CRISPR Gene Editing Fixes Muscular Dystrophy In Dogs, Humans Could Be Next (time.com) 112
schwit1 shares a report from Time: In a new paper published in Science, researchers led by Eric Olson, professor and chair of molecular biology at UT Southwestern Medical Center, reported that he and his team successfully used CRISPR to correct the genetic defect responsible for Duchenne muscular dystrophy in four beagles bred with the disease-causing gene. It's the first use of CRISPR to treat muscular dystrophy in a large animal. (Previous studies had tested the technology on rodents.) In varying degrees, the genetic therapy halted the muscle degradation associated with the disease. Duchenne is caused by mutations in the dystrophin gene, which codes for a protein essential for normal muscle function. People born with the disease are often eventually confined to wheelchairs as their muscles continue to weaken, and in the later stages, many rely on ventilators to breathe as their diaphragm muscles stop working. Eventually, they develop heart and respiratory failure.
Olson and his team "fixed" the mutated dystrophin gene in four dogs by splicing out an offending section of the gene using CRISPR. The gene editing technology, discovered in 2012, can cut out sections of DNA at precise locations (and also potentially introduce new DNA as well). In the case of Duchenne, says Olson, simply snipping out a section of the mutated dystrophin gene allows the gene to make enough of the proper protein that muscles need to function. The hope is that if those animal studies and human trials prove this technique is safe and effective, CRISPR could potentially lead to a cure for Duchenne, Olson says. "We are going for a cure, not a treatment," he says. "All of the other therapies so far for Duchenne muscular dystrophy have treated the symptoms and consequences of the disease. This is going right at the root cause of the genetic mutation."
Olson and his team "fixed" the mutated dystrophin gene in four dogs by splicing out an offending section of the gene using CRISPR. The gene editing technology, discovered in 2012, can cut out sections of DNA at precise locations (and also potentially introduce new DNA as well). In the case of Duchenne, says Olson, simply snipping out a section of the mutated dystrophin gene allows the gene to make enough of the proper protein that muscles need to function. The hope is that if those animal studies and human trials prove this technique is safe and effective, CRISPR could potentially lead to a cure for Duchenne, Olson says. "We are going for a cure, not a treatment," he says. "All of the other therapies so far for Duchenne muscular dystrophy have treated the symptoms and consequences of the disease. This is going right at the root cause of the genetic mutation."
Humanity 2.0 (Score:4, Informative)
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Good luck getting the people who refuse to eat GMO foods to be ok with genetically modifying themselves. They imagine mad scientists randomly saying "I wonder what happens if I put THESE genes in THIS organism?" and the result having three heads. You can say, "hey, stupid people weed themselves out of the gene pool, natural selection", but unfortunately stupid parents can get their children killed too, and that's also natural selection (or micro-scale social darwinism).
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Three heads? How absurd. Now excuse me while I go feed my monkey with eight asses. (If you don't get this, clearly you don't watch Southpark.)
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If you need to explain a joke, it's probably not funny.
If you need to explain a joke, it's definitely not funny.
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Wrong. Medical genetic engineering and GMOs are very different.Genetic engineering as up to now means "more poison" pretty much. All the Roundup ready poison, the Bt-corn poison,etc. The only exception there is golden rice from Asia. All our western corporations only want to put more poison into the environment and our bodies. So being against GMO is very much a no brainer. The mainly asian governments want to prevent blindness in poor people instead. While there hsa been some resistance against golden rice
Re: Humanity 2.0 (Score:1)
Water is a poison that kills you if you take enough of it. Poison means nothing without numbers telling how much of it is needed to kill you.
Plants without "poison" will get abused by insects and that can make the plant more poisonous to humans that what it would be with poison.
Most countries put poison into the tab water so that the water would not kill people. If that doesn't make sense to you then gene manupulation won't either.
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Organic pesticides are less effective and require higher doses and more frequent application.
Those are facts on the ground.
Re: Humanity 2.0 (Score:2)
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Now comes the equally important but less sexy, longer-term study to see what collateral damage might be caused by this treatment.
Re:Humanity 2.0 (Score:5, Insightful)
Start by treating the worst diseases, and we'll find out. I'm sure we'll be able to find volunteers.
Re:Humanity 2.0 (Score:4, Interesting)
I've been following CRISPR with intense interest as a result of my son's condition and it really does look like a golden bullet for curing DMD. But early detection, preferably in-uterine detection, will be key. The reason being is that this treatment would essentially freeze the boys level of muscular competence. If you treat a child who has yet to show any symptoms, then he will likely never experience any symptoms. But if you treat a wheelchair bound 10 year old, he is not going to recover the ability to walk, he is going to be wheelchair bound for the rest of his life. The good news would be that this would greatly extend his life expectancy.
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A good friend is in a similar situation. His son was doing breathing exercises to maintain his 'breathing score' and just squeaked into the study, despite being older.
