why this particular piece of science?
KNOX: I think it's a pretty typical Nobel in a sense that the Nobel Committee wants to recognize things that are really seminal, that lay the foundation for a lot of other scientists to go - to take it forward. And that's what these techniques do. We hear a lot about embryonic stem cell research today. Well, this work is connected with that. This work - these scientists show that you can take embryonic stem cells, manipulate the genes and grill up an animal that is cured of the genetic disease. We're a long way from doing that with humans, many obstacles of all kinds, but that's what the excitement is about in medicine right now, and that's why these guys are right at the - at the ground floor of it.
AMOS: Thank you very much. NPR's health correspondent, Richard Knox.
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