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Biotech Medicine Science

Fixing Faulty Genes On the Cheap 105

An anonymous reader sends an article about CRISPR, a system for modifying genes and moving them from cell to cell. It's notable because the cost to do so is dropping to the point where it's becoming viable to use on a patient-by-patient basis. CRISPR is one of those interesting inventions that comes, not from scientists explicitly trying to cure a disease, but from researchers trying to understand something fundamental about nature. Jennifer Doudna's research at the University of California, Berkeley has focused on how bacteria fight the flu. It turns out bacteria don't like getting flu any more than the rest of us do. Doudna says the way bacteria fight off a flu virus gave her and her colleagues an idea. Bacteria have special enzymes that can cut open the DNA of an invading virus and make a change in the DNA at the site of the cut — essentially killing the virus. Doudna and other scientists figured out how this defense system works in bacteria; that was interesting all by itself. But then they realized that they could modify these enzymes to recognize any DNA sequence, not just the DNA sequence of viruses that infect bacteria.
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Fixing Faulty Genes On the Cheap

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  • by i kan reed ( 749298 ) on Friday June 27, 2014 @01:18PM (#47334301) Homepage Journal

    Cheap almost certainly means "cheap enough to be on par American medicine" not any sort of actual definition of the term.

    • But I'd bet there are fans of fictional anthropomorphic animals who would be willing to pay American-medicine prices for gene therapy to look more furry.
      • Look, if there's one thing American Medicine beats the entire rest of the world yet, it's exploiting peoples' body image dysphoria to charge lots of money for unnecessary operations.

        (I'm a little scared that I'll offend some transsexual people by using the word dysphoria, but that's life, I guess)

        • by Kaenneth ( 82978 ) on Friday June 27, 2014 @01:52PM (#47334559) Journal

          As a trans-offended person, I'm offended by your reluctance to allow people to be offended.

          Some of us enjoy being shocked, offended, and triggered so stop cis-comfort-zone oppressing us.

        • it's exploiting peoples' body image dysphoria to charge lots of money for unnecessary operations.

          (I'm a little scared that I'll offend some transsexual people by using the word dysphoria

          Random pedantry, but you've conflated two different things.

          There is Body Dysmorphic Disorder [wikipedia.org], where people are really really concerned about the appearance of their pieces and parts. This is the broader one which includes why people get 30 plastic surgeries.

          There is Gender Dysphoria [dsm5.org], in which the pieces and parts don't match internal identify.

          I've known a few women (and a few men come to think of it) who fell into the former. It's not vanity, it can be a clinical issue where it becomes debilitating for you.

          I've also met a couple of people in the latter category -- and trust me, nobody would go through all of the stuff they do unless they were really really committed to it and felt they had no choice. It's hardly a glamorous (or easy) thing to do.

          • I wonder to what extent having all these disorders officially classified in the DSM is just a way for the medical profession to charge insurance companies for absolutely everything that goes wrong with someone.

            What else is really gained by these classifications, except profit for the medical industry and a hell of a lot more people walking around feeling like victims of horrible diseases.

            Oh, I guess the victimization industry also does pretty well by creating non-profit organizations that raise all sorts of money to "raise awareness" (whatever the hell that means) for their particular newly-classified disorder.

            Tell the truth, the entire in-bred confluence of people who desperately want to feel like there is a reason for their unhappiness that they can point to, and the non-profit outfits that profit from them, and the medical industry that apparently doesn't have enough really sick people to worry about that they want everybody to feel like they have some officially classified disorder seems a lot like a gigantic scan to me.

            Has the lot of people who believe they need to be better-looking at any cost really been made better by these classifications? I'm not sure.

            The only solution seems to be to stop thinking about one's self all the time and think about other peoples' welfare for a change. That does wonders for whole categories of dysphorias, I have found first-hand.

            • "Raising awareness" means informing the public that a cluster of symptoms is a distinct medical condition for which a distinct treatment or prevention exists or is being researched. For example, brushing teeth with a toothpaste is a protocol to prevent tooth decay, as is vaccination to prevent infectious disease. If a prevention or treatment exists, it's an invitation to see a professional to get screened for a condition so that one doesn't have to just suck it up and live with what had been believed to be "signs of aging" for the rest of one's life.

              Raising awareness is one of the three formats of direct-to-consumer prescription medication advertisements in the United States. The others are "reminders" that mention only the name of a drug, not a condition, and full drug ads that mention both the condition and the treatment but must also mention statistically discernible side effects and the name of a major national magazine in which the "package insert information" is published.

