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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?"

26 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/

  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 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!
    2. 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.

    3. 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...
    4. 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)
  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 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. "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 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)
  10. 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.

  11. 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
  12. Re:Fucking with HIV by The+Rizz · · Score: 4, Funny

    What could POSSIBLY go wrong?

    You could get HIV pregnant.

  13. 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.

  14. 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.

  15. 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.

  16. 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...
  17. 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.