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Medicine Science Technology

US Scientists Try 1st Gene Editing in the Body (apnews.com) 74

Marilynn Marchione, reporting for Associated Press: Scientists for the first time have tried editing a gene inside the body in a bold attempt to permanently change a person's DNA to cure a disease. The experiment was done Monday in California on 44-year-old Brian Madeux. Through an IV, he received billions of copies of a corrective gene and a genetic tool to cut his DNA in a precise spot. "It's kind of humbling" to be the first to test this, said Madeux, who has a metabolic disease called Hunter syndrome. "I'm willing to take that risk. Hopefully it will help me and other people." Signs of whether it's working may come in a month; tests will show for sure in three months.
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US Scientists Try 1st Gene Editing in the Body

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  • Dystopian Sci-Fi (Score:3, Insightful)

    by null etc. ( 524767 ) on Wednesday November 15, 2017 @03:01PM (#55556695)

    I tend to view news of such "wonderful and exciting" advances through the lens of wonderment, tinted with cautious fear. Can you imagine that through an IV, someone change the fundamentals of who you are, perhaps against your will? Someone could kidnap and drug you, and months after you wake up with an IV bag attached to you arm, you literally start becoming someone else.

    Are you too rebellious and anti-authoritarian? Here, have a timidity cocktail. Are you too smart and logical, and impervious to manipulation via base desires? Here, have the Trump cocktail.

    • by Merk42 ( 1906718 )
      If it's that dystopian, they could just, you know, have the person killed.
      • Killing is extreme (Score:3, Insightful)

        by lamer01 ( 1097759 )
        It would take 18-20 years to grow a replacement for the assasinated person. Why not just reform that person to someone more suited to what 'they' want them to be.
      • If it's that dystopian, they could just, you know, have the person killed.

        That could be a waste of *resources* (resources being you).

        • Usually it's just communist and socialist governments that view people as a resource, hence ID cards that tell the cops where you're supposed to be during working hours with truancy laws against people not showing up for work, and building walls that they claim are to keep fascists out while the real purpose is to keep its own citizens from leaving.

      • Re:Dystopian Sci-Fi (Score:4, Interesting)

        by interkin3tic ( 1469267 ) on Wednesday November 15, 2017 @03:49PM (#55557059)
        Mod parent up. Biology is just not that simple. The genetics behind most brain DISEASES isn't complicated enough to begin to solve like this. Behavior is exponentially more complex than, say, alzheimers.

        There isn't a gene for "be republican" or even "be smart." There are genes that are known to lead to low intelligence, but your'e talking down syndrome level dysfunction.

        On top of that, the vast majority of brain development is done in utero.

        Suggesting that a dystopian government could simply reprogram a person using crispr is an order of magnitude more ludicrous than saying "Well what if a rogue state hacks the internet and makes all the planes fall out of the sky."

        At the very least, a dystopian government even GIVING YOU CANCER with crispr would require millions of dollars in investment.

        Rope, tape, a chair, and a baseball bat meanwhile costs maybe $10 if you go to a garage sale and I'm guessing $10k to hire some biker types?
        • Mod parent up. Biology is just not that simple.

          That's actually not true. For example, see how the DRD4 gene could impart a propensity for risky behavior. This study [newswise.com] was performed almost a decade ago. I have no doubt that further studies would reveal specific genes that could be manipulated to entirely change the behavior of a person.

          • First off, that's correlation, not causation, which is a big issue in genetics. See below.

            Second, the effect is slight. [psu.edu]

            Third, as I said, brain development happens in utero. Changing genetics to rewire the brain of an individual you want to influence is nonsense.

            I have no doubt that further studies would reveal specific genes that could be manipulated to entirely change the behavior of a person.

            It's an understatement to say it's tough to quantify human behavior (hence the misleading study saying DRD4 is a risk-causing gene), but lets take it for granted you can tell if a person is going to have the desired behavior.

            The necessary

        • by Mkkby ( 4973999 )
          It's just code with a 4-letter chemical alphabet. While it may be difficult and expensive NOW, imagine the world in 100 years. Or 10,000.

          How long before those chemicals can be designed in CAD and precisely laid out by something akin to a 3-D printer? In utero, you say. Perhaps someday in printero.
        • by PKFC ( 580410 )

          Obligatory C&H (if that's a thing): http://explosm.net/comics/556/ [explosm.net]

      • If it's that dystopian, they could just, you know, have the person killed.

        That's the ultimate goal...comprehensive embryonic screening...detect and abort the freaks before they have a birth certificate mandate to drain our resources

    • That's too small. As a dystopian despot, what you'd really want to do would be take the pacifying cocktail and put it in a flu virus, immunize your elite subpopulation (may as well pacify your own proletariat) and drop it on everyone else.
    • This has already been done surgically -- lobotomies were used for social control in the 1940s through 60s. Read about what happened to Frances Farmer. (And no, it wasn't considered major brain surgery -- they literally did it using a tool through the eye socket: scary stuff.)
    • If I understand TFA the gene being edited codes for an enzyme that sufferers lack. This is pretty much the simplest level of genetic manipulation and anything much above that I suspect would have to be done in utero to be effective. There's so much interdependence among systems within an organism I doubt we are anywhere near (centuries perhaps) tinkering with fully developed beings to change their attributes. It just doesn't work like star trek where "altering your dna" turns you from a human to a frog.
    • Adenovirus therapy caused the death of Jesse Gelsinger [wikipedia.org] in 1999.

