2015's biggest breakthrough could deliver designer babies

GWEN IFILL: It's been called the scientific breakthrough of the year, and a development that could revolutionize medicine. It's all about a way of editing genes, known as CRISPR.

The new method allows scientists to snip out a faulty section of DNA and replace specific genes in living cells. Researchers have long edited genes in the lab, but CRISPR is cheaper, far more precise, and could even be used in DNA found in eggs and sperm.

It has lots of promise, but it has raised many concerns too.

We explore those questions with Jennifer Doudna, a professor at the University of California, Berkeley, who helped develop this breakthrough, and Paul Knoepfler, a biologist and writer from at University of California, Davis.

Welcome to you both.

Jennifer Doudna, I'm going to start by just asking you to define in laywoman's language gene editing and CRISPR.

JENNIFER DOUDNA, University of California, Berkeley: Well, I would say gene editing is a method of making very precise changes to the DNA in a cell, and the CRISPR methodology allows scientists to do that with just really unprecedented precision.

GWEN IFILL: So, there is a targeted, practical use for this?

JENNIFER DOUDNA: Indeed, actually many.

It's a technology that operates in basically all types of cells and allows very precise changes to be made to the genetic code that allows scientists to do things like explore the function of genes and also, in principle, correct mutations that cause disease.

GWEN IFILL: So, give me an example of how this would be applied practically. Blood diseases, for instance? Eyes? What kind of uses?

JENNIFER DOUDNA: Well, I think, in the future, there are very exciting applications to cure diseases in the blood and potentially in the eye and potentially in other kinds of tissues as well.

I think a lot of people are excited about the blood, because it's a tissue where, in principle, at least, we can deliver this kind of technology to cells outside the body, and then put those corrected cells back into a patient.

GWEN IFILL: Paul Knoepfler, I know this is already in use in some labs around the country. Do you find it exciting or do you see it a little bit troubling?

PAUL KNOEPFLER, University of California, Davis: It's actually very exciting. I have been working on gene editing in the lab, in test tubes and in rodents for 15 or 20 years.

And CRISPR really represents, you know, a transformative technology. I would say it — you know, if you put a number on it, it's probably, you know, 10 times better than what we used to do.

And, in a sense, this allows us to do all kinds of new research, but it's almost too good, because I think it's also kind of opening the door to a new frontier of gene editing in human beings.

GWEN IFILL: Let's talk about that new frontier for a moment, Mr. Knoepfler, because it seems to me that, when I think of gene editing, I think of being able to go in and actually change outcomes, change what a baby looks like, change who the baby is, for instance, in the womb.

Is this something that CRISPR would allow, not right now, but at some point?

PAUL KNOEPFLER: You know, it is possible in the future.

I think, right now, the vast majority of us who are working on CRISPR, you know, we really have no interest in making designer babies. We want to advance science through research. And, you know, I think our top priority, if we do go into humans, it's going to be preventing specific genetic diseases.

But I think where we run into trouble is, there is sort of a spectrum of what could be done with this, and there is certainly some proponents of actually enhancing people and going the designer baby route. And that's something that I find very concerning.

So I have been speaking out on that. And I actually just came out with a new book called "GMO Sapiens" where I address some of the concerns here.

GWEN IFILL: Jennifer Doudna, you're nodding in agreement, but you're also aware that the National Institutes of Health have put a moratorium on funding this kind of research. Does that mean that there are widespread concerns about its uses?

JENNIFER DOUDNA: Well, I think there is widespread awareness that this is powerful technology that needs to be employed in a prudent fashion.

As you may know, I have been quite involved in the ethical discussions around this technology. And, in fact, earlier in December of this year, we had a meeting in Washington, D.C., that was sponsored by three different national academies in China, the U.K. and the U.S. to discuss this very issue.

And so this is an area where many scientists and clinicians and obviously other stakeholders are getting involved in the discussion.