              • by Optali ( 809880 ) on Saturday June 28, 2014 @02:22PM (#47341369) Homepage
                "Exists or is being researched". An euphemism for "We don't have a fuycking clue but will continue massaging our stats until they look a bit better and until we are able to put some substance in the market that we can sell as wonder medicine"

                Until it's proven that thee medical wonder is nothing but snake oil, Just recall the antidepressants and the infamous ritalin.

            • by Anonymous Coward on Friday June 27, 2014 @03:53PM (#47335683)

              Then why do you troll others, & why're you running from answering a simple question? http://linux.slashdot.org/comm... [slashdot.org]

            • by Anonymous Coward on Friday June 27, 2014 @08:03PM (#47337763)

              Too bad we all see it, despite the downmod (along with your lies you're a PhD in English) http://slashdot.org/comments.p... [slashdot.org]

            • by Anonymous Coward on Friday June 27, 2014 @09:31PM (#47338187)

              You troll others like a bully that can dish it out but can't take it given back to him in return. See subject troll http://slashdot.org/comments.p... [slashdot.org] and you're failing at every turn here. You don't possess the intellect, wit, or intestinal fortitude necessary to take on your clear intellectual betters as seen all throughout this exchange and even steal their lines (despite your claim you are some 'great writer', which is clearly bullshit - you can't even spell right or use grammar properly) as you tried with apk's comeback to you that shut your puny mouth easily here http://slashdot.org/comments.p... [slashdot.org] troll. Don't like it? Too bad. It's truth in plain black and white, as well as your own blatant errors in what you *claim* you do, write. You're a fool. Lastly, no real martial artist looks for trouble the way you do. They KNOW anyone can go down and meet a superior opponent, especially if they are in the wrong. You are. You fail, loser. On all levels concerned here. I understand you completely and know you, better than you know yourself. You were picked on as a boy. It manifests itself in your trolling online. Too bad it got your ass kicked, yet again. Perhaps one day, you'll grow up, fool.

            • by Anonymous Coward on Friday June 27, 2014 @10:09PM (#47338371)

              "Tell the truth, the entire in-bred confluence of people who desperately want to feel like there is a reason for their unhappiness that they can point to, and the non-profit outfits that profit from them, and the medical industry that apparently doesn't have enough really sick people to worry about that they want everybody to feel like they have some officially classified disorder seems a lot like a gigantic scan to me." - by PopeRatzo (965947) on Friday June 27, 2014 @02:17PM (#47334761) Homepage

              It's spelled 'scam', not 'scan'. FTFY. So much for your alleged PhD in English. You can't write properly.

              You also repeatedly used "and" between items. Wrong.

              Giant run on sentences too? Please... lol!

              WTF was this mess also?

              "Has the lot of people who believe they need to be better-looking at any cost really been made better by these classifications? I'm not sure.." - by PopeRatzo (965947) on Friday June 27, 2014 @02:17PM (#47334761) Homepage

              Better looking is correct. No hyphen needed.

              Learn to write, and quit telling lies you're a PhD in English. You are most defintely not one after those errors above.

              Oh, & by the way: Keep "reacting" & downmodding this + my other posts (you're playing RIGHT into my hands like you no mind trolls always do). Think others are "fooled' by that, little midget? Guess again. I'll keep posting it to embarass your troll ass and perhaps, teach you a lesson you ought learn by - don't "f" with your betters, midget.

          • I haven't conflated those things. I was afraid someone would think I conflated those things. But I getchya.

          • by Anonymous Coward on Friday June 27, 2014 @02:52PM (#47335083)

            I've also met a couple of people in the latter category -- and trust me, nobody would go through all of the stuff they do unless they were really really committed to it and felt they had no choice. It's hardly a glamorous (or easy) thing to do.

            The same could be said of suicide. Don't try to tell me that people's feelings "like they had no choice" are in any way reflective of reality.

      • It would just become a political mess; Biofur would get mired in a war between the sweatshop garment workers' union and the hoover lobby.

      • by Immerman ( 2627577 ) on Saturday June 28, 2014 @01:33AM (#47339009)

        It's not just the furries who'd be up for animal-like body modification, I'd *love* to have a prehensile tail. How many times have you wished for an extra hand? And we've got the genetic blueprints from numerous relatively close relatives to guide us on how to add them gracefully - we may even carry many/most of the necessary genes already. Sure, it's not as dextrous as prehensile feet, but it can be used while walking. And besides I already have two hands, a third grasping appendage with a different set of strengths and weaknesses would add more options.

