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Biotech Science

Gene Therapy Restores Sight To Blind 157

Posted by kdawson
from the falling-scales dept.
An anonymous reader writes "Looks like we have found a cure for genetic blindness (clinical trialabstractpaper [PDF] — ABC News video). This gene therapy treatment increases both cone and rod photoreceptor-based vision. These engineered viruses are implanted to do our bidding to restore vision. Clinical trials on 6 children and young people proved the therapy and didn't find any notable side effects." Any blind person, especially any adapted and competent one, who wants to gain the sense of sight would be well advised to study Oliver Sachs's classic piece "To See and Not See."
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Gene Therapy Restores Sight To Blind

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  • um... (Score:2, Funny)

    by fyngyrz (762201)

    Any blind person, especially any adapted and competent one, who wants to gain the sense of sight would be well advised to study Oliver Sachs's classic piece "To See and Not See."

    How?

  • "I see! I see!!" said the blind man, but everyone knew he was full of shit.

    Until now when he CAN actually say it and follow it up with high fives to everyone.

    Every time I get cranky about all the dumb shit that we do in this day and age, I also think about all the cool and fantastic things we can do. It's a funny balance.
    • by KiloByte (825081)

      "'Pazhyviom, uvidim', kak skazal slepoy."

      A couple of cow orkers of mine say this Russian line all the time. Sadly, it doesn't sound as good in English: "we'll live ( = let's wait), we'll see, as said the blind guy".

  • I see said the blind man as he picked up his hammer and saw.
    • You're trying to be funny, but at the Louisiana Center for the Blind, they learn how to build furniture - with unmodified power tools. No accidents, and some really nice pieces. Has been done for YEARS.
  • Myopia (Score:2, Interesting)

    by mxh83 (1607017)

    Could this apply to myopia too? Could it be an option to LASIK?

    • Re:Myopia (Score:4, Informative)

      by dltaylor (7510) on Tuesday May 04, 2010 @03:00AM (#32082436)

      I wish, but ...

      When the rods/cones exist in the retina, and the nervous system connections to the brain, but the photo-chemical pathways inside the rods/cones are blocked, this therapy unblocks the chemical error, letting the other components work.

      For myopics the damage is different. Our eyeballs are not spherical, so the lens and cornea, matched to a spherical retina surface cannot focus incoming light "incorrectly" onto our distorted retinas. our best bet is still prosthetic. Although the cornea can be hacked up to make some correction, it is not really the issue (it is for astigmatism). What we need are lenses designed for non-spherical retinas. This can emulated by glasses/contacts, but the real solution would be corrective lens implants.

      Current materials are not as flexible as natural lenses, so cannot be complete replacements. However, lenses can be shaped for accurate vision at longer than reading distances, or within reading-to-desktop range. As we get older and cataracts appear, there is a stronger justification to replace the lenses, and many older adults no longer have to wear glasses due to replacement lenses. I'm really hoping that by the time I get to replacement, the materials will have been improved so that I can not only stop wearing contacts, but get rid of the reading glasses, too.

      • by Grishnakh (216268)

        For myopics the damage is different. Our eyeballs are not spherical, so the lens and cornea, matched to a spherical retina surface cannot focus incoming light "incorrectly" onto our distorted retinas. our best bet is still prosthetic. Although the cornea can be hacked up to make some correction, it is not really the issue (it is for astigmatism).

        What I want to know is: how did this situation get like this in the first place? Very large numbers of humans have poor eyesight that requires correction. Animals

      • As we get older and cataracts appear,

        I know for a fact, that this has nothing to do with getting old. But with having eaten junk for so long, that you are old.
        But hey, it’s easier to just make up excuses about how this is “normal”. It is not. And I have thousands of patients to prove it.

      • by mcgrew (92797) *

        Current materials are not as flexible as natural lenses, so cannot be complete replacements.

        When you reach middle age, the eye's lens hardens, making reading glasses (or bifocals if you're nearsighted) necessary.

        A new implant came on the market in 2003 when the FDA approved the CrystaLens. Although designed for cataracts, it also corrects myopia, presbyopia, and astigmatism. Most patients get better than 20/25 vision after the surgery, which replaces the eye's natural lens with the artificial lens.

