Genetic Engineering Might Help the Blind See Again

Do you have any high hopes for things like CRISPR? Things where there's going to be genetic alterations and they are starting to do some experience. You have a big smile on your face right now so I'll let you talk. Go ahead, tell me what's up. Well, so I'm a geneticist and I'm just down the hall from George Church who's in my department at Harvard and I'm a big believer in CRISPR in the sense that it will revolutionize medicine. Now right now... Can you explain it to people who don't know what it means? So CRISPR is an acronym for basically a system that is from bacteria that they use to kill and destroy the DNA of invading organisms like a virus. But we can now use that system to cut and change our own genomes. It's basically a DNA cutting enzyme that doesn't cut randomly. You can give it a barcode in the form of what's called the RNA molecule that tells where that enzyme will cut in the genome. Let's say you, Jerogan, have a terrible gene that's causing heart disease. We take this CRISPR system, we say here's where you need to go to cut. We can tell the enzyme to go and cut it, put it into your cells, it'll go cut it and destroy that enzyme and delete it. And you can also use it to cut the genome and insert new pieces. So you can both subtract and add DNA at will now, not just randomly, but what's important is you can tell it where to go. And that's the big breakthrough. And they're doing some experiments on human beings. I know there was something that they were doing, I believe, somewhere in Asia, if I remember correctly, I believe it was China, where they had done some manipulation to people to help prevent AIDS. And in the process of doing so, they may have boosted intelligence or the potential for intelligence, which was so convoluted that my puny little brain can't understand the study. I had to go over the same paragraph like four or five times just to try to figure out what the fuck they were saying. Am I making any sense? Yeah, you are. You are. And that was a study that I don't believe it's been published, but it's been reported that he is his name. His last name is he. He took embryos and engineered them to delete CCR5 gene, which is required for HIV to infect cells. Now, that was we can we most of us scientists think that that was reckless for the fact that first of all, HIV isn't a huge risk in China. It's one in a thousand chance of getting HIV. There are plenty of other things that you could do that could be more helpful. Let's say why not mutate what's called PSK9 to prevent heart disease, which would probably have 50 percent to kill the boys, a boy. So anyway, it wasn't the most risk benefit ratio modification. That's one thing. But the other is we don't know what happens when you cut genes in embryos. Does it have changes to the DNA clock? Did it accelerate their aging? Did it mess with other genes? Did it cut in other places and screw up those genes? We don't know that yet. And so that's why the scientific community had a negative reaction to it. But what's interesting is that the scientific community and the press has pretty much gone quiet on this. Imagine if this happened during the Bush era. We'd have protesters all over the place and be outlawed. And that hasn't happened. And I think it's because we live in a world with a 24-hour news cycle. But isn't that also because it's being if it was during the Bush world, I mean, where the protests would really take place if it was done here. The thing about things that are done in China or overseas, like, huh, it's like it's so far away, like, well, let's keep an eye on them. That's true. There is the fear that some countries are going to engineer an army of humans. Superhumans. I mean, we have the technology to do that right now. We believe we understand how to slow aging. There are genes that predispose you to long life. We could make offspring a family that would potentially live a lot longer. But is this something that can only be manipulated in embryos or in fetuses? No. Now we can do it in adults. Actually there are drugs that are in development to actually correct genetic diseases, such as vision loss. Really? Dude, my eyes are gone. I could barely read, like, print on a laptop. I need glasses to read my laptop. All right. So we just put up a study online on a site called Bio Archive. Anyone can go there and see it. Just Google my name and bio archive. B-I-O-R-X-I-V. The reason that's interesting is that what we're showing is in mice at least we can reverse the age of the retina and restore the vision of old mice. What do I have to do? Well, I think you have to blow me a few more times. Hey, come on, man. You're going to lose your job. You got to let me crack those kind of jokes. Sorry. Harvard, I was joking. I started it, folks. Yeah. It's not his fault. So what would someone, I mean, is this going to be available to the general public any time in our lifetime? Possibly. I'm trying my best. We're hoping to do clinical trials starting in two years from now. Really? And what would you do in those clinical trials? So we'd reprogram the eye to be young again. So we now know that there's a set of genes called reprogramming factors, also known as Yamanaka factors that are from named after this Japanese fellow who won the Nobel Prize in 2012. These factors are used all over the world, even probably in high schools, to reprogram skin cells, other cells to be what we call pluripotent stem cells. These are cells that can be used to make new organs or new blood cells. But what people hadn't tried until recently was, can you do this in a living animal or are you just going to mess it up? And what we found out is that if you do it the wrong way, you mess up the animal and it'll die. So what we've shown for the first time in this paper is you can do it in a safe way. And not only that, reverse the clock, make the cells young and restore how they work and get back vision. And what's the methodology? Right, good question. So the current method is using a virus that's on the market. These are called AAVs, adeno-associated viruses. You put them in the eye. There are already patients getting this on the market. Really? Spark Therapeutics is an example of a company that is curing genetic diseases in the eye with viruses. We're in a new world. Most people don't know about it. Wow, so what is the company again, Jamie? Look at this, Spark Genetic Engineering? Spark Therapeutics. Jamie's already got it. Look at that. Bam, motherfucker. Jamie Vernon in the house. So these folks are already doing this to people. So is this for people that are sort of desperate and they'll try something experimental? Right. Well, they're desperate in the sense that there's no other choice, no other cure. I mean, we're now curing genetic diseases. Someone was just treated and cured of sickle cell anemia. That's phenomenal. And I learned that that comes from malaria, right? That was the idea that people were, the resistance to malaria, that trait from people that evolved in the area where