  • by Anonymous Coward on Friday June 27, 2014 @01:28PM (#47334361)

    fixing the genes of humanity in one fell swoop

  • How long before... (Score:4, Interesting)

    by tchdab1 ( 164848 ) on Friday June 27, 2014 @01:31PM (#47334387) Homepage

    ... you can go to a local independent chop shop and tell them "my phone says I have an extra guanine in my 14 chromosome and it's causing my food allergy to modified mangoes - can you get it out this afternoon?"

    • by Wonko the Sane ( 25252 ) * on Friday June 27, 2014 @01:34PM (#47334415) Journal

      Did you know human livers are a single broken gene away from maufacturing vitamin C from glucose, just like almost every other mammal?

      The liver perform every step in the process except the final one, because of a single transacription error that was introduced into the germline back in ancient times

      It would be cool to see what happens when they fix that.

      • by Anonymous Coward on Friday June 27, 2014 @01:45PM (#47334509)

        Orange piss, I reckon.

      • by BenSchuarmer ( 922752 ) on Friday June 27, 2014 @01:47PM (#47334523)
        It's more efficient to get Vitamin C from food. If it wasn't, that mutation would have been selected out of existence a long time ago.
        • by gstoddart ( 321705 ) on Friday June 27, 2014 @01:57PM (#47334595) Homepage

          If it wasn't, that mutation would have been selected out of existence a long time ago.

          OK, smartass, what is the evolutionary advantage for stupidity?

          Because you'd think we'd have selected that away a long time ago as well.

          Hell, we have an appendix. Why do we have an appendix? Why hasn't evolution made that go away?

          Evolution is awesome, but it can do some silly things that stick around.

          • by NEDHead ( 1651195 ) on Friday June 27, 2014 @02:02PM (#47334645)

            There is some evidence that the appendix acts as a reservoir of the gut biota to repopulate when the need arises due to illness (or excessive antibiotics, etc).

          • by sideslash ( 1865434 ) on Friday June 27, 2014 @02:13PM (#47334731)

            OK, smartass, what is the evolutionary advantage for stupidity?

            I suggest you ask evolutionary biologists. Specifically, go ask that group of evolutionary biologists standing over there lamenting their inability to connect with females, who somehow prefer muscularly ripped albeit less cranially endowed surfer dudes.

            Am I kidding? I'm not sure.

            • by radtea ( 464814 ) on Friday June 27, 2014 @03:20PM (#47335357)

              I suggest you ask evolutionary biologists.

              And evolutionary biologists will ask, "What is the evolutionary advantage of intelligence?"

              What we think of as "intelligence"--the specifically human abilities to build complex machines and to use anything to represent anything else and to create unbounded chains of logical inference--is almost certainly an epiphenomenon of having a brain big enough to engage in the kind of complex social and cultural behaviour that developed due to sexual selection in our evolutionary history.

              The human brain is like the peacock's tail: men with big brains were more likely to get laid, probably because we could be more entertaining and interesting to women with big brains. Once the process started it ran away with itself, until both men and women ended up with these enormous brains that happen to be able to think deep thoughts.

              Evolution does this kind of thing, like flight-feathers evolving from modified scales that were selected for thermal rather than aerodynamic properties.

              • by Immerman ( 2627577 ) on Saturday June 28, 2014 @12:49AM (#47338869)

                > probably because we could be more entertaining and interesting to women with big brains

                Hell, if you actually focus on it big brains can help you attract pretty much anyone that doesn't actively prefer stupidity - and that's pretty rare. Give up math, science, comics, etc, etc, etc and instead focus all that brainpower on actually observing and analyzing the behavior of people around you, and experimentally modifying your own behavior to test your hypotheses on social interaction in a rigorous manner - and I can virtually guarantee you that your social appeal will improve. That so many intelligent people end up somewhat isolated is, I think, a testament to the wonder of the other fields an intellect puts within reach, as well as the appeal of immediate intellectual gratification: Every little step you take when climbing towards the shoulders of giants reveals new expanses of understanding, whereas social skills are a far less readily transferable kind of knowledge, thrusting you into experimental science from step one.