      The resulting moratorium caused extreme damage to the the field of gene therapy, the institutions involved in it, and the careers of those practicing and studying it.

      It has also been recently proven that CRISPR causes hundreds/thousands off-target changes in mice [geneticlit...roject.org].

      This seems rash.

      • Bank some gametes for the subject and worst case scenario they get cancer. Now that isn't great, but if it is better than no treatment for whatever disorder they have I'm comfortable with them being allowed to make an informed choice.
        • by emil ( 695 )
          The problem arrives when a few successful treatments in clinical trials which do not (immediately) reveal larger issues when deployed. Haste makes waste, as the adenovirus researchers can attest.
          • That's true for bulk treatments. I think any genetic modification is going to be so niche by necessity I'm not sure how you'd be able to make any of them at scale.
    • How much of you is genetics? And which gene in what sequence represents given traits?

      Actually finding genes that represent physical traits is at best difficult but possible. Altering genes which make up core personalities is difficult be a use of nature vs nurtures very real.

      Read up on gut bacteria and how that affects your metabolism, and weight and multiple that by a 1000 before we start drastically editing personalities with predictable results.

      We are century away from that kind of worry.

      • Altering genes which make up core personalities is difficult be a use of nature vs nurtures very real.

        No, that's definitely not the reason.

        The reason is that, once grown your medial frontal gyrus, which makes the "what/when/where" go/no-go decisions is already grown.

        Unless you intend to dike it out and grow a new on in its place, those genes have already been spent to create tissue that has a particular preference for function.

        Just like the hippocampus and amygdala would have to be diked out and regrown, in order to change someone base sexuality.

        • Not necessarily. If something can be changed by adding more cells in a particular place, it might be possible to turn cell division back on in that part of the brain, or trigged early apoptosis of undesired cells. True rewiring, of course, is a bit farther out of reach.
        • The reason is that, once grown your medial frontal gyrus, which makes the "what/when/where" go/no-go decisions is already grown.

          It's not quite that simple. Changing genes in a developed brain isn't going to change gross structure, but it could well change low-level biochemical behavior, perhaps changing the levels of specific neurotransmitters, or changing the way that the brain forms new connections or breaks old ones.

          It seems unlikely that gene editing could turn a Republican into a Democrat, but it doesn't seem so unlikely that it could turn a happy person into a severely depressed one, or maybe seriously decrease (or increase?

          • The reason is that, once grown your medial frontal gyrus, which makes the "what/when/where" go/no-go decisions is already grown.

            It's not quite that simple. Changing genes in a developed brain isn't going to change gross structure, but it could well change low-level biochemical behavior, perhaps changing the levels of specific neurotransmitters, or changing the way that the brain forms new connections or breaks old ones.

            It seems unlikely that gene editing could turn a Republican into a Democrat, but it doesn't seem so unlikely that it could turn a happy person into a severely depressed one, or maybe seriously decrease (or increase?) the ability to form new long-term memories, etc. Of course, those same things can be done with drugs.

            It's highly unlikely that the gene expression can be permanently changed at that level. You could certainly damage organelles to achieve that effect -- long term Marijuana and LSD use is known to permanently alter brain chemistry -- it's just not at a genetic level.

            All our current crop of Alzheimers and vascular dementia drugs operate through direct action, rather than indirect action.

            I understand that some people believe in epigenetic effects, but the only place they've been demonstrated is on rather simp

    • Are you too rebellious and anti-authoritarian? Here, have a timidity cocktail. Are you too smart and logical, and impervious to manipulation via base desires? Here, have the Trump cocktail.

      Considering we don't even know which genes make people tall or short, I'm not concerned.

    • Someone could kidnap and drug you, and months after you wake up with an IV bag attached to you arm, you literally start becoming someone else.

      Or something else.

    • by Agripa ( 139780 )

      I tend to view news of such "wonderful and exciting" advances through the lens of wonderment, tinted with cautious fear. Can you imagine that through an IV, someone change the fundamentals of who you are, perhaps against your will? Someone could kidnap and drug you, and months after you wake up with an IV bag attached to you arm, you literally start becoming someone else.

      Are you too rebellious and anti-authoritarian? Here, have a timidity cocktail. Are you too smart and logical, and impervious to manipulation via base desires? Here, have the Trump cocktail.

      Unlike in Hollywood, ontogeny recapitulates phylogeny.

  • $vi Brian Madeux

    :%s/badgene/goodgene/g

    :wq

    He's all cured.

  • by jddj ( 1085169 ) on Wednesday November 15, 2017 @03:41PM (#55557015) Journal

    They're just going to get people pissed off when they dissect the guy for the research paper.

  • I really and truly hope this works because it will open the door for creating better lives for people with other genetic diseases. Cures are always better than medicines to mitigate symptoms!
  • If this works, as it gets applied to more genetic diseases it's going to save a lot of lives, including my DMD son.
  • ... but since idea is "BRILLIANT" let's post it right away.

  • nothing wrong with being a mutant and the first real X-men
  • According to Wikipedia, the first successful nuclear gene transfer in humans was performed in 1989, and more than 2,300 clinical trials have been conducted since then. We are just getting the methods perfected and closer to mainstream.
  • and autoimmune disorders. Your doctor may prescribe AIDS to treat them.

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