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Italian Team Cures Wiskott-Aldrich Syndrome With the Help of HIV

New submitter tchernobog writes "An Italian team funded by Telethon and S. Raffaele of Milan, was able to cure six kids affected by lethal genetic diseases (in Italian, English video): the Wiskott-Aldrich syndrome and the metachromatic leukodystrophy. This is the culmination of a project lasted 15 years, and which cost more than 30M €; the researchers published some preliminary results last year in Nature, and are waiting for the results on more patients to submit another. The really interesting part is: they used a mix of advanced genetic techniques to achieve this result. Firstly, the DNA of a defective cell is corrected with a gene assembled in the lab. This procedure has been very dangerous for the past 20 years: that it can even be used is a good achievement alone. Secondly, the corrected DNA is propagated in the patient's body using a stripped-down version of HIV, of which less than 10% of its original genome remains. Might the feared HIV in reality prove to be salvation for some?"

65 of 109 comments (clear)

  1. Italy by Anonymous Coward · · Score: 5, Funny

    Soon after, the Italian government put them in prison on charges of manslaughter for not curing all genetic illnesses when clearly they could have saved lives. http://www.reuters.com/article/2012/10/22/us-italy-earthquake-court-idUSBRE89L0WM20121022

  2. Re:Holy crap by ArcadeMan · · Score: 4, Funny

    I'll be excited when they announce catgirls.

  3. Oblig. XKCD by Anonymous Coward · · Score: 5, Insightful

    http://xkcd.com/938/

    1. Re:Oblig. XKCD by SirGarlon · · Score: 1

      Here's another: http://xkcd.com/54/ OK, the science isn't relevant but the conclusion sure is.

      --
      [Sir Garlon] is the marvellest knight that is now living, for he destroyeth many good knights, for he goeth invisible.
  4. So... by Charliemopps · · Score: 2

    So... they can make a disease now that re-sequences our DNA? Anyone else find that terrifying?

    1. Re:So... by TWX · · Score: 1

      Maybe that terrible James Bond movie with the Korean guy turning into Hugh Grant wasn't as far off-the-mark as we thought it was...

      --
      Do not look into laser with remaining eye.
    2. Re:So... by ColdWetDog · · Score: 5, Insightful

      First of all, it doesn't resequence DNA (if that is really a valid concept). It just ADDS a small bit of DNA into the genome.

      Just like viruses have been doing since, well, since there were viruses. But yes, it's potentially scary. So are nuclear weapons, particle beam accelerators and most politicians.

      Man up here. It's a big, dangerous world.

      And you're not getting out alive.

      --
      Faster! Faster! Faster would be better!
    3. Re:So... by Anonymous Coward · · Score: 1

      If we're choosing deaths could you please point me to the particle beam accelerator que?

    4. Re:So... by Anonymous Coward · · Score: 3, Insightful

      start doing things like extending telomeres, and overiding sections related to aging with this, and you might just get out alive.

    5. Re:So... by Anonymous Coward · · Score: 1

      Gene therapy has been a thing for a while now. This is not new.

      Sure, could there be some Eugenics Wars using viruses to wipe out the weak and leave only the strong? Probably.
      But there has been wars since humans have existed, before, and will continue even when (if) we leave the planet and become a fully developed space society. There will be rebels, space pirates and all those fun things just like always.
      Even in a world where all the necessities of life were met and only luxuries costed any money, and even then that was cheap because we lived in a society where everything was plentiful due to us mining the solar system, there will still be fighting.

      Besides, most attack vectors could likely be defeated by simply wearing a mask. Then there would be water, food, those are considerably more vulnerable, food especially since it is in such a huge open area and it is going to be eaten. Water can be purified pretty easily, could you say the same for a viral-infected vegetable? Greenhouses would become a far larger deal, or possibly underground farms with massive massive metal sheets to prevent surface leakage to the ground below and a hole for the sunlight, or solar. Other things I am too lazy to think about ways to protect.
      Just feel lucky you weren't caught in the first attack, that is all you could really do. (I say first when there has already been many biological attacks over the years from man-made stuff)
      The world would change a lot of gene wars became a thing. It is crazy to even think about how much it would have to change for safety reasons alone.

