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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."
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.
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.
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?
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".
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.
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.
# 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!
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.
Will they usee same level of quality control as Microsoft?
Who would win this election: Andrew Weiner vs Andrew Weiner's weiner.
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.
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.
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.
Nuh-uh. Did you ever see 28 Days Later?
Zombies. The worst thing that could go wrong are zombies.
Ride the skies
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.
"Science flies us to the moon. Religion flies us into buildings." - Victor Stenger
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.
"Trolls they were, but filled with the evil will of their master: a fell race..." -- J.R.R. Tolkien on Olog-hai
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.
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?
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.
Why don't we rejoice that this gives patients a CHOICE in the matter. Let people make up their own minds about the risk.
Currently hooked on AMP
Oh yeah, did you ever see "28 Days" with Sandra Bullock? Now that is some scary shit...
Help fight poverty: Punch a poor person.
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...
Here's to hot beer, cold women, and Glaswegian kisses for all.
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.
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.
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 economic one. You're alive and reading this, not because you're the pinnacle of human health and fitness (lol, this *is* slashdot); but more likely because you avoided dying of poverty. Just like me (though I certainly had some close calls).
If we really wanted to, we could save hundreds of millions more people from dying just using the technology we already have. Heck, if we'd been doing that since the dawn of man - I bet we could have overpopulated ourselves right out of existence by now. We may yet.
Instead, we let hundreds or thousands die to gain the ability to save one. Yeah, sometimes it's Dick Cheney, but sometimes it's Stephan Hawkings.
Food gets eaten, medicine gets used up, research budgets get spent - but knowledge and discover remain. There's your cost benefit analysis.
And I write this as both someone who's spent the last year not getting properly treated for a spine injury because my insurance company decided pain pills were cheaper, and who has a sister who's dying of an unprofitably rare disease. But of course, both of us would have died in childhood anyway if it weren't for medical discoveries that didn't exist in our parents generation.
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.