                I suppose a big brain is like a peacocks tail in more ways than one - carelessly harnessed (from an evolutionary perspective) it can become a serious disadvantage to your genetic survival (reproductive fitness in this case)

          • by tepples ( 727027 ) <tepples.gmail@com> on Friday June 27, 2014 @02:32PM (#47334889) Homepage Journal

            OK, smartass, what is the evolutionary advantage for stupidity?

            A complex brain uses energy that could be used elsewhere to propagate the species. Humans are extreme K-strategists [wikipedia.org], which make few babies but put more effort into raising them. So-called "lower" animals may be r-strategists, which make lots of babies in hopes that some survive.

          • by WrongMonkey ( 1027334 ) on Friday June 27, 2014 @02:45PM (#47335029)
            You have the question backwards: what is the evolutionary advantage for intelligence? The smartest people certainly don't have the most kids.
          • by morgauxo ( 974071 ) on Friday June 27, 2014 @02:55PM (#47335123)

            In developed countries stupid people tend to have more children.
            Running a brain takes a lot of calories. In places where people have to worry about starvation I wonder if IQ might even be a liability.
            We are really lucky that humanity ever even achieved inteligence. It will be extremely lucky if we actually manage to keep it.

            • by Anonymous Coward on Friday June 27, 2014 @03:18PM (#47335337)

              us being abled to cheaply fix the genome should be the death to eugenics. The only question still remaining is whether we want to allow pepole to cripple their children's genome so that for example a deaf parent also gets a deaf child. There are people wanting this.

              • by Immerman ( 2627577 ) on Saturday June 28, 2014 @12:58AM (#47338897)

                A cheap and easy way to modify genes directly would the death of eugenics? Really? Unless you're restricting your definition to animal husbandry applied to humans I'd have to disagree:

                eugenics
                noun
                the study of or belief in the possibility of improving the qualities of the human species or a human population, especially by such means as discouraging reproduction by persons having genetic defects or presumed to have inheritable undesirable traits (negative eugenics) or encouraging reproduction by persons presumed to have inheritable desirable traits (positive eugenics)

                notice that it says especially by, not exclusively by. And I dare say that that's because until recently that was the only viable way to do such a thing.

            • by radtea ( 464814 ) on Friday June 27, 2014 @03:31PM (#47335461)

              In places where people have to worry about starvation I wonder if IQ might even be a liability.

              Brain size and IQ are not particularly correlated, and I've seen at least some research suggesting that people with high IQs or more education are actually more efficient at using their brains, to the extent that there is some thinning of the grey matter in such individuals in their late teens or early 20's.

              Thinking does take more energy than not, but this isn't a big effect compared to brain size: http://www.scientificamerican.... [scientificamerican.com]

          • by ShanghaiBill ( 739463 ) on Friday June 27, 2014 @03:18PM (#47335327)

            OK, smartass, what is the evolutionary advantage for stupidity?

            Smart people innovate. Dumb people follow routines because "we have always done it that way." So in a desert dwelling hunter-gather tribe enduring a drought, the smart guy innovates by digging for water and building a still. It comes up dry, and he dies of thirst. The dumb people follow the trail through the desert that their grandmother showed them decades ago, and find a waterhole.

            In an urbanized society, innovation has limited risk, and generous rewards. In a primitive society, innovation has big risks and limited reward. So people that have a long history of urbanization, such as the Chinese and Ashkenazi Jews [slashdot.org], tend to have high IQs, while the desert dwelling San Bushmen [slashdot.org] have the lowest measured. In both cases, they have adapted to the environmental conditions.

          • by dj245 ( 732906 ) on Friday June 27, 2014 @03:33PM (#47335483) Homepage

            If it wasn't, that mutation would have been selected out of existence a long time ago.

            OK, smartass, what is the evolutionary advantage for stupidity?

            Because you'd think we'd have selected that away a long time ago as well.

            Hell, we have an appendix. Why do we have an appendix? Why hasn't evolution made that go away?

            Evolution is awesome, but it can do some silly things that stick around.

            Most people think of evolution as "survival of the fittest", but this is a gross simplification. In a population bottleneck [wikipedia.org], genetic diversity can shrink rapidly if a large portion of the population dies out. Imagine what would happen if everyone in the world died except a small and distinct group- lets say the Vietnamese people just for example. If the population recovered and repopulated the world, humans would have lost a tremendous amount of genetic diversity which may or may not be beneficial to survival.