      But stopping research isn't a good thing either.
      This research could lead to cures to those things as well. Along with fast sequencers and so on.

    6. Re:So... by PRMan · · Score: 2

      First of all, it doesn't resequence DNA (if that is really a valid concept). It just ADDS a small bit of DNA into the genome.

      Just like viruses have been doing since, well, since there were viruses. But yes, it's potentially scary. So are nuclear weapons, particle beam accelerators and most politicians.

      Man up here. It's a big, dangerous world.

      And you're not getting out alive.

      Speak for yourself. I plan on going up in the rapture with Jesus...

      --
      Peter predicted that you would "deliberately forget" creation 2000 years ago...
    7. Re:So... by lister+king+of+smeg · · Score: 1

      as for the food being infected you could irradiate the food killing all of the contagions

      --
      ---Saying gnome 3 is better than windows 8 not so much a compliment as it is damning with light praise.
    8. Re:So... by St.Creed · · Score: 2

      "just" more cancers? That "just" means we don't fully understand the role of telomeres yet. They look like simple TTL counters. However, I have the feeling that, just as with most things in our bodies, they fulfill several different roles at different times or based on environmental triggers. I still think that if the cancers can be controlled, it would one of several very interesting options.

      Biosciences, the final frontier... If I was 18 and had to decide what to study again, I'm very sure it would be something in the biosciences. We're getting ready for one hell of a ride there.

      --
      Therefore, by the (faulty) logic you're using, you're just a cow with a keyboard - osu-neko (2604)
    9. Re:So... by mattack2 · · Score: 1

      Ya can't fool me, that involves RAY-DEE-AY-SHUN, we all dun found out that stuff is bad for ya.

    10. Re:So... by Lotana · · Score: 1

      That is right! Did you know that those ignorant suckers are not aware that light is radiation! Every second they spend outside they get bombarded with it! Fools! Sane people like us stay in basements and only come out at night.

  5. Re:What about the fundementalists. by Anonymous Coward · · Score: 5, Funny

    HIV kills the sinful but cures the sick. There's no inconsistency.

  6. Re:What about the fundementalists. by omnichad · · Score: 2, Insightful

    I hate this misuse of fundamentalists. Call them extremists. Those people are not basing their ideology on the fundamentals. There is no Biblical foundation for believing this way.

  7. Resident Evil by Infiniti2000 · · Score: 2

    I hope this isn't the real-world beginnings of the t-Virus.

    1. Re:Resident Evil by GeekWithAKnife · · Score: 1

      The scary bit is that something of that sort might already have happened somewhere behind closed doors...in 50 - 75 years when documents are declassified you might hear about it. -Unless we have an Edward Snowden moment.

      --
      A 'singular oddity' is an event that cannot be explained and only happens when you are alone.
    2. Re:Resident Evil by krakelohm · · Score: 1

      in 50 - 75 years when documents are declassified you might hear about it. -Unless we have an Edward Snowden moment.

      Or we have a Resident Evil moment...

      --
      You are all a bunch of idots.
    3. Re:Resident Evil by cellocgw · · Score: 2

      Yeah, well, may I speak for legions of geeks who say our idea of a Resident Evil Moment is a night with some wicked hot dark-haired, green-eyed babe who favors tight leather -- and is rather acrobatic.

      --
      https://app.box.com/WitthoftResume Code: https://github.com/cellocgw
  8. Re:What about the fundementalists. by i+kan+reed · · Score: 5, Insightful

    But they claim that they do. And in in the case of religion, if you take out the spurious claims, you're basically left with no beliefs at all.

  9. Re:What about the fundementalists. by Capt.DrumkenBum · · Score: 1

    I just heard last week about HIV being used in cancer treatment as well. It seems like HIV is turning out to be a very useful virus. Interesting that something so destructive can be used for so much good. Perhaps a metaphor for something.

    --
    If I were God, wouldn't I protect my churches from acts of me?
  10. Re: What about the fundementalists. by Anonymous Coward · · Score: 1

    Superstitions aside, this is an amazing Nobel grade development, if peer reviewed and confirmed of course.