        It sits o

        • Unfortunately, the higher-end lens replacements are still often not covered by insurance. I'm glad you got yours. I wish more people would pay the extra $1900 out of pocket like your journal post mentions.

          Interestingly enough, my sister's eyes were perfectly shaped to need no replacement lens, since any of the less expensive replacements would have given her no advantage over no lens at all. You have to be really myopic for that to work, though, and in her case she's already had retinal reattachment done on

    • Trust me, there's a big difference between a medical cure existing and actually becoming available to the average Joe. It'll be DECADES at least before this is available, to all but a few people who happen to have the perfect profile and know one of the researchers. Laser eye surgery is largely proven and dependable, and available here and now, so you're much better off just going with that.

      • There's also the fact that anything involving injecting genetically engineered viruses into your eyes tends to get a little extra scrutiny.

        Surgery, while ever so slightly barbaric(especially in places that you have to break bones to get to) has the advantage of being mostly predictable. The risks aren't zero, and some people heal better than others; but it is basically moving meat around.

        Genetic modification, even when the germline isn't involved, is less well behaved. Sometimes it works, sometimes ex
      • by Grishnakh (216268)

        Trust me, there's a big difference between a medical cure existing and actually becoming available to the average Joe. It'll be DECADES at least before this is available, to all but a few people who happen to have the perfect profile and know one of the researchers.

        While this may be true for this treatment, I wouldn't say it's true for all treatments. As with anything else, follow the money. Laser eye surgery (which is for an entirely different problem than this genetic treatment anyway) has improved by l

    • by mcgrew (92797) *

      No, myopia has nothing to do with the retina. Good thing for the myopic, as retinal problems can't be fixed by corrective lenses.

      • To be more exact, myopia isn't caused by a fault of the retina (other than perhaps the retina being misshapen along with the rest of the eye), but extreme cases of myopia can be a risk factor for certain problems with the retina. In particular, there's a link between myopia and retinal detachment [google.com].

        That fact still doesn't mean that rod and cone counts have anything to do with myopia. There really is nothing to do with genetic predisposition to low receptors and myopia as far as I've ever heard. The radically

        • by mcgrew (92797) *

          but extreme cases of myopia can be a risk factor for certain problems with the retina. In particular, there's a link between myopia and retinal detachment.

          Yes, my surgeon told me that when I had a detached retina [slashdot.org]. I was EXTREMELY lucky, it didn't cost me any vision, and the surgery got rid of all the "floaters".

  • coloublind (Score:3, Interesting)

    by retech (1228598) on Tuesday May 04, 2010 @01:46AM (#32082214)
    I'd do it for colourblindness.

    "...if only you could see what I've seen with your eyes!"
    • Re: (Score:3, Interesting)

      by MichaelSmith (789609)

      I agree. I can hardly see into the ultraviolet and infared and I would love to be able to see microwaves...

      • Re:coloublind (Score:4, Interesting)

        by Nefarious Wheel (628136) on Tuesday May 04, 2010 @02:16AM (#32082330) Journal
        Interestingly enough, the acrylic lenses used in cataract lens replacement therapy are a bit more transmissive in the infra red than the ones you are born with.

        I haven't noticed much improvement along those lines (I haven't done any empirical studies myself) although my night vision is superb compared with how it was at any time prior to the surgery.

      • Re:coloublind (Score:4, Informative)

        by Plazmid (1132467) on Tuesday May 04, 2010 @02:19AM (#32082340)
        Well if I recall correctly the military tried doing something like this(sans gene therapy) with fighter pilots during WWII. There was a research project to administer fighter pilots a chemical that would make their eyes sensitive to infrared light(night vision infrared not thermal infrared) so they would be better adapted to fighting at night. I don't think much became of it though. Now the only problem with doing this with gene therapy is the effects would be permanent.
        • Re:coloublind (Score:5, Informative)

          by TheLink (130905) on Tuesday May 04, 2010 @02:32AM (#32082378) Journal

          ttp://www.livescience.com/history/090429-military-experiment-1.html

          The U.S. Navy wanted to boost sailors' night vision so they could spot infrared signal lights during World War II. However, infrared wavelengths are normally beyond the sensitivity of human eyes. Scientists knew vitamin A contained part of a specialized light-sensitive molecule in the eye's receptors, and wondered if an alternate form of vitamin A could promote different light sensitivity in the eye. They fed volunteers supplements made from the livers of walleyed pikes, and the volunteers' vision began changing over several months to extend into the infrared region. Such early success went down the drain after other researchers developed an electronic snooperscope to see infrared, and the human study was abandoned. Other nations also played with vitamin A during World War II - Japan fed its pilots a preparation that boosted vitamin A absorption, and saw their night vision improve by 100 percent in some cases.

