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Scientists Reverse Muscular Dystrophy In Dogs
Posted by
Soulskill
on Mon Mar 23, 2009 11:58 AM
from the barking-up-the-right-tree dept.
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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Submission: Scientists Reverse Muscular Dystrophy In Dogs by Anonymous Coward
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Beware (Score:3, Informative)
The video link is pop up hell in IE.
Re:Beware (Score:4, Funny)
Parent
Re:Beware (Score:5, Funny)
"IE"? What is that? I cannot recall... :P
It's more commonly known as "the Firefox downloading tool".
Parent
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I know, I know. I was actually surfing in firefox but all the pop ups were in IE. I can't disable IE because all of my work related crap requires it.
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I can't disable IE because all of my work related crap requires it.
Clearly it's time to update your resume and pack your bags.
Re:Beware (Score:5, Funny)
It's called "work" for a reason. If it was fun, it would be called "fun", and financial compensation would not be required...
Parent
Re:Beware (Score:5, Funny)
Parent
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Does this mean... (Score:2, Funny)
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He could write a sequel to his autobiography, "Dean and Me".
Is it heritable? (Score:5, Interesting)
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I doubt it, especially from women.
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That all being said, I'm not a biologist, so it's entirely possible that what I've described can't actually happen.
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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.
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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)
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.
I read the headline as... (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".
Great News (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.
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Original Article (Score:2, Interesting)
I can see it now (Score:4, 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
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Can you post that with diff -u?
All this animal testing good for vets? (Score:2)
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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...
Slashdot makes my day (Score:5, Insightful)
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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.
Re:Tag: whatcouldpossiblygowrong (Score:5, 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?
Parent
Re:Tag: whatcouldpossiblygowrong (Score:4, Insightful)
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.
Parent
Re: (Score:3, Insightful)
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.
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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.
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Really? If I were the betting type, I'd say just about everyone is almost certainly going to die
"Just about" everyone is "almost certainly" going to die? Last time I checked, I'd say EVERYONE is going to die. The only questions are "when", and "by what". Of course, I could be wrong, there are ALWAYS statistical outliers...
Re:Tag: whatcouldpossiblygowrong (Score:4, Insightful)
Parent
Re:Tag: whatcouldpossiblygowrong (Score:5, 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.
Parent
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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 o
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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 electrici
You raise an interesting point (Score:3, Interesting)
Without even getting into a cost-benefit analysis of *any* form of medical care - it's astonishing how many people die from diseases that can be treated with substances like... food, clean water, even clean air.
Yes, that's right - every Flintstones chewable you give your kid *could* have been money spent on iodine which saves some other kid from life long brain damage.
So let's not kid ourselves into thinking that "survival of the fittest" is a primarily a biological test for mankind anymore. It's an economi
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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
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Granted, the carriers of this gene might not
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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
Re:Tag: whatcouldpossiblygowrong (Score:5, Funny)
Nuh-uh. Did you ever see 28 Days Later?
Zombies. The worst thing that could go wrong are zombies.
Parent
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Oh yeah, did you ever see "28 Days" with Sandra Bullock? Now that is some scary shit...
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Re:Tag: whatcouldpossiblygowrong (Score:4, Funny)
Parent
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I'm seriously asking, what do you think could actually go wrong?
Sometimes the worst side-effects of our actions are those that we hadn't even imagined before it happened. Thalidomide [wikipedia.org] is one example from the medical field-- as far as I know, nobody had an indication that it was dangerous when they first started using it.
Now, I'm not saying that something horrible will go wrong, but I am in favor of extensive testing and forethought into consequences of our medical technologies, particularly when dealing with genetics. Some of the dangers may be overblown by Hollywood,
Re:Patch Tuesdays? (Score:4, Funny)
Will they usee same level of quality control as Microsoft?
Parent
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Like domestic oil?
I was making a play on words with Exon/Exxon. Clearly, my attempt at humor this morning has flown way over the head of the moderators..
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