  11. Re:What about the fundementalists. by K.+S.+Kyosuke · · Score: 1

    HIV kills the sinful but cures the sick. There's no inconsistency.

    Why don't they call it the XOR virus instead?

    --
    Ezekiel 23:20
  12. "stripped-down" by Amorymeltzer · · Score: 5, Insightful

    There have been a number of these headlines the past few years and they all use the sensationalist headline of "HIV CURES DISEASE" which simply is not true. What is true is that genetic mechanisms that HIV, a lentivirus, uses to engineer cells are being re-purposed for medical benefit. The basic technique has been used in the laboratory for ages but the big headline here is not HIV but genetic engineering. This is as much HIV helping to cure a disease as getting X-rays at the dentist amounts to "DEATH RAY HELPS PREVENT CAVITIES!"

    --
    I live in constant fear of the Coming of the Red Spiders.
    1. Re:"stripped-down" by TheSkepticalOptimist · · Score: 1

      Thanks for clearing that up, I obviously originally thought that the cure was to go down on a few dirty man-whores in a back alley, but I am glad researches found a better way to do it.

      --
      I haven't thought of anything clever to put here, but then again most of you haven't either.
    2. Re:"stripped-down" by bill_mcgonigle · · Score: 4, Insightful

      The basic technique has been used in the laboratory for ages

      Yeah, a friend of mine worked for a private research lab a decade ago and they were curing MS in mice models using an HIV vector, as just SOP (the HIV vector part was already old at that point). BTW, they abandoned that work for something that could pay the bills as they didn't have a business model that could earn enough to pay for the FDA-mandated trials. He tells me this kind of thing happens at labs all over the country and when it's a for-profit lab, they don't publish if they're going to reuse part of the tech in their next endeavor.

      --
      My God, it's Full of Source!
      OUTSIDE_IP=$(dig +short my.ip @outsideip.net)
    3. Re:"stripped-down" by Lotana · · Score: 1

      I am curious: Why HIV in particular? Aren't all viruses work by chaning genetic structure of a cell?

    4. Re:"stripped-down" by bill_mcgonigle · · Score: 1

      Aren't all viruses work by chaning genetic structure of a cell?

      HIV is really good at its job. Check here.

      --
      My God, it's Full of Source!
      OUTSIDE_IP=$(dig +short my.ip @outsideip.net)
  13. Fucking with HIV by fnj · · Score: 1

    What could POSSIBLY go wrong?

    1. Re:Fucking with HIV by The+Rizz · · Score: 4, Funny

      What could POSSIBLY go wrong?

      You could get HIV pregnant.

    2. Re:Fucking with HIV by ciderbrew · · Score: 1

      Kids grows up to be the next Hitler?

    3. Re:Fucking with HIV by PRMan · · Score: 1

      There are plenty of atheists that want to round up and kill all Christians right now. We already have hundreds of candidates...

      --
      Peter predicted that you would "deliberately forget" creation 2000 years ago...
    4. Re:Fucking with HIV by terjeber · · Score: 1

      Nah, only the retarded ones, like you. For the common good you know.

      Or to put it differently, I challenge you to name one.

  14. Science, bitches! by Zaatxe · · Score: 1

    Only science can turn a scourge into a cure!

    --
    So say we all
    1. Re:Science, bitches! by LordLimecat · · Score: 1

      Thats sort of like saying "only math can add these two numbers together".

    2. Re:Science, bitches! by Zaatxe · · Score: 1

      Thats sort of like saying "only math can add these two numbers together".

      Some religious people can argue that faith can also add two numbers together, if you catch my drift...

      --
      So say we all
  15. It's not HIV any more... by Gavin+Scott · · Score: 4, Insightful

    These stories always play off the "we're using scary HIV to cure you" angle, but they're just using components stolen from the HIV virus as tools or building-blocks to make something useful.

    You might as well write a story that portrays bear-skin rugs as scary and dangerous because they were once part of a whole live bear.

