CRISPR pioneer Jennifer Doudna reflects on Nobel win, calls for 'responsible use' of gene editing technology

Originally published on October 10, 2020.

In October 2020, the Nobel Prize for Chemistry went out to American biochemist Jennifer Doudna and French microbiologist Emmanuelle Charpentier for a discovery that has completely revolutionized biology and biotechnology, opening up incredible possibilities that are just starting to be realized.

It's called CRISPR-Cas9, a powerful and relatively easy-to-use gene editing system that allows scientists to modify the DNA of animals, plants and microorganisms. 

In the eight years since Doudna and Charpentier unveiled their discovery, researchers around the world have been busy exploring its endless possibilities. It has potential for fundamental research in the biology of life, in preventing genetic diseases, in building more resilient agricultural crops, just to name a few. 

Jennifer Doudna spoke with Quirks & Quarks host Bob McDonald shortly after her historic win. Here is part of their conversation.

Was it a shock for you to get that call from Stockholm? 

Well, it was. And I'm embarrassed to tell you that I missed the call. I had gone to bed. I turned my phone off and didn't wake up until right before 3 a.m. when the phone was just starting to buzz like crazy. And I woke up and I picked it up and somebody was asking, would you like to comment on the Nobel? And I thought they were asking about somebody else winning it so I said, well, tell me, I don't know the news, who won it? And they said, you won it! So that was a real shock. 

When you first published your research on CRISPR back in 2012, did you fully appreciate its enormous potential? 

We had a sense right away that we were onto something big. It's one of those things in science where the project started as a curiosity-driven effort to understand a bacterial immune system called CRISPR. But once we understood how an enzyme called Cas9 cuts DNA, and can be programmed to cut DNA at any desired sequence, that was when we recognized that it could be harnessed as a gene-editing tool. 

Were you surprised by how quickly CRISPR became such an important tool in biotechnology?

Yes and no. No because it was already clear that the ability to manipulate DNA to rewrite the code of life would be a powerful way to understand genetics and in the end, to be able to correct disease causing mutations in disease. So many researchers over the years had been working to do this kind of thing, and there were earlier technologies for cleaning and repairing DNA.

With a powerful technology, there's always the potential for misuse. And this is certainly the case for CRISPR.- Jennifer Doudna, University of California Berkeley 

What made CRISPR so unique is that it's easy to use, relative to other things, and made it a tool that could be adopted widely by essentially anybody with a little bit of molecular biology training. And so it was clear that it would be a really, really useful technology. 

But I have to say, I could never have predicted just how widely it would be adopted and how quickly. 

It's certainly being used all over the world in laboratories now. Are there applications or discoveries that have resulted from it that you've been surprised by? 

Maybe surprised isn't quite the right word, but certainly astounded at the ability to use CRISPR already to correct disease-causing mutations.

And a great example of that is sickle cell disease. This is a disease of the blood. It's well known what the cause is — a single mutation, a single letter in the DNA code in a human cell that has gone awry.

And already it's been possible to use CRISPR to make changes to DNA in affected people so that they are essentially cured of their disease. And these results were announced just in the last year or so. That's been extraordinary. And I think it really highlights the potential of CRISPR as well as opportunities to use it for other kinds of genetic disease. 

What about applications outside of medicine? 

I'm very excited about opportunities in agriculture. And personally, I think that's the area where we will see the soonest global impacts of genome editing because it gives science the way to alter the DNA in plants quickly and precisely so that genes can be tweaked or even changed in a larger sense, to give plants the ability to deal with climate change, drought, increased nutritional value, things like that. So I think that's a very exciting area of biology right now. 

There are all these exciting areas, but have there been any applications of CRISPR-Cas9 that have disturbed you? 

There have, definitely. With a powerful technology, there's always the potential for misuse. And this is certainly the case for CRISPR.

I was aware early on that it would be possible to use CRISPR in human embryos to make changes to DNA that become inheritable. And this is, to me, just kind of an astounding technology to think that humans have now the ability to rewrite their own DNA and do it in a way that can pass those changes on to future generations.

How do we manage a tool like that?

I think it's been critical to have an open, transparent discussion about this globally, and that's really been going on for at least five years.

But that did not stop a scientist in China from announcing CRISPR babies that had been edited to protect them from HIV infection. And so that, I think, has galvanized the international community to rally around the desire to ensure responsible use and certainly transparent use of the technology. 

You've been active in calling for regulations to control the use of CRISPR. What are your concerns? 

I think we have to be careful with regulations because where do you draw the line? And so, in my view, I think at least here in the U.S., we have a pretty good framework from a regulatory perspective for use of technologies, including CRISPR. But really, this goes back to the 1970s and the early developments in molecular biology. 

That being said, I do think it's critical to have a particular safety net around the use of CRISPR in human embryos and the human germline. And so that's something where the active involvement of international groups, including the science academies, is so important. 

Do you think CRISPR has reached its full potential yet? 

No, I don't. I think it's still very much a developing technology. We're eight years into it and clearly still many advances happening in the field that are continuing to expand the capabilities of the CRISPR technology. So I don't see any end to that right away.

Q&A has been edited for length and clarity. Produced and written by Amanda Buckiewicz.