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Scientists Reverse Muscular Dystrophy In Dogs

Al writes "Scientists have taken a step toward developing a cure for Duchenne muscular dystrophy (DMD) by successfully treating the condition in dogs using a novel genetic technique. The scientists used a method called exon skipping, which involves adding a genetic 'patch' to block transcription of a portion of the gene involved in DMD. This puts the remaining genetic sequence back in order, essentially creating a much less severe version of the condition. The scientists recorded some remarkable video footage showing the resulting improvements in several dogs with naturally-occurring DMD. More work is needed before the treatment can be given to humans, however, because DMD sufferers often have different genetic mutations."

16 of 143 comments (clear)

  1. Re:Beware by brian0918 · · Score: 3, Funny

    "IE"? What is that? I cannot recall... :P

  2. Re:Beware by PrescriptionWarning · · Score: 4, Funny

    wait slashdotters don't use IE... Imposter! Who are you and what have you done with the owner of that computer!

  3. Is it heritable? by PotatoFarmer · · Score: 4, Interesting

    I'd be interested to see whether or not the "patch" is heritable; the article doesn't mention it. In any case, it's really impressive work.

  4. Re:Beware by Anonymous Coward · · Score: 5, Funny

    "IE"? What is that? I cannot recall... :P

    It's more commonly known as "the Firefox downloading tool".

  5. Re:Tag: whatcouldpossiblygowrong by MozeeToby · · Score: 4, Insightful

    Ok, seriously. What could possibly go wrong?

    We're talking treating people who are almost certainly going to die anyway with a genetic approach that doesn't have even a theoretical way to spread to other people. The absolute worst thing that could go wrong is that the people being treated die from the treatment. The second worst thing that could happen is that we don't do the treatment and they die anyway; though maybe a bit later.

    I'm seriously asking, what do you think could actually go wrong?

  6. I read the headline as... by Anonymous Coward · · Score: 4, Funny

    For some reason, I read the headline as "Scientists Reverse Muscular Dystrophy in Frogs". Reading that, I thought, "Well no wonder the French love Jerry Lewis".

  7. Great News by Nos. · · Score: 5, Informative

    I just found out that two nephews of three are positive for DMD. This basically confirms that my sister-in-law is a carrier. We're in the middle of trying to determine if my wife is a carrier, and thus if our two sons are at risk. To say the least this is a very stressful time in our lives, and there are no quick answers. However, seeing a big jump like this in treatment is great news.

  8. I can see it now by Rayban · · Score: 3, Funny

    # patch -p0 < cure-md.patch

    File to patch: chromosone/18
    patching file chromosone/18
    Hunk #1 FAILED at 47.
    Hunk #2 FAILED at 128.
    Hunk #3 FAILED at 308.
    Hunk #4 FAILED at 316.
    Hunk #5 FAILED at 328.
    Hunk #6 FAILED at 342.
    Hunk #7 FAILED at 397.
    Hunk #8 FAILED at 708.
    Hunk #9 FAILED at 1268.
    9 out of 9 hunks FAILED -- saving rejects to file
    chromosone/18.rej

    --
    æeee!
  9. Re:Tag: whatcouldpossiblygowrong by Mordaximus · · Score: 3, Insightful
    We're talking treating people who are almost certainly going to die anyway with a genetic approach that doesn't have even a theoretical way to spread to other people. The absolute worst thing that could go wrong is that the people being treated die from the treatment. The second worst thing that could happen is that we don't do the treatment and they die anyway; though maybe a bit later.

    Really? If I were the betting type, I'd say just about everyone is almost certainly going to die, not just those afflicted with MD. The most important thing anyone can ask for isn't longevity, it's quality of life. Your list of outcomes is incomplete - I'd at the very least put "the treatment leads them to suffer more than they already do" far ahead of any others.

  10. Re:Patch Tuesdays? by SnarfQuest · · Score: 3, Funny

    Will they usee same level of quality control as Microsoft?

    --
    Who would win this election: Andrew Weiner vs Andrew Weiner's weiner.
  11. Re:Tag: whatcouldpossiblygowrong by DanTheStone · · Score: 4, Insightful

    Since the fix isn't inherited, this could increase the rate of this disorder in the whole human race. If genetic disorders never select out, a lot more people would become dependent on the treatment in the future. There's a reason why natural selection is important to the survival of a species. In a nutshell: More people who have this disorder will be able to have children and pass it on.

  12. Slashdot makes my day by Naked+Jaybird · · Score: 5, Insightful

    As one diagnosed with Becker's MD, a milder form of DMD, I, for one, welcome my new exon-skipping overlords. For those of you who are wondering if you should go to the gym, run, jog, shoot hoops, or play soccer today. I give you the same advice that I give to my three boys: Run, because you can.

  13. Re:Tag: whatcouldpossiblygowrong by h4rr4r · · Score: 4, Insightful

    How is this different from eyeglasses/contacts?

    If we can fix it, why should it be selected against?

    Natural selection is not a force for the survival of a species, it is not some artist or designer. It is merely the natural tendency for some traits to be selected against from environmental pressure. If there is no environmental pressure against the traits they do not get selected against. This is no different than taller growing trees, lack of food at one height, making an incredibly long neck no longer a hindrance. If a cure was invented that means the environment changed and there is no longer a selection pressure against this trait.

  14. Re:Tag: whatcouldpossiblygowrong by vishbar · · Score: 5, Funny

    The absolute worst thing that could go wrong is that the people being treated die from the treatment

    Nuh-uh. Did you ever see 28 Days Later?

    Zombies. The worst thing that could go wrong are zombies.

    --
    Ride the skies
  15. Re:Beware by Mister+Whirly · · Score: 4, Funny

    If I quit every job I had every time someone higher up decided something stupid, I would be permanently unemployed.
    It's called "work" for a reason. If it was fun, it would be called "fun", and financial compensation would not be required...

    --
    "But this one goes to 11!"
  16. Re:Tag: whatcouldpossiblygowrong by nycguy · · Score: 3, Funny

    As a zombie, I'll tell you right now that I try to stay away from genetically modified people, and I think they should be banned from the food chain.