            Evolution in the traditional sense also only affects characteristics which affect the ability to reproduce. For example, it is impossible for humans to evolve the problem of cataracts or alzheimer's out of our genetic code. By the time these problems show up, the children of those affected have already become self-sufficient. Evolution is about "reproduction of the fittest" not the more general "survival of the fittest".

            • by Immerman ( 2627577 ) on Saturday June 28, 2014 @01:10AM (#47338941)

              Alternately, survival of the fittest is about *genes*, not individuals - and given mortal organisms, their reproduction usually correlates very closely to the survival of their genes.

              Interestingly, in many hive organisms this correlation fails in favor of the super-organism. Among honeybees for example the workers (female) are fertile, but share far more genes with their siblings than they would with their own children, and so survival of the fittest (genes) leads them to promote the health and reproduction of the queen rather than themselves - it's a better investment for their genes.

          • by Anonymous Coward on Friday June 27, 2014 @04:59PM (#47336239)

            > OK, smartass, what is the evolutionary advantage for stupidity?

            That you believe there is a single quantifiable trait that equals stupidity the same way a single genetic difference stops the processing of a single specific chemical reaction says you don't have a clue what you are talking about. That you are actually stupid even.

        • by Rigel47 ( 2991727 ) on Friday June 27, 2014 @02:08PM (#47334691)
          The error in your statement is that you believe evolution only accumulates the good and never the bad. Why is there a whole raft of genetic diseases in the human population now? Shouldn't they have been "selected out" a long time ago?
          • by radtea ( 464814 ) on Friday June 27, 2014 @03:05PM (#47335207)

            Why is there a whole raft of genetic diseases in the human population now? Shouldn't they have been "selected out" a long time ago?

            Many genetic diseases are the result of optimizations for other things (anemia is related to malaria resistance, there is some problematic gene in a Jewish sub-population that is related to plague resistance, etc.)

            Evolution is continuously running an extremely complex multi-dimensional optimization problem with a time-varying objective function. Local minima abound, and it's easy for organisms to get trapped in them.

            Furthermore, kin selection and possibly group selection play a role in human evolution, which makes the whole thing even more complex and non-linear. So looking at specific genes and saying, "That doesn't make sense!" as if there was some obligation for the universe to "make sense" to our naive pre-scientific intuition is fairly silly.

            The human genome is a Rube Goldberg apparatus that manages to make hundreds of thousands of products out of 40,000 strongly interacting templates plus a bunch of ridiculously inefficient secondary control mechanisms like micro-RNAs (which in some typically degrade already-transcribed mRNA). Pointing to one step as if it can be considered in isolation from everything else is not a good move.

            Loss of vitamin C manufacture could well have to do with the development of some other pathway that was more important at the time, and may well continue to be more important today. The only way to really find out is to either a) understand the genetic trade-offs in detail or b) ask some volunteer to have their vitamin C production turned back on by a technique like this. Personally, I'd recommend the former.

            Given how weird humans are developmentally, some things like this may be important when we're young and not so much when we're older, so in the fullness of time we may find we can turn on vitamin C production only after people mature, for example. The possible range of futures, given how little we know now, is large.

            In the meantime, we have plenty of people with genetic diseases that we know the cure will not significantly disrupt their cellular machinery, because we have lots of examples of people without those diseases who are just fine.

            • by Rigel47 ( 2991727 ) on Friday June 27, 2014 @05:21PM (#47336517)
              You have a good command of biology. My fundamental objection is with this statement:

              Loss of vitamin C manufacture could well have to do with the development of some other pathway that was more important at the time, and may well continue to be more important today.

              What if there was a co-occurring mutation that was, at that time, more advantageous than the loss of vitamin C production? What if the loss of vitamin C production happened to a population living in an area with abundant ascorbic acid in their food? Maybe they killed off their less-lucky neighbours? The notion that something is because it's got to be optimal is false. There's a lot of flexibility to life even within a species.

              It may be true that we've some advantage by not producing this vital anti-oxidant. I just rather doubt it.

        • by dixonpete ( 1267776 ) on Friday June 27, 2014 @02:18PM (#47334763)
          I always figured we lost that gene because our ancestors lived in trees and largely fed on fruit making the gene useless, in that context anyway.
        • by king neckbeard ( 1801738 ) on Friday June 27, 2014 @02:22PM (#47334803)
          Unless there was no reason to select against it, like if foods containing vitamin C had been available to an extent that there was rarely a shortage of it when there was an excess of glucose.
        • by Anonymous Coward on Friday June 27, 2014 @02:29PM (#47334863)

          It's more efficient to get Vitamin C from food. If it wasn't, that mutation would have been selected out of existence a long time ago.

