CRISPR Gene-Editing Experiment Partly Restores Vision In Legally Blind Patients (npr.org) 26
An anonymous reader quotes a report from NPR: Carlene Knight's vision was so bad that she couldn't even maneuver around the call center where she works using her cane. But that's changed as a result of volunteering for a landmark medical experiment. Her vision has improved enough for her to make out doorways, navigate hallways, spot objects and even see colors. Knight is one of seven patients with a rare eye disease who volunteered to let doctors modify their DNA by injecting the revolutionary gene-editing tool CRISPR directly into cells that are still in their bodies. Knight and [another volunteer in the experiment, Michael Kalberer] gave NPR exclusive interviews about their experience. This is the first time researchers worked with CRISPR this way. Earlier experiments had removed cells from patients' bodies, edited them in the lab and then infused the modified cells back into the patients. [...]
CRISPR is already showing promise for treating devastating blood disorders such as sickle cell disease and beta thalassemia. And doctors are trying to use it to treat cancer. But those experiments involve taking cells out of the body, editing them in the lab, and then infusing them back into patients. That's impossible for diseases like [Leber congenital amaurosis, or LCA], because cells from the retina can't be removed and then put back into the eye. So doctors genetically modified a harmless virus to ferry the CRISPR gene editor and infused billions of the modified viruses into the retinas of Knight's left eye and Kalberer's right eye, as well as one eye of five other patients. The procedure was done on only one eye just in case something went wrong. The doctors hope to treat the patients' other eye after the research is complete. Once the CRISPR was inside the cells of the retinas, the hope was that it would cut out the genetic mutation causing the disease, restoring vision by reactivating the dormant cells.
The procedure didn't work for all of the patients, who have been followed for between three and nine months. The reasons it didn't work might have been because their dose was too low or perhaps because their vision was too damaged. But Kalberer, who got the lowest dose, and one volunteer who got a higher dose, began reporting improvement starting at about four to six weeks after the procedure. Knight and one other patient who received a higher dose improved enough to show improvement on a battery of tests that included navigating a maze. For two others, it's too soon to tell. None of the patients have regained normal vision -- far from it. But the improvements are already making a difference to patients, the researchers say. And no significant side effects have occurred. Many more patients will have to be treated and followed for much longer to make sure the treatment is safe and know just how much this might be helping.
CRISPR is already showing promise for treating devastating blood disorders such as sickle cell disease and beta thalassemia. And doctors are trying to use it to treat cancer. But those experiments involve taking cells out of the body, editing them in the lab, and then infusing them back into patients. That's impossible for diseases like [Leber congenital amaurosis, or LCA], because cells from the retina can't be removed and then put back into the eye. So doctors genetically modified a harmless virus to ferry the CRISPR gene editor and infused billions of the modified viruses into the retinas of Knight's left eye and Kalberer's right eye, as well as one eye of five other patients. The procedure was done on only one eye just in case something went wrong. The doctors hope to treat the patients' other eye after the research is complete. Once the CRISPR was inside the cells of the retinas, the hope was that it would cut out the genetic mutation causing the disease, restoring vision by reactivating the dormant cells.
The procedure didn't work for all of the patients, who have been followed for between three and nine months. The reasons it didn't work might have been because their dose was too low or perhaps because their vision was too damaged. But Kalberer, who got the lowest dose, and one volunteer who got a higher dose, began reporting improvement starting at about four to six weeks after the procedure. Knight and one other patient who received a higher dose improved enough to show improvement on a battery of tests that included navigating a maze. For two others, it's too soon to tell. None of the patients have regained normal vision -- far from it. But the improvements are already making a difference to patients, the researchers say. And no significant side effects have occurred. Many more patients will have to be treated and followed for much longer to make sure the treatment is safe and know just how much this might be helping.
Yeah, no, not the first one by a long shot. (Score:5, Interesting)
This guy just casually created a virus to give himself lactose tolerance:
https://www.youtube.com/watch?... [youtube.com]
I've also seen videos of him designing genes from modules in software like it was Lego or electronics.
Example: https://www.youtube.com/watch?... [youtube.com] (Can't verify it's the one I watched right now.)
Yes, we're in the freaking future.
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Yes, we're in the freaking future.
Marveling upon the current technology of the day, anyone could have said that during any period of human history with the same genuine sense of awe.
This guy just casually created a virus to give himself lactose tolerance:
However, some idiot injecting untested gene modifications into his body sounds more like a cyberpunk novel than some Star Trek utopia to me. Unlike crazy sci-fi stories, the unintended consequences are much more likely to be mundane and sad, like cancer, rather than turning him into a super hero or something.
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Marveling upon the current technology of the day, anyone could have said that during any period of human history
From 470 AD to about 700 AD, technology regressed. Knowledge was lost.
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Tech Regression... (Score:2)
Tech regressed during that period, IN EUROPE.
But during that time, the Arabic world and parts of the far east had huge advances.
They considered 'moorish' doctors to be the best.
Re:Yeah, no, not the first one by a long shot. (Score:5, Interesting)
Marveling upon the current technology of the day, anyone could have said that during any period of human history with the same genuine sense of awe.
This is really only true for the last 300 to 500 years or so. Before that, technological progress slow. It isn't that there was no progress, but it occurred much slower, and importantly the dissemination was also slow. For example, it took over a hundred years from the invention of the spinning wheel for it become used throughout Europe. Similarly, while the water wheel systems in 1400 or incredibly sophisticated compared to those of a thousand years before, it is over a thousand years.