    And actually pretty much all of our recombinant DNA tools as well as many drugs like antibiotics are simply ancient things we stole from bacteria and other life forms. Somewhat annoyingly, Nature and 3+ billion years of evolution are still a lot better than we are at inventing things.

    My definition of modern biology that I use to introduce it to computer people is: Hacking into ancient alien computer systems (stochastic digital computers not designed by the mind of man) to look for technology we can steal to cure cancer, solve world hunger, and produce renewable energy as well as whatever else we discover along the way.

    G.

    1. Re:It's not HIV any more... by elfprince13 · · Score: 2

      I understand the science here, and mostly agree with your statement, but your analogy is disingenous. Bear skin rugs are typically capable of neither reproduction nor mutation.

    2. Re:It's not HIV any more... by Gavin+Scott · · Score: 4, Interesting

      True. Depending on the processes involved, the chance that a live intact HIV virus inadvertently makes it through the system into a patient is probably greater than getting home from the rug store to find out that a live bear made it through the rug making process.

      But on the other hand one of the researchers involved (or even TFA) might be able to explain to your satisfaction that the chances of these two different events are actually quite similar due to the methods being employed to produce the synthetic biology product.

      G.

    3. Re:It's not HIV any more... by RJFerret · · Score: 2

      According to the pictures I've seen in Playboy when I'm flipping between articles, it seems bear skin rugs contribute more than 10% toward potential reproductive activities (fireplaces another 10%, amazing what we can turn these destructive forces to). I haven't seen follow ups to know if there's been any distinct mutations as the result of any reproduction due to the bear skin rugs or fires.

  16. Re:What about the fundementalists. by Mitchell314 · · Score: 5, Funny

    We didn't steal fire, we just infringed on the gods' patents.

    --
    I read TFA and all I got was this lousy cookie
  17. P.S. If you find this stuff exciting... by Gavin+Scott · · Score: 4, Interesting

    MITx is offering the second session of their free massively open course 7.00x on Introduction to Biology - The Secret of Life taught by one of the best teachers I have ever listened to, Eric Lander of MIT, which starts on Sept 10th:

    https://www.edx.org/course/mit/7-00x/introduction-biology-secret-life/1014

    This class is mostly about the molecular biology machinery that makes cells work, and it should be fascinating to anyone who finds the way computers work interesting because most of what goes on at the cellular level is actually information processing and digital operations (though based on stochastic principles).

    Warning: this class might make you want to (or wish you could) change your career path...

    G.

    1. Re:P.S. If you find this stuff exciting... by Gavin+Scott · · Score: 1

      "Session" was the wrong term to use. It's the second time they're offering the class, and it's the complete class from the beginning, not a "part 2" as might have been suggested by my poor word choice :)

      G.

  18. Genetic Engineering by zbobet2012 · · Score: 2

    It is here folks, we just did it, and we did it on a living human being. Take a moment to think about what that means.

  19. Re:What about the fundementalists. by wagnerrp · · Score: 2

    It's cool. HIV has been around long enough that that patents on it have all expired.

  20. Re:What about the fundementalists. by LordLimecat · · Score: 1

    I say call them "strawmanists"; there arent really that many people who believe in the strawman GP has constructed.

  21. Re:What about the fundementalists. by PRMan · · Score: 3, Insightful

    And actually, to most Christians, the source of disease would typically be seen as random mutations, which God allowed after the fall. Is it random chance that it affects gays and drug abusers the most? Quite the coincidence, but hard to say. But ultimately, the ability to turn bad around for good is what God specializes in.

    --
    Peter predicted that you would "deliberately forget" creation 2000 years ago...
  22. Re:What about the fundementalists. by interval1066 · · Score: 1

    ...which God allowed after the fall.

    That 'God' has a torture chamber and 'allows' things at all is a sure sign that your making this God thing up, bubbie. Oy!