          That argument doesn't even apply because the liver goes through all but the last step. It might apply if all the prerequisite steps didn't happen, but really cutting out that last step just means more junk floating around that cost energy to make with no useful result whatsoever (completely disregarding the fact that in most climates it isn't more efficient to get vitamin c from food).

        • by ShanghaiBill ( 739463 ) on Friday June 27, 2014 @02:53PM (#47335103)

          It's more efficient to get Vitamin C from food. If it wasn't, that mutation would have been selected out of existence a long time ago.

          Not necessarily. It probably WAS more efficient, when our primate ancestors lived in the rainforest, and munched on fruit all day. Most primates can't synthesize vitamin c either. But once the ability to synthesize vitamin C was lost, it was no longer part of our gene pool. So when we left the tropics, and switched to a diet based more on grain and meat, it was too late to adapt.

      • by spacemky ( 236551 ) * <nick.aryfi@com> on Friday June 27, 2014 @01:59PM (#47334613) Homepage Journal

        ...people start crapping out citrus fruits?

      • by king neckbeard ( 1801738 ) on Friday June 27, 2014 @02:19PM (#47334773)
        It's amazing how much functionality the liver has. At times, it feels more accurate to claim that a human is a parasite attached to a liver.
      • by morgauxo ( 974071 ) on Friday June 27, 2014 @02:52PM (#47335085)

        Well, maybe in some third-world places people get healthier. That is, if they ever even get access to this modification. Everybody else just pees more. Given that so many are already drinking pop, coffee and beer when their bodies need water we might actually see health go down in the first world due to dehydration.

    • by Anonymous Coward on Friday June 27, 2014 @08:06PM (#47337783)

      How long? About never.

    • by Doubting Sapien ( 2448658 ) on Friday June 27, 2014 @09:18PM (#47338159)
      It is hard to predict the progress of technology, so - NO: I won't tell you "how long before...." But I'll try to explain why CRISPR is special enough to be exciting in my experience and what technological/engineering hurdles need to be overcome in order to reach your objective.
      At the moment, variations of the CRISPR-CAS system can only edit the genome of individual cells in vitro with varying efficiency. This is assuming you can culture the cells to begin with. For example, I work with human embryonic stem cells, which are particularly finicky. They won't tolerate much roughness and will even up and die on you if the growth conditions are just a bit off. This is very hard to achieve reliably as some culturing reagents (coating matrix, for example) are "undefined" products with variations in composition from batch to batch.
      To go to a chop shop and treat your issue at the genetic level requires an in vivo way to introduce a CRISPR-enabled vector into your cells. This is not easy to do with today's technology, but it may not necessarily be a deal breaker. In the example you gave, a food allergy can probably be addressed by treating only the GI tract and the immune system that comes into contact with the offending allergen. As such, there is no need to target every living cell in your body in this case. However, if you are treating an illness involving a more fundamental life process, that is not the case. For example, a mitochondrial disease where basic cellular metabolism is defective would probably be best tackled when an individual is still a developing embryo or at least very, very young. Otherwise, tissues and organs that are not convenient to access will still retain the genetic defect and present problems for the host organism.
      Another question is where in the genome you want to edit. So far, one of our experiments involving the targeted insertion (non-CRISPR method) of a construct into our hESCs have been a bust. Our best guess is that the intended site of transfection (sub-telemeric regions of chromosomes) is critical for cell survival and too much fiddling in the area is fatal. CRISPR-CAS was a compelling solution for us because of how ideally targeted it is supposed to be. We are not aware of anyone else who've used CRISPR with hESCs in the way that we are doing, but what has been reported so far with other experiments using notoriously difficult subjects has been encouraging. So far, the experiment shows clear evidence of true integration into the genome as opposed to a transient transfection. In about a week, a Southern blot verification will tell us if the integration was random or indeed targeted.
      As rosy as I can paint a picture about what is possible, however, strong caution follows the introduction of any new technology. Anonymous Coward may be an asshole, but (s)he isn't wrong for being a cynic about the commercial deployment of this as a consumer product. Considering how complex human biology is, the chance of an unintended edit with unanticipated consequences is more than likely. Many genes are linked in very convoluted ways. Even with the human genome project having ostensibly mapped everything, we are still looking at just the tip of the iceberg. Having a complete manuscript, is very different from understanding all the nuances of the story. To get back to the spirit of your question, I would imagine that the scenario probably is more similar to dental service, where you go back periodically to check on the integrity of any major service, with tweaks along the way as necessary.
  • by Nutria ( 679911 ) on Friday June 27, 2014 @01:34PM (#47334417)

    a crazy Arab, will try to weaponize it.