GWEN IFILL: And also another stakeholder in this are private companies. Even if the government won't fund the research, what's to stop a private company from forging ahead on this?

JENNIFER DOUDNA: Exactly. I mean, right now, nothing.

So, this is something where, I think, again, the scientific community needs to step forward and really point out what the issues are with the technology that can be employed for the kind of genome editing that could affect human evolution.

GWEN IFILL: Paul Knoepfler, could we be sacrificing accuracy for speed?


I think, you know, CRISPR is much more accurate than the kinds of technology we used in the past, but I think it's not accurate enough right now to actually use it either in humans or for the creation of a new human being, say, with a corrected genetic mutation.

And, so, one of the exciting things about CRISPR is that it's evolving so fast that I think even by, say, the middle of 2016, it's going to be more accurate, it's going to be more powerful than it is today. So, you know, perhaps one of the challenges is going to be, where do we stop and say, you know, it's good enough, it's accurate enough, where we could, say, use it for gene therapy or something like that?

GWEN IFILL: When we're talking about clinical trials to use it for something like that, are we anticipating that could happen relatively soon, Jennifer Doudna?


PAUL KNOEPFLER: Oh, sorry. Go ahead.

JENNIFER DOUDNA: Well, my guess would be, within a couple of years, we will see initial clinical trials. That's what I'm guessing, based on the pace of the field right now.

GWEN IFILL: And, Paul Knoepfler, you agree with that, obviously?

PAUL KNOEPFLER: Yes, I think so.

I think, for gene therapy work, it would be appropriate to go ahead with it in the next few years. Where I'm more concerned is, there is fertility clinics, some philosophers and others who are saying, let's not stop there, let's go ahead and let's try to make better human beings.

And I think we can kind of put better in quotation marks, but who decides what's better? And, you know, if they were trying to make a child who has dodged a bone disease, you know, what if that child is not just healthy, their bones are better than anyone else's in the world, you know?

And so, you know, would you resist the temptation perhaps to get stronger bones, bigger muscles? I guess I feel like it's a tough thing to draw the line between medical application and human enhancement.

GWEN IFILL: Well, then, how do you draw that line? Say you want to be able to find a cure for sickle cell anemia and this gets you a lot closer to it faster.

Jennifer Doudna, what happens if, say, that makes for a better person who has to say to their mate 10 years down the road, 20 years down the road, by the way, I am genetically altered, before we have children?

JENNIFER DOUDNA: Well, yes, I think that's still a bit in the future.

I do think that the CRISPR technology offers a way to discover the function of genes. And one of the things that currently limits the field is just the lack of knowledge about the genetic basis for all sorts of traits, including disease, but other things as well.

And so I think this is where we will see a lot of active work in the immediate future, is really on the research front.

GWEN IFILL: Well, let me assume for a moment that we figure out where these lines should be drawn and this is an unalloyed good in the long run.

I want to ask each of you, finally, Paul Knoepfler, starting with you, how big a leap forward is this?

PAUL KNOEPFLER: I think it's transformative.

I think it's as big as nuclear physics or something like that. I think, you know, one of the most exciting and also frightening aspects of it is that it's going to enable us to literally change ourselves, you know, and kind of hack into the human code.

And so I think, you know, that's unprecedented in history. And so, as we're doing that, as we're changing ourselves, I think, you know, whether that's in five years, 10 years, I do think someone's going to do that. And so, you know, that's very exciting again, but terrifically scary, because, you know, what will we become? What will we become in the future?

GWEN IFILL: Well, Jennifer Doudna, how do we balance that out? This hacking into the human code sounds exciting to a scientist, but might sound scary to a layperson.


Well, again, my feeling all along is has been that I feel, as Paul just said, very, very excited about the potential for this technology to affect human societies in the future in positive ways. But I do think that it's a powerful technology. There's no question. And it needs to be handled with appropriate caution.

GWEN IFILL: Jennifer Doudna, Paul Knoepfler, thank you both very much.