    --
    Python: 'And then suddenly you have a language which says "we're all stuck with whatever the whiniest coder wants".'
  23. Re:What about the fundementalists. by Anonymous Coward · · Score: 1

    There are enough of them around that, depending on where you live, you could bump into them regularly in public places. There is also enough variation between areas of the US and even within some towns and cities, so that some places you won't bump into any, or they are so far in the minority they won't be outgoing or vocal about things. But then there are places where they are more common and vocal. I've overheard plenty of such conversations on buses, planes and in parks, and have had people try to start conversations with me about such topics. I've at least been approached by them more often than I've passed by a Scientologist information table. I can't say much for absolute numbers, especially since most people of nearly any belief can function without bringing up religion every couple minutes in conversation, but definitely not zero or virtually non-existent.

  24. Lol, can't wait for the advertising disclaimers by TheSkepticalOptimist · · Score: 2

    Warning: This product might give you nausea, dry mouth, or AIDS.

    --
    I haven't thought of anything clever to put here, but then again most of you haven't either.
    1. Re:Lol, can't wait for the advertising disclaimers by iamacat · · Score: 1

      Hmm, for diseases we are talking about AIDS is wastly preferable to the original condition.

  25. and when they have kids by Ralph+Spoilsport · · Score: 1

    they pass on HIV DNA into the human genome? BRILLIANT.

    --
    Shoes for Industry. Shoes for the Dead.
  26. Re:What about the fundementalists. by i+kan+reed · · Score: 1

    This is not in the subset of posts I've made that I consider insightful. This is a case where I posted something I knew to be flamebait because I thought it was funny, but it's not a very good argument. :-/

  27. Re:What about the fundementalists. by bill_mcgonigle · · Score: 1

    you're basically left with no beliefs at all.

    "It's better to have ideas. You can change an idea. Changing a belief is trickier. Life should malleable and progressive; working from idea to idea permits that. Beliefs anchor you to certain points and limit growth; new ideas can't generate. Life becomes stagnant." - the Apostle Rufus

    --
    My God, it's Full of Source!
    OUTSIDE_IP=$(dig +short my.ip @outsideip.net)
  28. Re:What about the fundementalists. by lister+king+of+smeg · · Score: 1

    It's cool. HIV has been around long enough that that patents on it have all expired.

    yeah but its genome would still be covered by copyright

    --
    ---Saying gnome 3 is better than windows 8 not so much a compliment as it is damning with light praise.
  29. Ah, the Rapture. by stoploss · · Score: 1

    Speak for yourself. I plan on going up in the rapture with Jesus...

    Meh, no doubt so did most people present during the monologue of Matthew 16:27-28.

    Ha ha, but clearly the joke was on them because Jesus must have known that the Wandering Jew was standing just behind the disciples. I mean, that's basic Occam's Razor, right?

    Right?

  30. Re: What about the fundementalists. by smaddox · · Score: 1

    I'm not a geneticist, but I'm guessing any retrovirus could be used. HIV is likely used because its been the focus of the most research, and thus the genome is well known and cultures are readily available.

  31. Re:What about the fundementalists. by St.Creed · · Score: 1

    Your post probably lies somewhere in the intersection of the set of insightful postings, funny postings and "oops, I accidentally" postings.

    --
    Therefore, by the (faulty) logic you're using, you're just a cow with a keyboard - osu-neko (2604)
  32. Re:What about the fundementalists. by LordLimecat · · Score: 1

    I would call myself evangelical, and Ive only ever heard the position stated above coming from "the other side of the debate", or the WBC (whom noone takes seriously and can hardly be described as either fundamentalist or evangelical).

  33. Re:What about the fundementalists. by simtel · · Score: 1

    That's why the scientists got rid of most of the genome. All that's left should be covered by Fair Use, right?

  34. Re:What about the fundementalists. by terjeber · · Score: 1

    Nah, what God specializes in is to create havoc on the world, Man desperately tries to remedy his mistakes. What Man excels at is inventing new infantile fairies in the sky that are cause of this, that or the other.

  35. Re:So I have good news and bad news by Lotana · · Score: 1

    The good news is that we got everything out and you will be fine.

    The bad news is that we confused you with another patient and amputated your legs.

  36. More than 30M by RivenAleem · · Score: 1

    Is still only 5 million per life saved, which I think is a fair bargain. That's of course if they simply stop there. 30M for the development of a single drug is chump change in Pharma industry. 30M for a process that will define a whole new branch of medicine is amazing.