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posted by martyb on Tuesday June 30 2020, @09:04PM   Printer-friendly
from the make-it-retroactive dept.

A ‘Cure for Heart Disease’? A Single Shot Succeeds in Monkeys:

In the first gene-editing experiment of its kind, scientists have disabled two genes in monkeys that raise the risk for heart disease. Humans carry the genes as well, and the experiment has raised hopes that a leading killer may one day be tamed.

“This could be the cure for heart disease,” said Dr. Michael Davidson, director of the Lipid Clinic at the University of Chicago Pritzker School of Medicine, who was not involved in the research.

But it will be years before human trials can begin, and gene-editing technology so far has a mixed tracked record. It is much too early to know whether the strategy will be safe and effective in humans; even the monkeys must be monitored for side effects or other treatment failures for some time to come.

The results were presented on Saturday at the annual meeting of the International Society for Stem Cell Research, this year held virtually with about 3,700 attendees around the world. The scientists are writing up their findings, which have not yet been peer-reviewed or published.

The researchers set out to block two genes: PCSK9, which helps regulate levels of LDL cholesterol; and ANGPTL3, part of the system regulating triglyceride, a type of blood fat. Both genes are active in the liver, which is where cholesterol and triglycerides are produced. People who inherit mutations that destroyed the genes’ function do not get heart disease.

[...] Not only did the system work in 13 monkeys, the researchers reported, but it appeared that every liver cell was edited. After gene editing, the monkeys’ LDL levels dropped by 59 percent within two weeks. The ANGPTL3 gene editing led to a 64 percent decline in triglyceride levels.

One danger of gene editing is the process may result in modification of DNA that scientists are not expecting. “You will never be able to have no off-target effects,” warned Dr. Deepak Srivastava, president of the Gladstone Institutes in San Francisco.

In treating a condition as common as heart disease, he added, even an uncommon side effect can mean many patients are affected. So far, however, the researchers say that they have not seen any inadvertent editing of other genes.

Another question is how long the effect on cholesterol and triglyceride levels will last, Dr. Davidson said. “We hope it will be one-and-done, but we have to validate that with clinical trials,” he said.


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  • (Score: 3, Interesting) by maxwell demon on Wednesday July 01 2020, @09:54AM (8 children)

    by maxwell demon (1608) on Wednesday July 01 2020, @09:54AM (#1014919) Journal

    One would think that negative genes would tend to get eliminated over time by evolution, unless they also bring a positive effect. For example, it is no accident that sickle cell anaemia is most prevalent in malaria regions: While having two of the genes is disastrous, having only one of them gives some protection against malaria. Also, the reason why humans aren't immune against HIV is a genetic mutation that helped against another viral decease that got extinct millions of years ago. Also, I remember having read that people who are more likely to get diabetes when on a sugar-rich diet are better adapted to lack-of-food scenarios.

    So the question is: What is the positive effect of those “heart-decease genes”, and is that positive effect still relevant today? That is, if we switch off those genes, do we indeed get a net improvement, or do we pay the protection with a side effect that's worse?

    --
    The Tao of math: The numbers you can count are not the real numbers.
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  • (Score: 0) by Anonymous Coward on Wednesday July 01 2020, @10:17AM (3 children)

    by Anonymous Coward on Wednesday July 01 2020, @10:17AM (#1014927)

    By the time it's a problem, you should have already reproduced.

    • (Score: 2) by maxwell demon on Wednesday July 01 2020, @10:45AM (2 children)

      by maxwell demon (1608) on Wednesday July 01 2020, @10:45AM (#1014936) Journal

      If the genes were selected for, that is unlikely. Because otherwise they would not have been selected for to begin with.

      --
      The Tao of math: The numbers you can count are not the real numbers.
      • (Score: 2, Insightful) by Anonymous Coward on Wednesday July 01 2020, @12:36PM (1 child)

        by Anonymous Coward on Wednesday July 01 2020, @12:36PM (#1014974)

        The point is they wouldn't be selected for, but they also wouldn't be selected against, since it only manifests after you've already reproduced. Humans have hit more and more of those as average lifespan has increased, and will surely continue to find more in the future.

        • (Score: 2) by maxwell demon on Thursday July 02 2020, @12:25PM

          by maxwell demon (1608) on Thursday July 02 2020, @12:25PM (#1015356) Journal

          Since production of the enzymes costs energy, their production would be selected against even if they had no effect at all.

          --
          The Tao of math: The numbers you can count are not the real numbers.
  • (Score: 2, Interesting) by Anonymous Coward on Wednesday July 01 2020, @10:55AM (1 child)

    by Anonymous Coward on Wednesday July 01 2020, @10:55AM (#1014942)

    The issue is cholesterol and triglyceride. Those are required for your body to function. However when they're oxidized they damage blood vessels which causes the heart issues. Sadly this 'cure' is completely the wrong approach. From my non-medical understanding, it aims to reduce the amount of cholesterol and triglyceride in your blood. Thus the levels of oxidized cholesterol will also decrease thus vessel damage is decreased. Sadly this is treating the symptom not the cause, like trying to fix a wobbly ride from a flat tire by reducing the air pressure of all the good tires to match the flat one. The correct fix is to fix the damaged tire. For cholesterol, the correct fix is to stop oxidizing it. That is done by reducing stress, avoiding carbohydrates, and absorbing your fat soluble vitamins. Thus you need to eat more meat and fat and cut out sugar and carbs. But pizza and cake are too exciting so no one does that. Thus we're going to screw up our body's ability to move nutrients around (what cholesterol does) because we're too scared to eat fat. This 'cure' is going to have horrible long term effects. It's a "feels good fix" like skim-milk with added Vitamin D. You need fat to absorb Vitamin D which isn't present in skim-milk, so that added D is mostly unabsorble to your body.

    In terms of all those studies you hear about how eating meat is going to kill you, that's true. If you eat tons of meat and tons of carbs you'll get a ton of oxidized cholesterol. However if you cut out those carbs and just eat a ton of meat, you'll feel the best you've ever been in your life.

    The main point being those genes are only considered bad because we're eating improperly. Having more non-oxidized cholesterol is a good thing. Instead of eating like we evolved to we're going to perform genetic experiments on ourselves. Humans are crazy.

    • (Score: 0) by Anonymous Coward on Wednesday July 01 2020, @04:06PM

      by Anonymous Coward on Wednesday July 01 2020, @04:06PM (#1015047)

      Red meat comes with mostly saturated fats, which is not optimum. To improve on that, people should in addition consume monounsaturated fats (e.g. olive oil) and cis-polyunsaturated fats, both omega-6 and omega-3 (salmon, walnuts).

  • (Score: 2) by legont on Wednesday July 01 2020, @12:13PM

    by legont (4179) on Wednesday July 01 2020, @12:13PM (#1014963)

    What is the positive effect of those “heart-decease genes”, and is that positive effect still relevant today?

    The carriers fuck and fight better until sick. Having them makes lots of sense with life expectation below 50.

    --
    "Wealth is the relentless enemy of understanding" - John Kenneth Galbraith.
  • (Score: 0) by Anonymous Coward on Thursday July 02 2020, @12:41AM

    by Anonymous Coward on Thursday July 02 2020, @12:41AM (#1015219)

    Oops, I posted this as a response to another post when I meant to post it here.

    There was a whole discussion about this on Slashdot. The discussion is actually quite similar to the one here but might still be worth reading about.

    https://science.slashdot.org/story/20/06/28/1510204/a-cure-for-heart-disease-a-single-shot-succeeds-in-monkeys [slashdot.org] [slashdot.org]