          • by norpy (1277318)
            I was always told that this was a propaganda campaign fed to ze germans to cover up the fact that we had developed radar to see at night.
            • Re:coloublind (Score:4, Informative)

              by dave420 (699308) on Tuesday May 04, 2010 @05:15AM (#32082892)
              Well, it definitely was in Britain, when the cavity magnetron was put into use in night-fighters. It provided the first centimetric radar, capable of detecting fighters and even breached submarine periscopes, while being small enough to mount in a fighter. To explain the sudden increase in the nocturnal accuracy of the RAF, the old "carrots help you see in the dark" myth was spun, which had the added benefit of encouraging children to eat healthy food.
              • Re: (Score:3, Interesting)

                by Vahokif (1292866)

                which had the added benefit of encouraging children to eat healthy food

                And, incidentally, food you could get with rationing.

        • Re: (Score:3, Interesting)

          by RealErmine (621439)
          Chlorophyll eye drops help night vision: Article here [discovermagazine.com].
      • by retech (1228598)
        I'd love to take the colour markers from Eagle's DNA. They have 10 instead of the pitiful 2 for a human male. No doubt they can see deep into the non visual spectrum.
      • by CarpetShark (865376) on Tuesday May 04, 2010 @04:51AM (#32082824)

        I would love to be able to see microwaves...

        Kitchen tours. $10 a pop. Kids unwelcome.

      • by V!NCENT (1105021)

        Walk through a forest, in pitch black darkness. Take out your cellphone and let the backlight shine into one of your eyes. Literally place your cellphone's screen onto your skin and look around with your left eye still open also (or the other way around).

        I don't know why the hell this effect works, but it's some damn good nightvision! (or some kind of mindtrick that amplifies tiny differences in light).

        Always wondered why that happened.

  • Blindness (Score:5, Interesting)

    by Thanshin (1188877) on Tuesday May 04, 2010 @01:49AM (#32082224)

    Losing sight has always been my greatest fear. I understand a lot of blind people can live perfectly fine lives, but I can't think of many worse futures. (I know the news are about genetic blindness, but still).

    The day someone comes up with a way of completely bypassing the eyes, for example by perfecting the technology of connecting cameras directly to the brain, will feel as important for me as the day someone finds a way of curing all medular wounds.

    It may sound stupid but one of the few reasons I've got for accumulating more money is being able to pay the medicine I hope will exist by the time my body starts failing in those kind of ways.

    • Re: (Score:1, Funny)

      by Anonymous Coward

      Dont be stupid, you dont need to worry about such things!

      God created faith healing for a reason!

      Praise the Lord Hallelujah! Walk the righteous path and go with Jesus for he is your one true savior!

    • Re:Blindness (Score:5, Insightful)

      by Nefarious Wheel (628136) on Tuesday May 04, 2010 @02:21AM (#32082346) Journal

      Losing sight has always been my greatest fear. I understand a lot of blind people can live perfectly fine lives, but I can't think of many worse futures

      Agree wholeheartedly. I was blind for a year, and was cured. Once you lose your sight you would crawl through broken glass if it meant you could get your eyesight back.

      I can see my wife's face, and my daughters are beautiful. Bring on science.

    • Losing sight has always been my greatest fear. I understand a lot of blind people can live perfectly fine lives, but I can't think of many worse futures.

      Interestingly, my biggest worry if I lost sight would be to figure out how to successfully use my synthesizers. I've made music for fun since my teens, and if I had to choose I'd probably rather be blind than deaf. Not that either option is in any way desirable, but still...

    • Losing sight has always been my greatest fear.

      Don't underestimate deafness. Allegedly it's worse than blindness.

