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Scientists Reverse Muscular Dystrophy In Dogs 143

Posted by Soulskill
from the barking-up-the-right-tree dept.
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."
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Scientists Reverse Muscular Dystrophy In Dogs

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  • Beware (Score:3, Informative)

    by dnormant (806535) on Monday March 23, 2009 @12:09PM (#27300507)

    The video link is pop up hell in IE.

  • ...the end of Labor Day Weekend Telathons? What will Jerry Lewis do now? Guess my 25 cents in a fireman's boot actually worked.
    • Re: (Score:1, Insightful)

      by Anonymous Coward

      What will Jerry Lewis do now?

      I don't know about the immediate future, but I guess this means he gets to die knowing that he has made a positive contribution to humanity as a whole.

    • by Chabo (880571)

      He could write a sequel to his autobiography, "Dean and Me".

  • Is it heritable? (Score:5, Interesting)

    by PotatoFarmer (1250696) on Monday March 23, 2009 @12:17PM (#27300665)
    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.
    • I doubt it, especially from women.

      • Re: (Score:3, Informative)

        by PotatoFarmer (1250696)
        I guess it would depend on the potential methods of inheritance. If, in addition to directly modifying the production of male sperm, the patch could be delivered through the placenta to a fetus similar to how antibodies are transferred then it could still be heritable through the female.

        That all being said, I'm not a biologist, so it's entirely possible that what I've described can't actually happen.
    • Re: (Score:1, Informative)

      by Anonymous Coward

      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.

      It's not.

    • by Shikaku (1129753)

      The patch will have to be in either the eggs of the female (very unlikely it will reach there) if it even COULD work at all because of the way eggs are special (giant, hard large shell) or in the site where male sperm does its meiosis; sperm cells are made by dividing like mitosis then dividing again to form 4 cells with half the DNA. It's possible in males, but very unlikely in females.

    • by MarkRose (820682)

      That's okay, just call Tank and he'll upload one! "Tank! I need a patch!"

      *goes back to bending spoons*

    • Re: (Score:3, Informative)

      by vivin (671928)

      IANAG (IANA Geneticist), but from what little I know about genetics, I doubt it is heritable. The only way something can be heritable is if it modifies any of the germ cells (sperm or ova). In fact, some of the "junk" DNA that we have are actually inactive sequences of ancient retroviruses (ERVs - Endogenous retroviruses [wikipedia.org]) that infected the germ cells in our ancestors.

    • by pesho (843750)
      Nope. They are not touching the DNA.
  • by Anonymous Coward

    If this does work, will our descendants have to deal with a more personal variation of Patch Tuesdays??

  • by Anonymous Coward on Monday March 23, 2009 @12:27PM (#27300825)

    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".

  • Great News (Score:5, Informative)

    by Nos. (179609) <{ac.srrekeht} {ta} {werdna}> on Monday March 23, 2009 @12:31PM (#27300877) Homepage

    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.

    • Re: (Score:3, Insightful)

      by JorDan Clock (664877)
      Pray to dieties, sacrifice every living animal you can get your hands on, and avoid every single bad luck superstition. My brother has DMD and at the age of 30, he can't even feed himself anymore. I sincerely hope for the best with your children.
      • by Nos. (179609)

        We were waiting to get genetic testing done on my wife, but they've come back and said get CK levels done on the kids first. We're hoping to get those done this week. We did get a CK level done on my wife, and hers was 320. Given she's physically active, it pretty much doesn't tell us anything.

        The one positive is that no one else on my wife's side of the family has had, or shown symptoms of muscular dystrophy, which leads me to believe that her sister became a carrier as a result of a mutated egg, and di

        • Re: (Score:2, Informative)

          by anderesa (445239)

          Dear Andrew,
          as a daily reader of Slashdot and also father of four young kids, two of them having Duchenne, I'm surprised that this terrible desease is discussed among this community.

