Jennifer Doudna, Co-Founder Of CRISPR, On The Future Of Gene Editing

Speaker 1: 00:00 The founder of CRISPR, a technology that can edit DNA, came to San Diego this week. KPBS science and technology reporters. Shalena John Lonnie got a chance to speak with Jennifer Doudna. They talked about the future of gene editing and the ethical concerns surrounding it Speaker 2: 00:17 at the Scripps institution of oceanography on the UC San Diego campus. Biochemist Jennifer Doudna has just arrived. She's sitting in a brightly lit room with the doors that open to the seashore. Well, it's great to be here and to have an opportunity to share the world of CRISPR and genome editing down here in San Diego. Doudna co-discovered CRISPR CAS nine a gene editing tool with her colleague Emmanuel sharpen TA in 2012 in a nutshell, CRISPR is a protein that can go into a cell or tissue in any biological organisms. So plants, animals, humans, and like scissors, cut open a string of DNA. And when that happens, DNA coding can be altered. What type of potential does it have? What I'm excited about is the opportunity to cure genetic diseases. Things like sickle cell anemia or Huntington's disease, potentially in the future, something like cystic fibrosis. And what CRISPR technology does is to provide a strategy for correcting or at least mitigating those disease causing mutations. Speaker 2: 01:18 And that's not a, not a fantasy. It's not, you know, 200 years in the future. It's something that I think over the next decade we will see those kinds of cures coming to fruition. This past July, doctors for the first time in the United States officially use CRISPR to treat a patient with sickle cell anemia, a disease that creates to foreign blood cells and can cause a shortened lifespan as well as some painful conditions. The doctors use CRISPR to give the patient her own but modified blood cells and she's now being monitored. But while examples like these show promise, some ethicists have raised questions, especially since CRISPR is widely deployed around the world. What do you have to say about some of the potential negative side effects of this? Well, you know, I think anytime there's a powerful technology that comes along, it, it, it often comes with both the opportunities to, you know, create great value and benefit to society, but also risk. Speaker 2: 02:11 For example, being able to change the DNA in developing humans in the germline that would create changes to DNA that affect not only an individual but also can be inherited by future generations. So that's something that I've been working on for several years with my colleagues to educate people about that possibility and to really a welcome it, a global discussion about how to appropriately regulate this technology. As simple Google search of gene editing brings up stories on the potential like genetically modified crops that can resist climate change. But these stories exist alongside headlines on designer babies and super soldiers for the military. A new Netflix series titled unnatural selection considers these scenarios. It also makes it seem like CRISPR technology's fairly easy to access. Doudna Speaker 1: 03:00 says while gene editing is widely available, it still requires biochemical expertise to use. Speaker 2: 03:06 There are a lot of folks who say it could lead to a Frankenstein individual, but obviously that's not the case. I think it's important to separate fact from fiction. Of course, storytellers love to, uh, you know, scare us and, and, and bring up ideas that are sort of fantastical. And, and I think that's, that's true for this Netflix series. But I think that it's important for people to understand that, you know, those of us that are actually working in the field appreciate that this technology has tremendous positive potential. Speaker 1: 03:38 Donna says there's a lot of next steps with this technology, but for now she's working on a genetic research nonprofit in the San Francisco Bay area. The goal she says is to take practical biomedical ideas off the ground and make sure gene editing is equitable. Speaker 2: 03:53 I don't personally want to create a, a cure for genetic disease that's only affordable by the 0.001%. Speaker 1: 04:00 Downness has funding for science for the sake of curiosity is a huge part of making sure the research can happen. Speaker 2: 04:05 Why should the public support, uh, you know, curiosity-driven scientific research. And the reason is that that's how science is, is that we don't know where it's going in the future. And every now and then you, you know, you turn over a proverbial rock and you find something that you couldn't have imagined was there. And that's true for CRISPR. Doudna Speaker 1: 04:23 the recipient of this year's Nierenberg prize for science in the public interest in the Scripps institution of oceanography. Joining us now is KPBS science and technology reporters. Shalina chat, Lonnie Shelina. Welcome. Hey Jay, thanks for having me. So look, most people might have heard about CRISPR after a story that had to deal with a Chinese scientist who last fall announced he created twin girls from an embryo whose DNA was edited using CRISPR. Does Jennifer Doudna believe there's no use for gene editing in human embryos? So she thinks it's kind of a [inaudible] gray area. There are obviously some ethical implications when it comes to editing embryos and potentially creating what some people call designer babies. But she thinks that there's a lot of great outcomes that can come from this technology, like being