they would get malaria was also what led to people getting sickle cell, correct? Correct. I learned that from Tiffany Hausch, by the way. Amazing. Tiffany, shout out to Tiffany. That Lux-Turner stuff, is this something that someone like me could take right now? No, not easily. Your doctor would need to prescribe it. And so if he did prescribe it, I could literally get vision back? Well, this is not the same technology that I'm talking about it from my life. This is inherited retinal diseases, our commitment to our IRDs. This is gene replacement. Not reprogramming the body to be young. But it's the same virus that we'd use to correct aging. So they're using this for certain retinal diseases, or they're correcting it? Now, how is this bacteria fixing your vision? Well, the viruses are just a way to get the genes into the cells. That's all. And these are benign viruses. They don't hurt you. But they're a carrier. And maybe eventually we'll have other ways to do this. But right now, the virus is the best way. And in the mice, to restore the vision, we have this three gene combination of these Yamanaka reprogramming genes. We put them into the eye, and then we turn them on with a drug. In fact, the same drug that I took when I was in Africa called doxycycline is the same drug we can feed to the mice, turns on the reprogramming genes for a few weeks, restores their vision back to a young mouse. And then we just take away the doxycycline and antibiotic, and the mice have their vision back. And how long does it take for it to start deteriorating again? We don't know yet. But we think that it's permanent. Because the age of the cells has gone back. Those are young eyes again. So you might have a whole full cycle from like 20 or 10 to 40 years old again. That's the future, that you'll get a delivery of this virus. You'll take the antibiotic for a few weeks, be fully rejuvenated. And the doctor says, come back in a couple of decades. We'll fix you again. We'll give you some antibiotic in a couple of decades. But then it gets really weird if you engineer your children to have this system. If that ever happens, let's imagine it could. We could do this right now with technology. And you have people engineered to be able to be reversed in their age. Or let's say they have an accident and their optic nerve gets damaged, or they lose their hearing from a bomb or something, a spinal injury. Give them an IV of antibiotics, and they become just like an embryo. They can rejuvenate, they can regrow their optic nerve, regrow their spine, fixed, back like new. The vision thing, do you think that we're going to see that in our lifetimes? I mean, is this something that you're going to see that's going to be available to the general public? Well, so I've got an entrepreneur, as we discussed before. And so one of the companies that I've started is exactly that, raised money to be able to make this virus. We're making it now. It takes a few million bucks. And we'll hopefully, with the FDA's approval, inject it into people's eyes. Now first it won't just be guys like you. First of all, we have to go into an area where it's FDA-approval, which is a disease like glaucoma, which is pressure in the eye, or macular degeneration. That's our first goal. But then if it's safe, why not do old eyes? Wow. That's incredible. Now, what about people with injuries? Yeah. Well, yeah, you could theoretically put it into the spinal cord or give an IV. But people with eye injuries. Oh, for sure. So one of the things we also did in this paper that we've put online is we pinched the optic nerve. And what normally happens is it just degrades. I mean, nerves don't grow back, right? Unless you're a baby mouse or a baby human. But we made those cells so young that the optic nerve grew back to the brain. Wow. The first time that's been able to happen. I know a guy from fighting. He's got a detached retina, detached so bad that his vision and his right eyes, extremely poor. Shout out to Michael Bisping. Do you think that that's something that inside of his lifetime, they could see something, use of this technology that could regenerate his eye? Well, I get a lot of emails. I'm not really trying to overpromise anything. What I think is possible is that initially it'll be used for disease, a chronic disease. Then it'll be used for injuries like that, but fresh injuries. I think it's probably likely to work better if it's fresh. I don't know where this technology is going. I can imagine a lot. We can all imagine that you could get vision back and people walking again. But that's where this technology is going. We only... So I described the discovery in the book as actually what happened while I was writing the book is we were making these discoveries and they were remarkable. And so I wrote them down in the book as we went along so people can see how it feels to be a scientist to make these discoveries. But it's only been a year or less that we've known about this. So imagine 50 years from now what we can do, even 10. It's going to be a remarkable future. It's very exciting. Now, what kind of a timeline are you anticipating for bringing this to people with injuries? Well, injuries... Already we have a study plan for spinal injury in mice and that we'll probably know the results in less than a year. And then we could, as fast as the FDA allows us, go into a clinical trial. Now is the same scenario applicable for people with spinal injuries as vision, like people that have a more recent spinal injury will be more likely candidates than people that have had older spinal injuries? I think so. That would just be my guess that it's easier to fix a recently damaged system, anything in the body that's fresh. But I wouldn't rule out anything. When we first discovered this, the experiment was to have a fresh injury, the pinching of the optic nerve. But then I said to my student, why don't you just try old mice? And he said, come on, old mice, are you kidding me? How's that going to work? Just try it. Just try it. So he did it in collaboration with another lab at Harvard. So they're the experts. And so Bruce Cassandra is his name. So Bruce called me, a professor at Harvard, and it's 10.30 at night. I just got off a plane. He said, David, you won't believe it. I didn't believe it. I just looked at the data. It freaking worked. Old mice are seeing again. He said, I want to go down to the FDA and tell them about it. Because right now, eye disease is typically, all you can do is slow them down. And he is actually a reversal of lost function. Now, does this apply to injuries as well, do you believe, old injuries or just old macular degeneration? We haven't tried old injuries. Now, we've done glaucoma, which is an old injury. So theoretically, what we could do is, at least with the existing nerves, if they're still attached, we should be able to rejuvenate those and make them work better. Because he has some vision in his eye. So, yeah, so that's possible. That makes more sense. But very little, very limited in one eye. Yeah. Well, we'll have to see.