STEVE INSKEEP, HOST:Reearcher are aking if they can cure certain health problem imply by going to thegeneticdefect that caued them.
RACHEL MARTIN, HOST:Thi i a goal of what' called peronalized medicine. That mean tailoring a treatment o preciely to you that if a ingle damaged gene caue you trouble, doctor could fix the gene. A cientit at the Univerity of California in Berkeley developed a tool that may make that poible.
INSKEEP: We're going to hear about thi from NPR cience correpondent Joe Palca. He' been exploring the mind and motivation of cientit and inventor a part of hi project Joe' Big Idea. And Joe, along with hi producer, Rebecca Davi, went to viit thi cientit Jennifer Doudna in Berkeley.
JOE PALCA, BYLINE: For the pat couple year, I've been hearing about thi new tool that cientit have gotten their hand on that' letting them do really intereting thing with gene. And I wanted to meet the people who invented it, o I aid, come on, Rebecca, let' go to Berkeley and viit Jennifer Doudna 'caue he' one of the inventor. And a we're walking acro the campu to Doudna' office, Rebecca potted omething that eemed kind of oddly prophetic.
REBECCA DAVIS, BYLINE: Hey, Joe.
PALCA: What?
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PALCA: You ee, Nobel Laureate get primo parking pot right next to their office. And the reaon I thought thi wa prophetic i there' ome people whipering that Doudna might be in line for a Nobel for thi new tool he' invented.
DAVIS: Well, it' certainly made her a rock tar in the cience world. And if you remembered, Joe, when we got to her office, we realized we were lucky to get any time with her at all.
PALCA: Yeah, yeah. Jut get a load of her chedule.
JENNIFER DOUDNA: I'm going to New York City for the Janen Award ceremony. I'm going to Rockefeller Univerity for a lecture. I'm going to the Univerity of Edinburgh. I'm going to Heidelberg...
PALCA: What' Doudna done that make her uch a hot ticket? Well, actually he wa working on a fairly eoteric ubject - how bacteria fight off viral infection - when he tumbled acro omething that he wan't expecting to find at all. And it' omething that could really have a huge impact on biology. She realized that you could modify what bacteria do to virue and intead ue that to make pecific change in the equence of a pecific gene.
DOUDNA: Ye, ye. You can take it out, or you can change it, or you can add to it.
DAVIS: She' talking about editing, right?
PALCA: Yeah, you can edit the genome. You can make change. And the reaon that' important i that thi ha been a kind of a frutrating time for geneticit becaue the Human Genome Project gave them lot of information about gene. It kind of gave them the book of life.
DOUDNA: The quetion i, what do you do with that information? You have the information. You've got the book, and you can ee there' a word that' incorrect on page 147. But how do I get there and erae that word and fix it, replace it?
PALCA: And now you can.
DAVIS: And o what Jennifer and people like her are thinking i, aha, here' a tool that' going to allow u to really hammer at ome really intractable problem with genetic dieae.
PALCA: Like, what about a family with a child with an inherited genetic diorder like cytic fibroi?
DOUDNA: You could tell couple that you could repair that and repair it uch that a child that, you know, otherwie would grow up having ome very difficult-to-deal-with genetic dieae could be cured.
PALCA: Or he aid you could alo do it for adult dieae, like let' ay you have a blood dieae like ickle cell anemia that' caued by a problem with a ingle gene.
DOUDNA: You can enviion removing blood cell from a patient, doing the editing and then put thoe cell back into the patient.
PALCA: Now, really being able to fix people' gene uing thi tool, that' till a way off. But Jennifer' really confident that it' going to happen.
PALCA: And even before coming up with thi new gene-editing tool, Jennifer Doudna had a pretty tellar cientific career. But there' nothing about her early childhood that would even make you think he wa going to be a cientit omeday.
DAVIS: That' right. She wa born in Wahington, D.C. but moved to Hawaii when he wa 7. And the move wa a bit traumatic. Compared to the native Hawaiian girl, Jennifer really tuck out. And they didn't heitate to let her know.