    Hilarity will not ensue...

  • by Anonymous Coward on Friday June 27, 2014 @01:42PM (#47334481)

    It could be worth it. This could be huge. Besides sickle cell, there's cystic fibrosis and a bunch of others. Not to mention high vulnerability to cancer caused by faulty genes. Angelina Jolie could still have her breasts. I could stop taking medication for gout.

  • by L'Ange Oliver ( 1521251 ) on Friday June 27, 2014 @01:46PM (#47334513) Homepage
    This technology has been advertised everywhere in my institute... and now its on Slashdot... I wonder if there is someone behind their marketing campaign?
  • by Joe Gillian ( 3683399 ) on Friday June 27, 2014 @01:54PM (#47334569)

    From the Wikipedia article, it seems like CRISPR works by injecting a strand of "neutral" genetic material into a genome and cause genes to not be transcribed - so you can "turn off" an improperly expressed gene, but can't actually replace it with a normal one. The NPR article, however, has people mentioning the idea of replacing improperly expressed genes with normal ones.

    From what I understand, the difference between the two is that if Wikipedia is correct, CRISPR would only be useful in humans (once they get it to be accurate) to cure diseases that arise from a gene being expressed when it shouldn't be, for things like sickle cell or Huntington's. However, if NPR is correct, CRISPR can also cure diseases that arise from a gene not being expressed when it should, such as hemophilia.

    Which one of these is correct? What is CRISPR actually good for?

    • by paskie ( 539112 ) <pasky.ucw@cz> on Friday June 27, 2014 @02:24PM (#47334813) Homepage

      CRISPR is a tool that allows you to cut the DNA in two disjoint pieces at a specific point (specification of this point is a parameter of a particular CRISPR instance). What happens then depends on your setup; bacteria will just insert some junk at that break point, or you can pack your custom DNA sequences along the CRISPRs and they will be spliced in, connecting to each of the two disjoint pieces by one end. Thanks to this, at that specific point, you can disable a gene or modify or add an extra sequence.

      We had tools to do this before - restriction enzymes or TALENs. They weren't really usable for therapeutic purposes, though, due to much less reliable targetting, more laborous engineering (parametrizing your instance for a specific sequence) and low effectivity (the break happens only in a a few percents of cases). CRISPRs are easily parametrized, can be precisely taretted, and have effectivity in tens of percents (in general; can vary organism by organism). It's still a work in progress, but looks pretty promising!

      • by interkin3tic ( 1469267 ) on Friday June 27, 2014 @05:22PM (#47336527)
        To add onto that (since I was reading up on this), the cell itself splices in the new DNA sequence very rapidly and efficiently, since it's a mechanism the cell uses to avoid cancer.

        In the double helix, when one strand of the DNA is broken, the string of DNA is held together by the other strand, it's an easy fix. Both strands broken at the same place means the DNA has come completely apart, has sustained some serious damage. The cell detects that pretty rapidly. The cleaner way is to find the sequence on the other chromosome and use that to repair the broken chromosome. A simpler but more dangerous way is to just grab two broken ends of DNA and stick them together. It's possible the cell will grab the wrong broken strand of DNA though and the result will be cancer.

