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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."

13 of 112 comments (clear)

  1. Humanity 2.0 by Jzanu · · Score: 3, Informative

    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.

    1. Re:Humanity 2.0 by 93+Escort+Wagon · · Score: 2, Insightful

      Now comes the equally important but less sexy, longer-term study to see what collateral damage might be caused by this treatment.

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    2. Re:Humanity 2.0 by klingens · · Score: 2, Interesting

      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, it's mainly "but the people only need to eat a little more vegetables". Which comes from dumb rich western organizations who cannot comprehend that the poor asians simply don't have the money to buy this. There are no walmarts or rather Whole Foods supremarkets in rural Asia.

      Medical genetic engineering is different. There you have insulin for diabetics and erythropoetin for anemic people. Granted, most of the erythropoetin is used for doping in sports, but it's still a godsend for anemic people on dialysis: no more 16h a day sleep. And for diabetics insulin a literal live saver.
      Both of these drugs are wholly accepted in any society, even with the doping problem. So another genetic tech which saves lives will be accepted too

    3. Re:Humanity 2.0 by religionofpeas · · Score: 4, Insightful

      Start by treating the worst diseases, and we'll find out. I'm sure we'll be able to find volunteers.

    4. Re:Humanity 2.0 by Anonymous Coward · · Score: 2, Informative

      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 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, but they are not, they evolved, and the complexity is mind boggling, as evolution only cared about whether things worked, and did not require modularity to generate the design.

    5. Re: Humanity 2.0 by Zero__Kelvin · · Score: 2

      If it removes a general only found in MD sufferers I wouldn't be too worried about that.

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    6. Re:Humanity 2.0 by morethanapapercert · · Score: 2
      Who can afford this? Anyone fortunate enough to live in a country with universal health care. (roughly thirty countries, most are in the G20) Also any one without universal health care but at least have a sane and rational health insurance provider. Giving a 5 year old boy a CRISPR treatment is going to be SIGNIFICANTLY cheaper than paying for all the supports he would other wise need in his life. (as a side note, most boys with DMD die in their twenties from cardiac or respiratory failure because the muscles involved are no longer strong enough function. My youngest son has DMD)

      Where do we stop with the ability to decide life or death for millions? I have no idea. But I would suggest that if a society can make a choice that makes the difference between life or death for mass numbers of people, it would monstrous to chose the option that leads to death. Allowing the sick and disabled to suffer and die simply because they are unfortunate to have a condition which is treatable is one of the more common legs to arguments for eugenics.

      What is the ethical basis? It can be expressed very simply: Imagine I have a vaccine that prevents smallpox, a disease which used to kill millions, but I chose not to make it available, or special interest groups prevent me from making it available. Then your son contracts smallpox and dies. Who is responsible for his death? If I can save a life and choose not to do so, then I am at least partly responsible for that death. I support the right to die with dignity and the death penalty. I am comfortable with accepting the responsibility for helping to ease the suffering of the patient and the joint responsibility of when society decides, after due process and a fair trial and appeals process, to execute a criminal. I am NOT comfortable watching children die of preventable or treatable conditions.

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    7. Re:Humanity 2.0 by morethanapapercert · · Score: 3, Interesting
      You're right. My son has DMD, the very disease this treatment is aimed at. I'd volunteer my son for this in a fucking heartbeat because the alternative is seeing him wither away and spend years struggling to even breathe before dying in his late 20's from cardiac or respiratory failure. Unfortunately, my son is already permanently wheelchair bound. He is already considered to have declined to far to be a viable test subject. That is a heart breaking disappointment we've had to swallow several times now as he keeps falling outside the study requirements for several different life extending trial therapies even as those trials accept worse and worse patients.

      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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  2. Bill Hamilton would be optimistic now by SurenEnfiajyan · · Score: 3, Interesting

    Genetic engineering improved beyond everything Hamilton could have dreamed of.

  3. Will this repair the genes in the gametes? by Solandri · · Score: 5, Interesting

    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).

    1. Re:Will this repair the genes in the gametes? by Kjella · · Score: 2

      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 are being passed on and none of those children are asked to sign away their right to reproduce, there's nothing unique about muscular dystrophy that is significantly worse or different.

      If you want to argue we should be screening out who gets to reproduce or not, then just go full tilt. At age... 14? everyone has their medical history, physical and mental health reviewed, those who pass go through and the rest are sterilized/given a vasectomy. A license to breed, more or less. Also make sure to say how hardcore you'd get, like is it muscular dystrophy or gluten allergy that's enough to disqualify you. I'm guessing you're not very likely to get much support, but hey... that's kinda what you really want with this "if we do this for you, you agree not to have children" right? And I'm sure there would be no potential for abuse or corruption in such a system, not at all...

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    2. Re:Will this repair the genes in the gametes? by TeknoHog · · Score: 2

      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.

      The GP asks: Should we allow heavy medical procedures that, while helping individuals, will make humanity more dependent on these procedures in the future? There's no easy answer.

      In fact, it's a GM issue and we've already seen something like it with food production. GM promises to increase yields and make food cheaper, but at the same time it makes us more dependent on certain technology. Personally, I'm OK with GM foods, as long as we don't let them destroy current biodiversity, so we can always back up to the old ways if necessary. Something similar should apply here too.

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  4. Re:Humanity 3.0 by guruevi · · Score: 2

    Why are you promoting genocide of leftists?

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