able to catch a genetic mutation that could cause debilitating life conditions and stop it before it develops. Speaker 1: 05:19 So she thinks it's a gray area basically. Um, and it's going to take a very, uh, educated and, uh, scientific community that's working together to figure out what's the right move. You mentioned that term designer baby. Explain that. Yeah. So this is kind of a PSI Phi type of idea that the idea that, you know, parents when they, uh, are, are when they have an embryo, they can decide what kinds of traits they want the baby to emulate. Um, a, aS a smartness or a, you know, a certain way of looking, um, that can be modified in your genetic coding from very early on. And that's not happened. So not there yet, not there yet, but there's some implications. There are, there are some very serious implications. And dr Doudna actually had a very interesting interview with the guardian not too long ago where she was talking about being in a dream state and having this nightmare where Hitler actually came to her in the dream and said, so what, tell me about this gene editing thing. Speaker 1: 06:29 What can it do? And so she, you know, she woke up with a startle from that. And that's the kind of demonstrates the implications around this technology, which is that we could run the risk of creating a people, um, that have certain traits and eliminating others. But that's, that's very far into a future potentially. Yeah. Sounds like you could take a dark turn. [inaudible] the concern that does some scientists have, what are some other examples of how CRISPR is being used to help cure diseases? Yeah, so CRISPR in the last few months has actually started to be used in clinical trials involving human beings. So this July for the first time officially in the United States, doctors used CRISPR to treat a patient with sickle cell anemia. And so she's still being monitored. But basically what they did was take her own red blood cells. So with sickle cell anemia, it's a condition where you have deformed blood cells and some of them die and it creates a lot of debilitating life conditions. Speaker 1: 07:35 So they took her cells and they modified them using CRISPR and gave it back to her. And, uh, there were no major side effects and now they're monitoring her condition. The fact that there weren't any major side effects is a good sign in and of itself. And a recent trial and China used sales modified with CRISPR in an attempt to cure a patient's HIV. What can you tell us about the results of that study? Yeah, so this was a pretty big study because it basically showed that in the one patient where there was modified cells from bone marrow being transplanted into the patient, um, that there were no major side effects. And so it showed that CRISPR is a viable technology that can be used to treat patients with diseases like cancer. Um, but there were also mixed results because the, the scientists in this study wanted to originally treat five patients, but when they were developing these modified cells in the lab, they were only able to change 18% of the cells that they collected from a bone marrow donor. Speaker 1: 08:38 So it shows that this is still kind of in the works, but for the one patient that was able to receive the cells, his leukemia has been in remission for 19 months and counting I was so no major side effects were detected. But what did Jennifer down and a half to say about whether this is something scientists should continue to pursue given the ethical implications we mentioned before, she absolutely thinks that this is something that scientists should be pursuing all around the world because of the incredible impacts it could potentially have to cure, uh, diseases, genetic disorders that, you know, especially single gene, gene related disorders that are, uh, that would be easier to cure. But she also thinks that there are some potential negative side effects that are going to require a lot of public education to, to handle. Um, so she thinks it's kind of an ongoing process. Speaker 1: 09:37 What is her role in actively combating the unintended consequences of the tool? So she is currently a professor at UC Berkeley and is involved in a number of, uh, different nonprofits. And she has given many talks about the ethical implications. She has a Ted talk. Um, so she and her colleagues are continuously kind of bringing up the, these ethical sides of the question. And, and nearly every interview that I've seen her in this question of, you know, what about, uh, the, the ethics of this. She has answered and says, you know, that yeah, there could be negative side effects of this. And she says, you know, with any big technology, there are great benefits that can come to society, but there are also risks when might we know if any of the trials using CRISPR are actually working. Yeah. So it's still in the very early stages. Speaker 1: 10:31 And I th, and I'll go back to the study, uh, that happened in China. You know, the patient has been his condition. Leukemia has been in remission for 19 months, but cancer is one of those things that can come back. And so it's hard to say with any, you know, genetic disease, whether something is completely cured or with a disease like cancer, whether it's completely cured. So it's kind of a, you know, the trials are happening now, and it'll take time to figure out whether the, the technology works. I've been speaking with KPBS science and technology reporters. Shalina chaat Lonnie Shalina. Thank you. Thank you. Speaker 3: 11:12 [inaudible].