Now they are just scared that he was assigned to the control group and got placebo. There is at least one study accepting boys as old as 18.
Re: Humanity 2.0 (Score:3)
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The problem isn't that MD sufferers have a gene that healthy people lack. In most cases the patient has the bit of code, it's just malformed in some way. For the most severe cases such as Duchennes or Limb-girdle, the patient may actually be missing the relevant structure entirely.
An individual chromosome is made u
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The x.0 release are always buggy (Score:2)
Humanity 2.0
The x.0 release are always buggy, its best to avoid them.
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Get back in the pile! They tuk er jerbs!!!!!
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Will this repair the genes in the gametes? (Score:5, Interesting)
People equate death = bad. But if the death results from bad genes, the death is actually good (for the species) because it's functioning to reduce the prevalence of the bad genes from the population's gene pool. What's bad for the individual may be good for the species.
An alternative is to require people receiving this treatment to consent to forced sterilization (there are plenty of kids who need adopting anyway). But sterilization is a touchy subject which encroaches on the abortion debate (you're saying society can override an individual's right to control their own body).
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Citation needed, otherwise this is just a variant of the "let gays marry - and marrying dogs is next" argument.
Also I sure does hope it doesn't change reproductive cell until it is deemed to be completely safe.
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Citation needed, otherwise this is just a variant of the "let gays marry - and marrying dogs is next" argument.
Your comparison is seriously backwards, because gay couples and human-canine couples cannot reproduce by themselves. Besides, gay marriages are not heavy medical procedures. They are human conventions that would probably happen naturally anyway, were it not for specific restrictions on gay couples in many jurisdictions. By allowing gay marriages, we are making legislation simpler by removing these restrictions. Gay marriages don't take away anyone's rights and don't mean additional expenses for the society
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Married couples with one person providing most of the income have a slight federal income tax advantage. If the federal budget is fixed and the money has to come from somewhere, a married gay couple paying less under this scheme means a slightly increased burden on everyone else.
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Gay people have always been able to reproduce, how fucking stupid are you anyways?
How do you even get out of bed in the morning and get your shoes on without falling over and hitting your head? You don't seem smart enough even for that if you didn't know that gay people can have babies naturally.
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Gay people have always been able to reproduce, how fucking stupid are you anyways?
Can you read the part "by themselves"? I'm well aware of gay couples who have had children by various means, but this always involves a third party of the opposite sex (possibly via donated sperm or eggs). So the couple won't have genetic children together, which was the essential point here.
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Yes, by themselves, dipstick.
The amazing thing about your lack of reading comprehension here is that you said those words before, and I was already replying to them. You not only don't comprehend what I said, you didn't even comprehend what you said!
You seem to not really comprehend reproduction, or what the important elements are.
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You've got some word salad there, maybe you should just keep playing with your food until it spells out whatever crazy bullshit is in your head.
You understand, little babies get born, it's been happening for millions of years. When I observe that, and you can't figure it out, and want to argue with it, you're not going to convince me that you're actually trying to communicate with the other humans. You're just playing with your word salad, and you have no idea even that the words you don't get to choose are
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Passing genes along (Score:2)
As this is a insensitive issue, the truth is far simpler.
For something to be inside the genepool, and have its own "genetic disease", it needs to be passed along. So once paired with the right combination, it triggers, and you have a surfaced gene disease. So there is MDM, and there is carrier MDM.
The person you are replying is failing to bring along this component to the discussion, and is the core reason his argument is flawed.
Because he fails to address carrier MDM, where the carrier only can pass it on:
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An alternative is to require people receiving this treatment to consent to forced sterilization
Ah, you want to remove undesirables from the gene pool. I think the 1930s called and want their genetic hygiene back. Have you looked at what hospitals do today already? We try to fix everything, no matter how poor the fertility is, how high the tendency to miscarry, how unfit the mother is to give vaginal birth, how premature the child is born and no matter what kind of physical and mental handicaps or hereditary diseases they're born with or what health problems they have as a child. All those poor genes
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Also make sure to say how hardcore you'd get, like is it muscular dystrophy or gluten allergy that's enough to disqualify you.
Forget gluten, what about peanuts?
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Forget gluten [allergy], what about peanuts?
Shoot on sight. Take no prisoners. Also I'm totally not allergic to peanuts. *throws smoke bomb*
Re: Will this repair the genes in the gametes? (Score:2)
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If we now use gene therapy to remove the negative symptoms of the disease, but without repairing the damaged gene sequence in the gametes, parents with MD will end up passing the disease on to their children. And eventually that sequence will end up spreading throughout the entire population.
