Jennifer Doudna谈CRISPR现状：尚未治愈任何疾病

This is where you accepted the Nobel? Yes. Like right here. Right Here. Tell me about that moment. Well, it was in the heart of the pandemic, and normally it's done in Stockholm, Sweden, and so the Nobel Committee said, well, we're going to send the Nobel to you, and we're going to give it to you in your garden. When you wake up the next day, does the world just open up, or do you feel a weight of responsibility? If I'm honest, I mean, it was so overwhelming.

I didn't go into science with any thoughts of winning prizes. Believe me. You know, I just hoped I could get a job someday. My husband said to me, well, now your main job is being an ambassador for science. And I thought, how do I do that? Being the face of a scientific revolution is a lot of pressure. Even for Jennifer Doudna, a legend in biology who helped turn CRISPR into a breakthrough method for altering DNA, tackling everything from genetic disease to climate change and spawning dozens of companies in the process.

But some critics claim the tech hasn't lived up to its hype just yet. Not to mention the ethical concerns. At the same time, movie depictions of biotechnology haven't exactly been great PR for gene editing. More human than human is our motto. It didn't matter how much I lied, my real resume was in my cells. So to get to the real story, I went to Berkeley, California to meet with Professor Doudna to talk about where CRISPR is actually headed.

I do have a sense that over the next three years, we're going to see some continued breakthroughs To parse through fact versus fiction in the AI race. I'm not seeing chatbots in our own experience innovating And to gauge how excited or worried we should be. So do you feel a sense of urgency? Oh, yeah. Every day. Yeah. So this is your famous garden? Well, such as it is, yeah. How much time do you spend back here? Not enough is the answer.

But I love being in my garden, Emily, because it's a way I refresh my mind. It's amazing how when I come up here and I'm, even if I'm just pulling weeds, I feel connected to nature in a way that I find very relaxing. Doudna's Nobel origin story began in the rainy town of Hilo, Hawaii where her family moved when she was seven. What inspired you there when you were growing up? Well, let's just start with, nobody in my family was a scientist.

I haven't found a single scientist on either side of my family, but I remember how fascinated I was with just the beauty of the place, and I think that was one of the things that first got me interested in science, was thinking about why, you know, different plants and animals were able to survive there and thrive there. When she's not untangling life's biggest questions in her backyard, Doudna is teaching, running a lab, mentoring young scientists and advising startups looking to turn her life's work into real world cures.

Today, she took us inside the Innovative Genomics Institute, a research hub she founded that's part breakthrough lab, part startup incubator. It's fun, right? Yeah. I still kind of get chills when I come in the lab. Yeah. You know, I, I love the feel of it. I mean, I still remember my first time going into a research lab and feeling this sense of wonder, you know, kind of the sense of discoveries are being made here and wanting to be part of it.

I can't believe I never realized this, but it was pointed out to me that your name literally spells do you DNA. I mean, that has to be fate. It has to be fate, right? I had to work on this. So what kind of research is happening right now? Well, we've got all kinds of work going on. We're doing some very fundamental science trying to figure out the original ancestral origins of Crispr in bacteria, which is where it comes from.

I'm really excited about all the students and and postdoctoral trainees that are here that are doing really creative, interesting science. I can't tell you how many times they come up with ideas that I would've never thought of. So recently, a person in my lab figured out that he could extend the efficiency of Crispr in a whole organism. He's doing experiments in a mouse model of disease by simply accelerating the pace at which Crispr molecules are able to edit cells.

Wait, let's pause here for a moment. Crispr, we've been talking about it, but we should probably explain exactly what it is and how it works. Right. Okay. The discovery began with Doudna and her collaborator, Emmanuelle Charpentier and their obsession with the remarkably adaptive immune system inside bacteria. When a virus attacks a bacterium, the defending cell saves a fragment of the viral DNA and turns it into guide RNA, which is like a tiny genetic GPS.

If the virus returns a protein called CAS-9, kind of like molecular scissors pairs with the RNA GPS then locates and cuts the invading virus. The breakthrough from Doudna and Charpentier was realizing this guide, RNA GPS, could be reprogrammed directing CAS-9 scissors to cut genomic sequences beyond viral DNA, including human cells. Suddenly editing DNA was no longer theoretical. And over time, Doudna launched a bevy of biotech companies out of IGI, all working in different ways to translate Crispr technology into treatments for patients.

Give us a sense of the scope of, you know, the number of companies and new ventures that have come out of IGI. So, from the very beginning of the institute, one of our goals was to ensure that we had a pretty smooth pipeline between fundamental discoveries and the kind of organizations like startup companies that would be able to build real world value around those discoveries. I think we've now spun out 31 companies at last count, $9 billion valuation.

If you add it all up, over 2,500 jobs created largely here in California, but elsewhere too. It's, it's really extraordinary. Long before founding IGI, Doudna spent years studying RNA at Harvard and Yale, but it wasn't until 2006 at UC Berkeley that she turned her attention to Crispr. Were there nights that you couldn't sleep because you felt you were close to something? Oh, yeah. It had that kind of detective story feel to it.

You know, when you're hot on the trail of something, you know that it's very exciting, can't wait to do the next experiment. Crispr was very much that way. What are the current use cases that inspire you the most? Well, I always think about Victoria Gray, who was the first patient with sickle cell disease to be treated with Crispr here in the United States. Her story is so inspiring. Victoria Gray, an activist and lecturer is the first person with sickle cell disease to receive an experimental treatment using Crispr.

I want to reach others who feel like no help is coming. We're coming. Do you have patients reaching out to you all the time? All the time. So you're getting a lot of calls? We do, yeah. I'll just say that I, I always thank people for reaching out because they're often sharing very personal stories. They're taught, a lot of them are about children, you know, their kids. A lot of people will send pictures of their children.

It really brings home the importance of what we're doing, and frankly, the need to work as fast as possible on real therapies that can benefit people. So do you feel a sense of urgency? Oh, yeah. Every day? Yeah. Yeah. Because every day that passes, you could be coming up with a miracle cure or saving someone's child. Or just realizing that there's still a big divide between the kind of work that goes on in a lab like this where we're making fundamental discoveries and actually taking that knowledge and having it benefit people.

In a real sense, there's a big gap there. So far, Crispr therapies like those for sickle cell have been built to treat entire groups of patients with the same genetic disease. But in 2025, a new milestone was achieved at the Children's Hospital of Philadelphia, when an infant known as baby KJ became the first patient to receive a fully personalized Crispr based gene therapy. He was born on Thursday. Friday morn

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