          Unfortunately, the discussion doesn't go very deep with few interesting threads. I cross my fingers for your family. My wife also is a carrier but my two sister-in-laws. In fact, we found out that my wife got the defect (3 Exons are deleted on one X-chromosom) from her mother but she's been the only one among five kids to inheri

    • by elrous0 (869638) *
      Good news for humans. Great news for dogs.
  • Original Article (Score:2, Interesting)

    by Elenseel (1510087)
    Is there a way to get the original article published by the scientists who developed the technique? My mentorship is heavily rooted in genetic analysis, so I'm interested in these kinds of things.
  • by Rayban (13436) on Monday March 23, 2009 @12:35PM (#27300957) Homepage

    # 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

  • I'm heading for the mountains with my shotgun. Be sure to act intelligent if you see me or else I'll have to assume you're infected!

    • by elrous0 (869638) *
      According to Battlestar Galactica, its those damn Japanese robots that should worry us. Leave the Roomba behind when you go.
      • actually the Japanese are the ones making robots with the intentions of making them friends, whereas others tend to militarize them and think of them as becoming our new overlords. That's possibly why they ended the series showing only the robots that weren't killing machines.
  • does these sort of medical tests on animals end up with better treatment of animals aswell by passing the info onto vets or is it generally not considered worth it for mans best friend.
    • In a substantial number of cases, human therapies do become available for animals. They even have vets that specialize by disease area instead of by animal type(ie. Veterinary Oncologists vs. Large animal or small animal vets). This is also why you can now get animal health insurance.
      • by TheSync (5291)

        In a substantial number of cases, human therapies do become available for animals.

        It is easier and cheaper for these therapies to become available for animals because of less regulation. For example, you can clone animals today, but cloning people is illegal...

  • by Naked Jaybird (1190469) on Monday March 23, 2009 @12:56PM (#27301259)
    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.
    • by sherriw (794536)

      That, Naked Jaybird is an EXCELLENT comment. I copied it down and put it next to my 'work out plans' that I've been neglecting.

    • by Xaedalus (1192463)
      Amen. Thank you for that. I plan to do so.
    • by gurps_npc (621217)
      By the way, if you read the article, tehy don't really cure DMD. They transform DMD into Becker's MD, what you have.

      In other words, You are the new exon-skipping overlord

      • Beautiful. Is this the first time we have had a real overlord on Slashdot? Bow down to me! But seriously, what I have is a single point mutation, so skipping errors is a reason to be optimistic. I do not know if skipping a single point will increase dystrophin in people with Becker's MD, but skipping an error and running execute something else would be the winner. Actually, what I need is a substitution regex.
        • by idontgno (624372)

          Some people, when confronted with a problem, think "I know, I'll use regular expressions." Now they have two problems.

          -- Jamie Zawinski

          But he's not a geneticist, so what does he know?

    • MD diagnosed here too, FSH (Fascio/scapula/humeral) variety. Great advice Jaybird.

      The thing I find hardest is adjusting to limitations, with no possibility of improvement.

      This at least offers some hope of treatment and maybe less pain.

  • Will this "patch" remove the "I'm going to butt-scoot across your white carpet" and the "I'm drooling cause you said the word treat" genes as well?

    Or, heaven forbid, will this treatment have Viagra-like side-effects?

  • ... Is that they end up regressing, will someone just bite the bullet and fix upstream?!

  • The videos? I certainly hope they based their findings on more then that.

    Correct me if I am wrong, but the untreated dogs shown at the beginning of the video do not appear to be the treated dogs shown in the latter half of the video. The age given for one of the treated dogs is actually 3 months younger then either of the untreated ones shown.

    So, what exactly is the video supposed to portray? It is impossibly to make any comparison based on the video because there is no "before" and "after" nor do we have t

  • They'll just fuck the dog until the disease kills the patient rather than pay for the cure that could turn him/her back into a productive citizen. Otherwise, Wall Street won't like the numbers and the CEO will have to settle for a 140 foot yacht instead of a 150 foot one.

  • Somewhere, Jerry Lewis is hugging a dog in a wheelchair right now and saying "Good news, Puppy, We've found away to reverse Muscular Dystrophy."
  • I thought we were against animal testing?

I am here by the will of the people and I won't leave until I get my raincoat back. - a slogan of the anarchists in Richard Kadrey's "Metrophage"

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