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CRISPR Gene Editing Fixes Muscular Dystrophy In Dogs, Humans Could Be Next (time.com)
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."
We have the technology to change the mistakes introduced by the random processes of our evolution, so it is our moral imperative to use these tools to fix the diseases affecting humanity. If you are paranoid stick a million samples of human DNA in Svalbard, and another in an orbiting satellite, but everyone should be in favor of these modifications that can transform people's lives from suffering to joy and freedom.
Genetic engineering improved beyond everything Hamilton could have dreamed of.
This sounds like fixing a weak building wall by changing the blueprints. That does not affect the existing building at all.
The rich people will control this though. So they will make the poor even more gullible and what you end up with will make you wish you had Trump back.
... 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.
Humanity 2.0
The x.0 release are always buggy, its best to avoid them.
I can't wrap my mind around how I feel about this. I'm not afraid of technology, and yet this seems ridiculously risky. Plus the world that we live in is simply not devoid of biased, corporate or self-serving interest. Ew. It hurts to try to wrap my brain around it but if, say, a corporation ties its bottom line to gene editing, well, that could get a little ugly. This is simply wild. Even if we do simply use this to remove things we deem as suffering.. what will be the side effects?
Excellent! Soon the creatures we can engineer will make the regular human look like nothing more than a dog with muscular dystrophy in comparison. And it's not ethical to breed dogs with muscular dystrophy.
We have the technology to change the mistakes introduced by the random processes of our evolution
If we are to achieve Humanity 3.0 we ought to use what we have to eradicate STUPIDITY
Gametes = sperm and egg cells. If you're not fixing the genetic defect in those, then this will actually make the situation worse. Previously, people with MD were less likely to reproduce because of the disease (it usually manifests between age 2-15, with most afflicted persons dying by their 20s). That kept the damaged gene sequence relatively rare in the population's gene pool. 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. And we'll end up with a world where the a large percentage of people need this therapy just to have a normal life. According to TFA, this treatment has only been applied to muscle tissue (where the bad gene sequence is needed by the muscles).
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).
Why is it that the dogs always have to get the good stuff first?
Slashdot, fix the reply notifications... You won't get away with it...
"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.
Screw human treatments, please make dogs live as long as horses. Thanks.
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: He can't argue for what this do the genetic pool.
And because he can't argue for the impact his talk of sterilization or sustainable treatable damage caused by MDM becoming a common treatable disease in infancy.
His argument also indirectly talks about access to medical care, and the technology needed to treat MDM. Or the costs by making treatment common without addressing the hereditary concerns.
... 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.
... 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.
Yeah, you're confused, in the movie it was apes, in the story it was beagles.
This worked on 4 beagles "bred with the disease-causing gene."
Questions:
1. is the gene that was bred exactly the same as the "wild" gene in humans?
2. are 4 beagles enough to come to a conclusion? (Answer, uh, no.)
3. does this gene also do something beneficial that will cause harm if altered?
A well known example of #3 is that the gene that causes sickle cell anemia also confers in some people some resistance to malaria.
On over enthusiasm with new treatments: In the book The Emperor of All Maladies: A Biography of Cancer, Siddhartha Mukherjee
writes how desperate people glom onto new cancer treatments, in some cases disrupting scientific studies. In one case, a "double blind" study of a drug was turned worthless. It seems nurses and doctors figured out which group was getting a placebo and which group was getting the drug. It was no longer a blind test. So they snuck in people, friends, relative, patients, strangers who they know had cancer into the group that received the real drug. This wasn't discovered until after the test was completed. This made the test pretty much worthless, as patients who got the drug or the placebo were no longer randomly selected samples.
In Ireland in the late 1980's there was a study to see if folic acid given to pregnant women prevented spina bifida in newborns. When the preliminary phase results came in it was shown to greatly reduce spina bifida. It was so good the ethics committee decided it would be unethical to continue the test (denying the control group the folic acid) and recommended all pregnant women get it.
In the USA when this was presented to groups like the March of Dimes, the prejudice against fantastic claims for mere vitamins kicked in. It was met with scorn. It was also met with the fact that the Irish study ended after preliminary testing, which was not good enough. And possibly some chauvanism--US researchers sometimes dismiss science studies whose results they don't like that were not done in the US. Eventually more studies were done, some in the US, and in the early 2000's US policy was changed to recommend all pregnant women get folic acid. Folic acid is now added to more foods and vitamin formulas than before.
Folic acid worked. But a lot of drugs (and supplements) after deeper and careful research research end up not working.
Conclusion: this MS research may represent a breakthrough. And if it were me or my child with MS I would be chomping at the bit to get it. But rushing forward without a heck of a lot more research? Hard decisions.
Nope, its still over your head. My confusion about all the dogs dying in the movie (70s) was unfounded, I was correct. The connection you are failing to make is that one of this risks of genetic engineering is things going wrong, unintended consequences. Genes don't always control one thing, they often affect many different things. To fix one thing in one spot may mess up something somewhere else. Its not a given those beagles will be as healthy as they first seem, they may be prone to other problem now.
Again, my bad for making too obscure a reference, expecting readers to connect the dots.
Genetically engineering dogs --> someone f's up --> the dogs all die --> Planet of the apes.
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!
Actually, I understood your comment and the direction you were coming from. And the conversation is still over your head. Focus on errors and unintended consequences of genetic engineering and a potential catastrophe occurring in the species. Then perhaps you will see the connection you are missing. Again, my fault for making such an obscure and subtle comment. But lets not pretend you are getting anything yet, your "bridge" does nothing more that demonstrate you are not getting it.
My son has this disease. I pray for this cure.