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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.
n/t
...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.
Think what could possibly go *right*. My mother is a physical therapist and she wouldn't mind being out of a job if children suffering from even just the DMD type could be cured. Can we stop with this pointless tag? You're disaffected and parroting unoriginal sarcasm, good for you.
If this does work, will our descendants have to deal with a more personal variation of Patch Tuesdays??
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!
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!
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.
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.
Like domestic oil?
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..
Taking guns away from the 99% gives the 1% 100% of the power.
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?
Sent from your iPad.
it's chromosoMe!
File to patch: chromosone/18
Path chromosone/18 not found.
... Is that they end up regressing, will someone just bite the bullet and fix upstream?!
Sticking feathers up your butt does not make you a chicken.
Everybody talks about the exons. Won't someone please think of the introns?
Intron: the portion of DNA which expresses nothing useful.
Didn't you see I Am Legend? Thats the worst possible thing.
It will be better to purchase from an owner who is a good farmer and a good builder.
How come this article isn't showing up on /.'s main page? I happened to notice it from RSS feed on my Goggle homepage, but it's not on the main page anymore?
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 the opportunity to see ANY of the dogs in more then one state, treated or untreated.
All I see is several dogs, never more then once, suffering varying degrees of motor skill loss.
Am I missing something here?
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?
I don't read AC A human right
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.
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.
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...)
Previously: "Linux... Toward the Sunrise..." Now: "Linux... Toward the-- No, now, part of Every Sunrise"
I don't care about what others say, the video worked for me. And what I saw impressed the hell out of me! Seriously, and all internet /doesn't work here/ junk aside, this video is freaking awesome! It shows before and after videos of dogs with muscular dystrophy going down a hall. The before vids show the dog barely able to walk, with people shuffling along (slower than walking) to encourage the dog to keep going. The dog can't move their legs very well. It then goes to show the same dog after 5, then 11, then 20 treatments. After 20 treatments, the researchers are basically running (not sprinting, but certainly a full run) down the hall, with the dogs keeping up. I don't have muscular dystrophy, but for people /families-of-people who have it, this must look like a miracle. Un-freeking-believable!
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."
The Rapture is NOT an exit strategy.
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 the weeding of natural selection promptly removes the bad ones(on average).
I'm not saying that we don't do a thorough workup, it's just that there are any number of hereditary diseases that don't actually have a benefit. Heck - for sickle cell it might be a benefit to simply make sure that no new babies have the double recessive trait. There might be some other recessive diseases that one copy would actually provide some benefits, such as 'Wiedemann-Beckwith syndrome' aka 'double muscle'. A double copy turns average joe into an involuntary Mr. Universe, a single copy makes it possible. Overall the double copy is negative because it produces so much muscle it over strains the body - the heart and other organs remain normal sized. But with today's sedentary society, a mild case might actually help.
Still, on average I'd say that we actually already DO know, at some point we can say 'For disorder X, there are no discernible benefits, even for the latent/recessive gene'. There's a whole host of genetic defects that can be classed this way.
I don't read AC A human right
Nope. They are not touching the DNA.
I thought we were against animal testing?
Might I recommend Firefox Portable for such people? It's a bloody godsend.
Meta will eat itself
Sometimes, it's not so useful to rely on /. for breaking cutting edge news...
The drug you are interested in is PTC124, otherwise known as Ataluren. It is currently in phase 2 clinical trials IN HUMANS (in the UK, at least) - We have boxes of it...
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.
Yours was the only kind and careful post on this topic. Was wondering is the lack of response. Mike has used ultrasonic vibration therapies to remedy and heal muscular distrophy on people. Give him a call 630.627.8880 or look through his website http://www.wakeupwell.org./