    • by ls671 (1122017) *

      > It may sound stupid but one of the few reasons I've got for accumulating more
      > money is being able to pay the medicine I hope will exist by the time my body
      > starts failing in those kind of ways.

      Hehe, your post reminded me the movie "The Island".

      http://www.imdb.com/title/tt0399201/ [imdb.com]

    • Good news, the cure has been discovered for the ailments of old age! This miracle cure, death, is also very affordable. Of course for those of you with extra money, the imminent Japanese camera-vision system for looking up skirts will allow you to meliorate your suffering.
      • by Grishnakh (216268)

        That's not a cure. Sorry for the car analogy, but it's like fixing a broken car by crushing it and just building a new one. There's a large energy cost to this.

        And unlike cars, there's a big difference between humans and simple machines like cars: experience and education are what make us useful. Those things take a lot of time and work to gain. So if you toss out old people and replace them with young people, for them to be as useful, you have to spend a couple of decades raising them to adulthood and

    • by Bob-taro (996889)

      It may sound stupid but one of the few reasons I've got for accumulating more money is being able to pay the medicine I hope will exist by the time my body starts failing in those kind of ways.

      Ahh, youth. I'm not mocking, just reminiscing about that kind of optimism. However, I will point out that socialized medicine could make your investment irrelevant.

  • The clinical trial and abstract are from 2008 and the pdf is of a paper published in 2005... this is 2010.
  • by ^switch (65845) on Tuesday May 04, 2010 @01:51AM (#32082236)

    "All three patients showed a statistically significant increase in visual sensitivity at 30 days after treatment localized to retinal areas that had received the vector."

    Well, one notable side-affect of the virus was improved vision.

  • by ZuchinniOne (1617763) on Tuesday May 04, 2010 @02:00AM (#32082276)

    http://www.wired.com/wiredscience/2009/09/colortherapy/ [wired.com]

    I have a feeling this will be up for a Nobel Prize. It was seriously groundbreaking work and the entire vision science community is excited about it.

    • by Grishnakh (216268)

      Nobel Prizes aren't for seriously groundbreaking work any more. They're for people who talk about serious groundbreaking work, but haven't done anything yet.

  • by EvilDingo (1803734) on Tuesday May 04, 2010 @02:09AM (#32082294)
    My children might have an incurable genetic blindness (we haven't tested them) that causes progressive blindness. After researching a bit, I found that the blind and visually impaired can use computers quite well with screen readers, but there wasn't a lot of accessible software -- especially games. http://www.audio-games.net/ [audio-games.net] was a great resource and helped me design an accessible audio-RPG called Entombed. http://www.blind-games.com/ [blind-games.com] - Full disclaimer: my site. I think the biggest hurdle (obvious from reading some of these comments) is that there isn't a lot of awareness that the blind can navigate and use computers.
    • Have you ever found any MUD clients with built in text-to-speach?
      some things might happen too fast to be spoken.Not sure.

    • by MrCrassic (994046)
      Software like JAWS makes computing for blind people very, very easy. It's just sad that operating systems do not provide the level of accessibility that this program has, especially considering that JAWS itself is quite expensive (unless one gets it from an organization or group at a discounted price).

      There are also braille readers that have internet access and can "display" web pages. I've never played with one of those, but I did play with a braille reader and thought it was pretty neat stuff.
    • I watched blind computer scientist T. V. Raman [wikipedia.org] give a lecture in which he played audio Tetris at the end, although he apparently hadn't known to that point that the visual part of audio Tetris (which existed solely for the benefit of the audience) didn't actually work. This appears to be the Emacs code for audio Tetris [koders.com].
    • by Grishnakh (216268)

      Sorry if this comes off as insensitive, but did you know your children would have this condition before you created them?

      I've known of other people who had genetic illnesses they knew would be passed to their children, causing them to almost certainly die an early death. These people had themselves sterilized (vasectomy) to avoid this, and when they wanted children, they used a sperm donor instead, so that they wouldn't condemn their children to such a fate.

    • My friend, there is no such thing as “incurable”. There is only ”we don’t know how to cure it (yet)”.
      “incurable” is something, that arrogant physicians with a god complex use, because they actually believe that when they themselves can’t cure it, nobody ever in the whole of the universe will be able to. Which for plainly obvious reasons is not the case.

