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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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  • by MozeeToby (1163751) on Monday March 23, 2009 @01:22PM (#27300741)

    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?

  • by Anonymous Coward on Monday March 23, 2009 @01:31PM (#27300879)

    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 Mordaximus (566304) on Monday March 23, 2009 @01:36PM (#27300959)
    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.

  • by DanTheStone (1212500) on Monday March 23, 2009 @01:43PM (#27301055)
    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.
  • Re:Beware (Score:3, Insightful)

    by fuzzyfuzzyfungus (1223518) on Monday March 23, 2009 @01:47PM (#27301113) Journal
    Slashdotters goofing off on (unenlightened) company time do...
  • by Naked Jaybird (1190469) on Monday March 23, 2009 @01:56PM (#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 h4rr4r (612664) on Monday March 23, 2009 @02:01PM (#27301315)

    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.

  • by vlm (69642) on Monday March 23, 2009 @02:08PM (#27301421)

    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.

    Yes for cosmetic genetic engineering stuff like changing eye color or womens chest size I'd agree, the possible downsides could be pretty icky.

    But, MD is not exactly a joyous party... Even if you intentionally tried, how do you suggest you'd make it even worse? You'd have to do some pretty ridiculous scaremongering like claiming they "could" get something like rabies or ebola, or "could" become lycanthropes. But that doesn't sound very responsible in their situation.

  • Re:Great News (Score:3, Insightful)

    by JorDan Clock (664877) <jordanclock@gmail.com> on Monday March 23, 2009 @02:09PM (#27301433)
    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 Red Flayer (890720) on Monday March 23, 2009 @02:14PM (#27301505) Journal
    I'm not totally cruel, but:

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

    Because it's expensive to fix it, and letting it propagate in the gene pool means we'll have to pay to fix it in a higher and higher proportion of the populace.

    From an economic perspective, the miraculous state of modern medicine will bankrupt us. From a moral perspective, it's a hard choice to make, about whether we can afford to cure everyone of everything curable.

    But I think the simple truth is that the cost/benefit ratio of curing (or partially curing) certain diseases is far too high... especially among the elderly (who have little economic productivity left in them).

    I know it's cruel and morally questionable, but at what point do we realize we are bankrupting future generations just to extend our lives a few measly years?

    Note that curing MD is something completely different, since it is not a disease of the elderly.

  • by JerryLove (1158461) on Monday March 23, 2009 @02:22PM (#27301607)

    Eyeglasses don't fix poor vision. They compensate for it.

    Why would you want to deslect for it? Because a population that needs braces, eyeglasses, custom shoes, and a pace-maker at birth is not a laudable goal. In addition to the clear inferiority of "overcoming problems" to "never having problems", there's the issue of what happens if the technology infrastructure breaks down.

    On the other hand: the beauty of gene-therepy is that it should be applicable to reproductive cells. Alter the MD gene in an egg or zygote and you remove it from future generations as well. Presumably the same applies to altering the semenal-creating cells in the testies.

  • by lenehey (920580) on Monday March 23, 2009 @02:24PM (#27301639)

    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.

    That's a good reason not to give kids eyeglasses or braces or, hell, lets not give any medical care to kids at all. And, maybe if you get beat up in the schoolyard, you should be left to die because, well, "survival of the fittest" and all that... You need to explain why Muscular Dystrophy should be singled out for non-treatment, or if not singled out, where you draw the line. Is it because its a genetic treatment? How is that worse than injecting yourself with insulin the rest of your life to keep you well? Or laser eye surgery for the blind or cochlear implants for the deaf?

  • by h4rr4r (612664) on Monday March 23, 2009 @02:24PM (#27301645)

    We are not bankrupting anyone, look at doctor in a box places and what nurses can do these days. Health care is just stating to be commoditized, once that really gets going prices will fall dramatically. There is little need for our current see the MD when you feel ill system. Seeing a nurse, having some tests and letting the doctor review that information is much cheaper and will make healthcare accessible to more and more people.

    Conserving healthcare is as dumb as pretending that conservation of electricity will bring about a solution to that issue. Only when we consume so much that the price rises to an unacceptable level is any progress made. This is why war drives progress, bullets at $1 a piece are fine until you need 1 million of them. This is why the electric car will only take off when oil prices surge once again beyond $100 a barrel.

  • by Anonymous Coward on Monday March 23, 2009 @02:31PM (#27301727)

    idiot,

    I hope you never need a blood transfusion, organ transplant, vaccine or pretty much any other medical procedure developed since blood transfusions were made safe enough to be useful, That research was done using (not surprisingly) animals too.

    You peta types are the epitome of clueless hypocrites, and prove it every time you open your mouths.

    If you had paid attention to even a semester of actual science classes you would understand the way research actually works, and you wouldent say such obviously stupid things.

    Your decision to remain ignorant allows me to feel justified in calling you a willful idiot and ignoring anything else you say on any subject.

  • by wealthychef (584778) on Monday March 23, 2009 @02:38PM (#27301799)
    Why don't we rejoice that this gives patients a CHOICE in the matter. Let people make up their own minds about the risk.
  • by Tubal-Cain (1289912) on Monday March 23, 2009 @03:04PM (#27302143) Journal

    Massive immune system response to the gene tinkering leading to immediate death.

    To quote the OP (MozeeToby):
    The absolute worst thing that could go wrong is that the people being treated die from the treatment.

    Some gene gets tinkered in the wrong spot and you get cancer too.

    Cancer isn't the death sentence it once was.

    Go through a costly and/or miserable treatment with no effect.

    Baseball analogy: If you don't swing, you will be in for somewhere between 3 and 6 pitches and might get on base if the pitcher sucks (he doesn't, in this case). If you swing at every pitch, you might strike out after 3 pitches. Or you might keep fouling out indefinitely, and get much more than 6 pitches. Or you might get a base hit.

  • by greenkite71 (910398) on Monday March 23, 2009 @11:31PM (#27307591)
    I have FSHD (another mild form of muscular dystrophy). If this approach is ever applied to FSHD, I will try to be one of the first in line. To those of you who prefer that we die so that the your conception of the human race doesn't become weaker, I suggest your line of thinking presents a far greater risk to humanity than my genes.

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