DOUDNA: They would ay, oh, why i your noe o big, and why are your eye blue, and why i your hair like it i? And, you know, I wa alo taller than mot of the other tudent. So, you know, I jut felt like, gee, I really mut be ome creature (laughter).
DAVIS: You're laughing about it now, but I can imagine a a child, that it would've had an effect of ome ort?
DOUDNA: Well, it wa incredibly - I mean, I jut remember waking up every morning. I really couldn't eat. I had no appetite. I jut had to face going to chool, and I knew I wa going to be in thi ort of omewhat hotile environment all day. And o I jut ort of felt pretty iolated.
DAVIS: So with all that time on her hand when he wan't hanging out with friend, he actually tarted hanging out in librarie and reading book and book about cience and nature and really kind of getting into it. But he never thought about being a cientit herelf, until one day he met thi woman.
DOUDNA: Beautiful, blonde hair, you know, very feminine woman who came to our high chool. And he wa a cientit working at the Cancer Center on Oahu, and he wa talking about how cell go from being normal tiue to becoming cancerou. And I uddenly realized that' what I want to do. I want to be her.
PALCA: And in a way, he did become her. She didn't go into cancer reearch pecifically, but he went into reearch about tudying how cell work and how the tuff inide them work. But now he' added thi new element - modifying how cell work by changing their gene. And there' a cary ide to all thi. I mean, after all, we're talking about tinkering with the very eence of life.
DAVIS: And that really reonated with me when we were out there in California actually becaue I wa reading Mary Shelley' "Frankentein," which i very much a tale of what happen when you tinker with the very eence of life. And in that cae, it went horribly awry. But we talked to Jennifer about thi darker ide of the technology, and he admitted, yeah, he' thought about it, too.
DOUDNA: I have definitely had moment of thinking about, you know, you can't put the genie back in the bottle. Once the dicovery i made, it' out there. And it' a technology that' eay enough to ue that anybody with baic molecular biology training can ue it for genome editing. It' a bit cary. You know, a time goe by, it' more and more clear how powerful a technology it really i. And o I've had moment of - I wouldn't ay cold weat, but (laughter), you know, waking up in the night thinking, wow, that' kind of profound.
PALCA: But it' profound in a way that anybody can grap, and I wa really truck by the way he decribed her on' reaction when he told him about it.
DAVIS: Andrew, that' right.
DOUDNA: Well, mom, doe that mean that you could - could you actually change the gene in a cell? Could you change the DNA equence in - could you change the DNA equence in my cell? And then I realized, wow, he get it.
DAVIS: Which really pleae Jennifer becaue he figure the more people get it and ue it and work with it and think about it, the more really ueful thi new editing tool will become.
PALCA: And one other thing I like about thi tory i how unexpected her dicovery of thi powerful new genome-editing tool wa. I mean, there he wa, tudying how bacteria get the flu eentially. And while it wa intereting and intellectually atifying work, he tarted having thi kind of uncomfortable nagging feeling like maybe her work wa intereting, but maybe it wan't really going to make a difference.
DOUDNA: And honetly more frequently and recently, a I've got a bit older, I gue, you know, and you tart to - I don't know if it' middle-age crii or what it i - but, you know, you tart to think about, what' been the real impact of our work, right? Are we olving any problem in ociety? Are we doing work that' going to make people' live better?
PALCA: And now with thi new tool, he feel he i going to make people' live better. And it all came about becaue he wa tudying how bacteria get the flu.
DOUDNA: For me, thi jut kind of really hammer home the erendipity of cience.
PALCA: That' what I really like about thi. Thi dicovery of omething that i really important, but it wan't exactly what you were looking for.
INSKEEP: That' NPR cience correpondent Joe Palca and producer Rebecca Davi. You can hear more torie in Joe' Big Idea erie at npr.org. And if you know an intereting cientit you think Joe hould peak with, end him a tweet @JoePalca that' P-A-L-C-A.