      From what I know about this method (I already read about it, years ago, in scientific publications), we can b

    • by Quirkz (1206400)
      I run a web-based superhero game that's mostly text, with some supporting graphics. I've got several dedicated players from a school for the blind, and a smattering of blind or limited-sight players from other places. They've all expressed delight that my game is very accessible. It wasn't especially intentional on my part, just a side effect of a game that's text-heavy and mostly HTML.

      I also play another game called Kingdom of Loathing which fits a very similar profile. Their setup uses a lot of text and

  • wrong paper (Score:5, Informative)

    by scapermoya (769847) on Tuesday May 04, 2010 @02:15AM (#32082326) Homepage
    after a quick look at the paper linked in the article (Identifying photoreceptors in blind eyes caused by RPE65 mutations: Prerequisite for human gene therapy success), it is clearly not about gene therapy in humans. it is a study of the thickness of the retina in humans homozygous for a mutation in a specific retinal gene. as the title says, it is a prerequisite for gene therapy.

    the actual paper, Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics, can be found here [pnas.org]. It concerns the same gene, incidentally.
  • Oblig... (Score:4, Funny)

    by Psychotic_Wrath (693928) on Tuesday May 04, 2010 @02:17AM (#32082334)
    Nothing to see here move along..... wait a second...
  • I took a high-level bio class at UC Berkeley this past semester that concerned exactly this type of genetic therapy. someone brought up the idea of doing this to normals with the pit viper IR heat-sensitive ionic channel gene, tie it to some downstream color of choice.

    sign me up.
  • by frank_carmody (1551463) <pedrogent AT gmail DOT com> on Tuesday May 04, 2010 @02:37AM (#32082388)
    I have retinitis pigmentosa which affects me in a number of different ways. At the moment it's the night blindness that's the most problematic. But as the disease is a degenerative one and as there's no way to predict (or even give a rough estimate of) the time when I will be fully blind, not a day goes past when I don't think of what it will be like to be completely in the dark. I read these stories all the time and they're all like stories on holographic storage tech: Just 5-10 more years and it'll be here for me to enjoy...
    • Re: (Score:3, Insightful)

      by Anonymous Coward

      Me too. I was diagnosed 25 years ago, with a predicted 10 years before I would be completely blind. The disintegration has actually been a bit slower.

      Lots of things I can't do, but it doesn't hurt, and it really doesn't make life that difficult.

      The only problem I have at all is when I walk in to people by accident. One one occasion, I was accused of being on drugs, and on a couple of occasions (spilling a pint in a pub, for example), people have been pretty rude and wanted to start a fight.

      Honestly, without

  • by 2phar (137027) on Tuesday May 04, 2010 @02:38AM (#32082394)
    As usual, ABC News reaches for the M word. Nothing supernatural.. more like many years of painstaking and brilliant science.
  • At least, superficially related in that it's to do with how the brain interprets visual data, which covers a similar topic to the New Yorker article:

    TED Talk: Pawan Sinha on how brains learn to see. [ted.com]

  • I mean, wow, hasn't restoring sight to the blind been one of the attributes of divine powers? I hope this advance which comes from the ingenuity and intelligence of MAN will help shake the faith of those who believe in such fairytales as the flying spaghetti monster et all. Maybe when we all have hoverboards, walking on water won't seem such a big deal as well.

    • While I'm not remotely theistic, there's rather a difference between the use of tech to accomplish something and that same thing being (allegedly) accomplished without tech. People who are prone to take the bible and other works as, well, holy writ, are just going to say, "Sure, but Jesus did it without a board."

      To give a less charged example, which one of these things is amazing:

      Person jumps out of a plane, falls 10,000 feet, lands with minimal to no injury using a parachute

      - or -

      Person jumps out of a plan

  • by Citizen of Earth (569446) on Tuesday May 04, 2010 @04:30AM (#32082744)

    would be well advised to study Oliver Sachs's classic piece "To See and Not See."

    Yeah, now it's 5:30 in the morning. Thanks a lot.