Duchene Muscular Dystrophy is a recessive condition carried by the X Chromosome.
https://www.genome.gov/19518854/learning-about-duchenne-muscular-dystrophy/
We already have this now, and the genes haven't spread t
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And what if the MD variant is a genetic transition state towards a much more efficient/capable genetic variation? Or what if there's a linked gene that is hugely beneficial, and given enough people it will find more variations that stabilize to something survivable? You can't select until you have the variations and combinations. Having more variations of human genes survivable and reproducible just gives us more to select from when we need the adaptation in the next selection event. So no, eugenics isn't j
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1) In order for defective MD genes to crowd out healthy genes as you suggest, the treated patients would have to be reproductively superior to people who didn't need the treatment in the first place. Is a treated MD patient going to have more children and grandchildren then the average? Note that; as you say, MD is the result of a rare genetic defect, so the healthy people have one hell of a head start as it is.
2) Anyone with access to the sort of early geneti
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This is ridiculous on TWO fronts.
1. The "situation" of a human being having a severely disabling and life threatening condition should be treated like any other medical "situation". Would you propose type I diabetics go without insulin and die, or children with leukemia go without care because there is a genetic component to these diseases? Their offspring may be more likely to have these same diseases, but they will also be in the same or better situations to receive treatment than their afflicted parents.
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And eventually that sequence will end up spreading throughout the entire population.
I suppose one could argue that long before it becomes an issue we would have mastered the technology to the point that we make it go away. Also people would have to agree to forgo kids without assistance. Fixing one cell seam like a much lower goal to set. Or you can watch them die.
But genie is almost out of the bottle in a much more disturbing way. I would suggest to start preparing for fallout. The whole idea is just too tempting to forego or even wait. It is happening. Now that we have the tools. Ready
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I don't know, I was rather looking forward to world with lots of muppets.
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Don't get too excited though, most medical problems cannot be boiled down to a single gene. Most genes have been found to do many things and interact with hundreds and thousands of others. Even the yeast genome, one of the simplest we know has been found to be far more complex than we thought [plos.org] in how genes result in particular traits. In the human genome it is likely that thousands of genes contribute to particular phenotypes. Some people seem to think organisms are like little machines a human might design,
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>"but everyone should be in favor of these modifications that can transform people's lives from suffering to joy and freedom."
I can totally understand why some people are worried about tampering with human DNA. The worst case scenario with doing so is screwing up the collective gene pool. But there is another alternative- in cases of severe disease, one option would be to sterilize the person getting the gene treatment so they can't have any children. That way the risk is only confined to the consenti
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You're a dick. Yes, let's let millions of people suffer needlessly while we pontificate about whether or not helping them is the "right thing" to do.
Who can afford such life-giving changes?
We can.
Where do we stop with the ability to decide life or death for millions of people en masse?
I suspect you'd see this differently if you or your child had a crippling condition that this could technology be used to mitigate or cure.
What ethical background do you base your simplistic "we must help" dogma upon?
I would ask you the same. What kind of "ethical background" allows a person to blithely ignore the suffering of millions of people?
Bill Hamilton would be optimistic now (Score:3, Interesting)
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Planet of the Apes suggest this will not end well (Score:1)
... successfully used CRISPR to correct the genetic defect responsible for Duchenne muscular dystrophy in four beagles ...
The Planet of the Apes movies suggest this will not end well.
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... successfully used CRISPR to correct the genetic defect responsible for Duchenne muscular dystrophy in four beagles ...
The Planet of the Apes movies suggest this will not end well.
If it was beagles we could at least hide in the trees.
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... successfully used CRISPR to correct the genetic defect responsible for Duchenne muscular dystrophy in four beagles ...
The Planet of the Apes movies suggest this will not end well.
If it was beagles we could at least hide in the trees.
Sorry, obscure movie reference. And maybe my recollection is confused but I think there was a global pandemic that killed off all dogs and cats, people turned to primates as a substitute.
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Yeah, you're confused, in the movie it was apes, in the story it was beagles.
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My comment was not over my head, but perhaps your own comment was over your own head?
You made a movie reference. I bridged the gap between what happened in the movie, and what happened in the actual fucking story we're talking about. Those are my comments, not yours. If they went over somebody's head, why would you start with me? I might have even understand what I was saying!
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Excellent! (Score:1)
That's unfair (Score:2)
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Like that dog that helped prove how the lung works! Dogs always get the good stuff first.
https://www.drlindseyfitzharri... [drlindseyfitzharris.com]
Reimbursement Model (Score:1)
"We are going for a cure, not a treatment,"
The first thing a Medical Industry executive will ask when someone makes an assertment like the above is "What is the reimbursement model?"
Because drug and medical device makers view treatment or cure of a patient as a side effect.
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Yes, and that's a problem with the US medical system, where government regulation has killed pretty much all competition. Specifically, the the "medical industry executive" will ask "how much does the government let us get away with charging for this, and who in government can we bribe to increase that".
In a free market, what "medical industry executives" will ask is: "c
Longer life span (Score:2)
Screw human treatments, please make dogs live as long as horses. Thanks.
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Screw dog treatments. Please make cats less like jerks.
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