        Either way can be used to insert DNA where you want it.
    • by Chris Rackauckas ( 3716821 ) on Friday June 27, 2014 @05:48PM (#47336779)
      CRISPER works via the connection between the CRISPER and the enzyme Cas9 (that's why it's actually called CRISPER-Cas). I will simply talk about the synthetic bio version, not the naturally occurring. So let's say you want to do a targeted genome edit at a specific point in the genome. To do this, you need to make your CRISPER RNA as follows: you have the "Cas section" (crRNA) and "localization" section (tracrRNA). The way it works is that you design the localization section to be complimentary to the DNA you wish to cut (people have worked out how to make RNA strands bind to the DNA in specific points). You then inject some of the Cas9 enzyme which binds to the "Cas section" (or in bio terms you tend to say the CRISPER promotes the enzyme) and holds it steady. This enzyme, when held near the DNA, will cause it to cut. So now you have a tool for cutting. Let's say you want to cut out a gene. You can just make two CRISPERs, one before the gene and one after. Inject the CRISPERs, inject the Cas9, and that gene is gone. But I forgot to mention the important part: you have to fix the break! You can then do genome editing by pairing this with something called Homology Directed Repair to put any sequence you want in there. There are two questions to address: why is this better than what we had before and how good is it? Before the main tools were restriction enzymes and TALENs. Restriction enzymes had a specific sequence they could cut (for example: BamHI cuts GGATCC). You can use this with some ligation (gluing back together) tools to attempt to do this stuff, but it's quite hard if you cannot specifically target an area. TALENs I haven't really used (I'm too new, my lab just started by telling me about CRISPERs) but from what I know people say they weren't reliable and were hard to make. However, CRISPERs are just an RNA sequence. There are online tools that help you design the CRISPERs as long as you know the DNA you want to target (which you hopefully do thanks to the magic of genome sequences). Making this RNA is a simple technique and once you get it, you basically have an infinite amount of the CRISPERs in case you mess up. You just take that, inject it with the Cas9 enzyme that you buy, and it works surprisingly well. Thus it has taken over in genome editing for many species. I know that in zebrafish it has become the tool to use, and from my reading it sounds like it's being used a lot in mouse, plants, and flies as well. It's just easy and reliable. Will this mean that you will use this to change your baby's eye color? I don't know, but from what I hear about its effectiveness in the zebrafish lab, although you get it working a good amount of the time, it's still not all of the time. People are okay if it doesn't correctly re-glue the DNA in 10% of your fish embryos, but would you be okay with it failing 10% on your baby? I'm not sure.
  • by gurps_npc ( 621217 ) on Friday June 27, 2014 @02:12PM (#47334723) Homepage
    "CRISPR is one of those interesting inventions that comes, not from scientists explicitly trying to cure a disease, but from researchers trying to understand something fundamental about nature."

    There is no such thing as a researcher trying to understand something fundamental about biology that is not trying to cure disease.

    As for not trying to cure a specific disease, no real innovations come from trying to cure a specific disease. All the really cool stuff comes from doctors trying to figure out how something works, in the hope that some day their knowledge will cure a disease.

    • by Alopex ( 1973486 ) on Friday June 27, 2014 @02:57PM (#47335145)

      This is patently false. There is a whole swath of biological research under the banner of "basic science" which, while it may purport to address a far-off disease application (for the sake of grant $$$), is only aimed at understanding how life functions at the most fundamental levels. Thousands upon thousands of researchers in this country are funded by the NSF and NIH (among others) precisely to figure out things we know that we don't understand.

      For an anecdote, I did this kind of research for a few years. My lab was trying to understand what the function of a motor protein was because we could see it, we could see processes it was involved in, but had no idea how or why it was behaving the way it did. There was no disease focus. Part of research is cataloging the natural world so that, maybe, we will one day use that knowledge for our benefit (not necessarily for disease).

      Disease is one of many applications of basic research. The amount that goes into producing chemicals through engineering bacteria and producing food through engineering plants is staggering. These applications are currently enabled by CRISPRs. I'll be interested to see how eugenics develops in the next few decades.

      • by radtea ( 464814 ) on Friday June 27, 2014 @03:39PM (#47335537)

        There is a whole swath of biological research under the banner of "basic science"...

        Absolutely. I've worked both with pure biologists and physicians (and biologists in a medical context) and they have dramatically different outlooks and mindsets. Many, many biologists are deeply interested in understanding what is going on, while physicians and medical-focused biologists are much more interested in finding stuff that works to solve this problem.

        The divide is very similar to that between pure and applied physicists, although for some reason we don't talk about "applied biologists" (perhaps we should.) Pure physicists are simply trying to find answers to questions; applied physicists are trying to find solutions to problems. The same is true in biology.

    • by Anonymous Coward on Friday June 27, 2014 @05:08PM (#47336361)

      "There is no such thing as a researcher trying to understand something fundamental about biology that is not trying to cure disease."

      So wrong on so many levels.

      Yours
      Practicing scientist.

  • by Anonymous Coward on Friday June 27, 2014 @11:32PM (#47338657)

    So, this could potentially be the cure for viral diseases as well as genetic?

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