As a result, people really weren't that aware that technology changes in the long-term. This is discussed for example in James Gleick's book "Time Travel:A History" for one reason why time travel as an idea only arose pretty late; if one's view one century looks pretty similar to an earlier one, then time travel sounds a lot less interesting. Tech was improving but on a scale that people didn't really notice that much.
It is also worth noting that one reason technology has been improving so much that isn't emphasized that much is that because technology is improving, people are aware that technology can improve, and they can consciously work on it. But that actually undersells the point. A lot of historical cultures attributed major ideas and inventions to either mythological figures or real historical geniuses even when they had nothing to do with it. Examples include Abraham, Imhotep and Archimedes. To some extent, one of the most important realizations of the last few hundred years has been not just that advancement occurs, but that regular people can contribute to advances. The Iowa State Fair has a prize for best agricultural invention in the last year. That sounds reasonable to us, but it is worth realizing how absolutely weird that would sound to our ancestors.
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Very true all af above, specially the difference between before and now.
But you forgot one thing: Also in our current systems innovation is rewarded. In most earlier systems if a normal person came up with an idea, it might have helped them personally to do something better and maybe even few more people, but there was no incentive to try to spread it widely.
Re:Yeah, no, not the first one by a long shot. (Score:4, Interesting)
My great-grandfather came to the US on a sailing ship. He lived through the move to steam-powered ships, expansion of the railroads across the country, telegraph lines being strung across the US, and phones allowing people to talk to each other across distances. But my father related that the one piece of technology he marveled the most about was radio. Someone could be speaking in a studio downtown yet he could here them in the living room 10 miles away, and with no wires attaching the two!
Sub-microscopic logic gates in vast arrays to do computations, viruses engineered to change to DNA within cells - these are all things outside the realm of normal experience, and so, unlike macro mechanical improvements in machines, are marvelous.
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I think the most important tech to technology progression was the Internet.
With the internet, almost instant communication was possible, with new ideas and inventions spreading around the world almost instantly. This allowed collabration of like minded people, regardless of distance to improve on even more tech as well.
It does not matter if you build a better mouse trap if it takes the rest of the world another century before they know about it.
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There were occasional breakthroughs in the older eras, but it's not like the world would know about it all at once, dampening any "explosion".
But yes, mostly it was incremental gains and over eras besides that.
OnTopic: I could see the argument that gene editing could be considered more of a leap ("omg freaking future") than an incremental change. An arguably new field that will hopefully increment in results, consistency, stability, etc.
Re: Yeah, no, not the first one by a long shot. (Score:2)
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I wonder if this kind of thing will force a change in the medical world. Right now it takes a very long time for most stuff to get approved. The testing required is rigorous and expensive.
But now you can edit your own genome with relatively inexpensive equipment and experiment on yourself. If someone figured out how to fix Chronic Fatigue Syndrome I'd be very very interested, even without much in the way of testing. It would be hard to resist and use my better judgement, especially since the alternative is
What does "navigating a maze" mean? (Score:2)
So two patients "improved enough to show improvement on a battery of tests that included navigating a maze".
Did they have humans do a lab rat style maze? I'd love to see that setup.
Probably has to be given the improvements listed: make out doorways, navigate hallways, spot objects and even see colors.
Very cool research. And scary. This is obviously a massive part of the future of medicine.
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Battery of tests (Score:2)
This is great - learning to see takes time (Score:4, Interesting)
From the article:
They're also optimistic that the vision of the patients treated so far could improve with time.
"When you improve the function of the retina, sometimes there's a lag for the brain being able to recognize and use that vision," Pennesi says. "It takes time to learn how to use that improved vision."
I hope that the people being treated recover all their vision over time.
When a part of the brain falls into disuse due to a physical problem like gradual blindness, it gets 'taken over' for other processing. When the physical malformation is corrected, there is still the issue of the person retraining their brain to make sense of the images. This training seems to be a very personal learning process -- a baby takes 3-5 years to see clearly [babycenter.com]
When Jesus restored sight to a blind person [biblehub.com], that person remarked:
And he looked up and said, “I see men like trees, walking.”
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I just hope they improve this stuff enough to repair damaged lenses so that people don't have to wear glasses. Not because it would be great for everyone involved but because some asshole corp has a virtual monopoly on selling glasses and has jacked up the price of glasses to insane levels.
forensic science (Score:1)
That sentence makes no sense (Score:1)
Carlene Knight's vision was so bad that she couldn't even maneuver around the call center where she works using her cane
It sounds like her sense of touch isn't up to scratch either, because you need exactly zero vision to find navigate the world with a cane.
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Do you know if her work environment was set up for people using canes? Canes don't work that well with things that protrude into the space above floor level. See this [access-board.gov] for example.
"legally" blind (Score:2)
What is the difference between a legally blind person and a non-legally blind person?
Are they not blind.
Are there legally quadripledic(sp) people?
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From The American Foundation For The Blind [afb.org]
"Legal blindness" is a definition used by the United States government to determine eligibility for vocational training, rehabilitation, schooling, disability benefits, low vision devices, and tax exemption programs. It's not a functional low vision definition and doesn't tell us very much at all about what a person can and cannot see.
Part 1 of the U.S. definition of legal blindness states this about visual acuity:
A visual acuity of 20/200 or less in the better-seeing eye with best conventional correction (meaning with regular glasses or contact lenses).