Scientists Reverse Muscular Dystrophy In Dogs 143
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."
Beware (Score:3, Informative)
The video link is pop up hell in IE.
Re:Beware (Score:4, Funny)
Re:Beware (Score:5, Funny)
"IE"? What is that? I cannot recall... :P
It's more commonly known as "the Firefox downloading tool".
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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...
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Dealing with morons that have a higher position than you is part of life. You have to pick your battles carefully or you will be fighrting for nothing all the time.
Re:Beware (Score:5, Funny)
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Useful alternative (Score:2)
Re:Beware Then beware of Dog? (Score:1)
If so, then all THREE of these are dog-gone shames:
The MD is gone
the pop-up-hell site is a doggone shame
the Dog vs God is a dog gone shame
(woof woof...)
Does this mean... (Score:2, Funny)
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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.
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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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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.
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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*
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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.
No (Score:1)
Patch Tuesdays? (Score:1, Funny)
If this does work, will our descendants have to deal with a more personal variation of Patch Tuesdays??
Re:Patch Tuesdays? (Score:4, Funny)
Will they usee same level of quality control as Microsoft?
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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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
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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
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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?
So that's how the Rage Virus will get made! (Score:1)
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!
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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.
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In other words, You are the new exon-skipping overlord
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-- Jamie Zawinski
But he's not a geneticist, so what does he know?
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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.
Side effects? (Score:1)
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?
The problem with patches... (Score:1)
... Is that they end up regressing, will someone just bite the bullet and fix upstream?!
Results based on? (Score:2)
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
Good luck getting your HMO to pay for it (Score:1)
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.
A collective win for Jerry's Dogs! (Score:1)
hang on.. (Score:1)
I thought we were against animal testing?
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?
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.
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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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Massive immune system response to the gene tinkering leading to immediate death.
They are injecting short nucleic acids that block a signal on RNA (a splice site) from being recognized which causes part of it to be skipped and at the end you get a shorter protein. The nucleic acid fragments are not immunogenic, and the shorter protein does not have any new sequences to which the immune system has not developed tolerance. So this is not real concern.
Some gene gets tinkered in the wrong spot and you get cancer too.
This is a concern when developing gene therapy. In this case this is not an issue, because you are not 'tinkering' with the gene. What they
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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...
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Re:Tag: whatcouldpossiblygowrong (Score:4, Insightful)
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.
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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
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Oh? How much does health insurance cost? What, about $20k a year for the average family? That is a HUGE burden on employers/families/the government. It's a huge factor in employment costs, especially in relation to competition that doesn't have that huge overhead.
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As Red Flayer noted, medicine isn't on a 'getting cheaper' trend. Sure, some basic care is cheaper than ever, but we're still very much making more treatment options - this is good for our health, bad for our wallets.
By that token, I support any families that chose to eliminate inheritable diseases such as MS, MD, even diabetes. Even to the point of aborting fetuses with those traits. Yes, I know about various slippery slope arguements, but let's face it, under older conditions such babies would be chose
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Why have medecine at all, then? We should all just die of gangrene and dysentery. Only the tough deserve to survive!
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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There's no money to be made on future generations that way. Think of it as pay-per-play genetics.
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Granted, the carriers of this gene might not
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Then don't allow selection for 'designer' babies, allow selection for broken metabolic paths and such. There's not really an upside to MD, diabetes, multiple sclerosis, etc...
Sickle cell is a bit iffy - but by the time we get around to doing the selection for it, Malaria shouldn't be a problem, or we might have genetic engineering to the point that we can do a custom modification to provide immunity on our own.
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One doesn't really know - perhaps some of these problems in less sever forms provide some advantage at some point in the organism's life - enough to offset their disadvantages - similar to Sickle cell anemia. I heard some discussion on a possible benefit for the fetus to implant on the uterine wall for one of them - a significant fraction of conceptions end up as unnoticed miscarriages I am led to believe. It is at least conceivable
Natural Selection... (Score:2)
However, Hawkins was a genius even before his ALS got bad, and on average the benefits from avoiding ALS in the first place exceed any theoretical gains. For that matter, it's likely that we'll lose Hawking early, compared to if he was healthy. As for ATP production, does having it be more efficient at the cost of MD make it worth it? Looking at animal life - these are traits that get selected out quickly and efficiently by nature. By nature bad mutations pop up far more frequently than good ones, but t
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It's spelled "Gattaca" (Score:2)
Gatica here we come.
There's no "i" in "Gattaca". Just like there's no base in DNA that has "i" as it's first letter. Adenine, thymine, guanine, cytosine - see, none of these starts with an "i". If you put any letters other than a, t, g and c in Gattaca, then you didn't get the joke.
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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
Unnatural selection? (Score:2)
How about we take a step back and simply encourage such people to perform genetic screening, perhaps IVF to avoid passing the genes to their kids?
We're intelligent animals, actually capable of guiding the genetics of our offspring - why wouldn't we want to make sure they have the best?
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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.
Talk about a slippery slope. By that logic all medicine should be banned. It's a flawed argument, but regardless, something tells me that's an argument you're not likely to win because people would never be prepared to accept that conclusion even if you could prove you were right.
Certainly one thin
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'Survival of the fittest' hasn't applied for at least a few hundred years when it comes to humankind,
Sorry, "survival of the fittest" is alive and well, even for the human race. You're "fit" if you can produce offspring and keep it alive until it in turn reproduces. There's really no other criterion.
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.
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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)
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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,
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They didn't do enough testing of thalidomide. The proper response to mistakes is not to stop doing new things, it is to be more careful (like doing more testing, and developing models that help predict the impact of a molecule prior to that testing).
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They didn't do enough testing of thalidomide. The proper response to mistakes is not to stop doing new things...
Yes, which is why I said, "Now, I'm not saying that something horrible will go wrong, but I am in favor of extensive testing and forethought into consequences..."
wost thing? (Score:1)
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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 st
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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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