  • Good news, but... (Score:3, Interesting)

    by vegiVamp (518171) on Tuesday May 04, 2010 @04:38AM (#32082768) Homepage
    Is it just me, or does 6 patients seem rather few for a significant trial ?
    • Re: (Score:2, Informative)

      by Jaydee23 (1741316)
      I seem to remember that this is the kind of size for a first human study. I guess this is to make sure that the patients didn't die / develop cancer / turn into zombies. The more detailed studies will happen, but I think when you get to the human trial stage, the ethical considerations suggest a small group.
    • Re: (Score:3, Informative)

      by thesandtiger (819476)

      Well, it's certainly small for a later stage clinical trial prior to deployment of the treatment, but it's about right for an early trial of efficacy.

      With gene therapy you don't want to just start pumping people full of it - there have been some less than fortunate situations in the past, so limiting the initial trial is a wise choice.

      Now that this demonstrates that there may be some beneficial effect without horrific side effects, they can ramp it up to a larger trial size and go from there in good conscie

    • by sjames (1099)

      It's just you!

      More seriously, it's a fine number of people for a preliminary trial to decide if you're just wasting time or if you might have something worthy of a larger safety or efficacy trial. It also has the advantage that if there is an unforseen problem, at least you minimize the number of affected people. For example, the somewhat infamous TGN1412 [wikipedia.org] trial.

      Now that nothing terrible has happened to the few subjects tested and some positive results were seen, they know that a larger trial is worthwhile a

  • Proved?!?!? (Score:4, Insightful)

    by Secret Rabbit (914973) on Tuesday May 04, 2010 @06:27AM (#32083172) Journal

    Um, it takes a fuck load more than SIX kids to /PROVE/ something. SIX isn't anywhere close to statistical significance, nor does it even remotely demonstrate safety. Proven/proof are VERY big words and shouldn't be thrown around lightly. These preliminary results may be encouraging, but are FAR from proof. Especially, in the medical field.

    • Re:Proved?!?!? (Score:4, Informative)

      by MrCrassic (994046) <deprecated&ema,il> on Tuesday May 04, 2010 @07:08AM (#32083418) Journal
      Kind of troll-like, but it's breakfast time. This is the way clinical research works; it's all normal procedure. First, you test the new drug on mice. After that doesn't yield disastrous results, you go on to test it on a *handful* of people who express the condition pretty severely. After *that* works, then you test on a much larger sample size, and after that works, the drug is practically ready for mass manufacturing and production.
    • by flynt (248848)

      I am a biostatistician.

      I have not read the details of this study, but consider the following example, with included R code so you can replicate it. It is a hypothetical study where 6 subjects are randomly determined to be administered treatment, and 6 subjects are randomly given placebo. All 6 in the treatment arm are cured of blindness. None of the 6 in the placebo arm are. The p-value for Fisher's exact test, which is a *conservative* test (i.e., has lower size than the proclaimed alpha level) yields

  • I'm not a medical student, but from the first sentence:

    Early-onset, severe retinal dystrophy caused by mutations in the gene encoding retinal pigment epithelium–specific 65-kDa protein (RPE65) is associated with poor vision at birth and complete loss of vision in early adulthood.

    Along with their solution:

    We administered to three young adult patients subretinal injections of recombinant adeno-associated virus vector 2/2 expressing RPE65 complementary DNA (cDNA) under the control of a human RPE65 promoter.

    Makes me think that this seems like a highly specific approach and will only work on people who've had damage done on that protein, not general blindness altogether. There are MANY people who are or become blind for genetic or developmental reasons, and it doesn't seem that this work will help them much, if at all. For instance, the only woman who I had a "serious" multi-year relationship with (so far) has aniridia , which is

    • I think the only near universal cure for all kinds of blindness would be direct connection to the brain - and even then, if the optical processing capability of the brain is destroyed/never developed, some people would still not be able to benefit.

      So, as you point out, the fact that the mechanics for blindness differ in many ways, so, too, must the treatments used to correct it, which of course means it'll be highly specific.

  • To be clear, there are multiple forms of blindness. I happen to be stuck with severe myopia and cone dystrophy - so shit's blurry and the daystar messes me up. The therapy described won't fix the myopia, but holy crap that's the closest thing I've heard of to a fix for the cone-rod mess in my life to this point. Mitigating that might not improve my ability to focus, but it might help reduce the strain on my eyes from things like looking around outside, even on cloudy days. A pair of good wraparound sunglass

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