The Next Gene Engineering Revolution: Curing Genetic Disease and "Designer Babies" Using Crispr

CrisprCRISPR Genome Engineering ResourcesToday The Independent has a fascinating article on a new extremely precise gene editing technique called Crispr. The enzyme CAS9 derived from bacteria enables researchers to edit genomes at will. The technique clearly has major implications for treating genetic diseases in people. Current techniques can insert new genes but often they land fairly randomly and disrupt other genes. The new technique appears to be so accurate and so safe that it could be used to correct genetic flaws in human embryos. As The Independent explains:

A breakthrough in genetics – described as “jaw-dropping” by one Nobel scientist – has created intense excitement among DNA experts around the world who believe the discovery will transform their ability to edit the genomes of all living organisms, including humans.

The development has been hailed as a milestone in medical science because it promises to revolutionise the study and treatment of a range of diseases, from cancer and incurable viruses to inherited genetic disorders such as sickle-cell anaemia and Down syndrome.

For the first time, scientists are able to engineer any part of the human genome with extreme precision using a revolutionary new technique called Crispr, which has been likened to editing the individual letters on any chosen page of an encyclopedia without creating spelling mistakes. The landmark development means it is now possible to make the most accurate and detailed alterations to any specific position on the DNA of the 23 pairs of human chromosomes without introducing unintended mutations or flaws, scientists said.

The technique is so accurate that scientists believe it will soon be used in gene-therapy trials on humans to treat incurable viruses such as HIV or currently untreatable genetic disorders such as Huntington’s disease. It might also be used controversially to correct gene defects in human IVF embryos, scientists said....

In addition to engineering the genes of plants and animals, which could accelerate the development of GM crops and livestock, the Crispr technique dramatically “lowers the threshold” for carrying out “germline” gene therapy on human IVF embryos, added Professor [Craig] Mello [of the University of Massachusetts Medical School and 2006 Nobelist for his discovery of RNA interference]....

Germline gene therapy on sperm, eggs or embryos to eliminate inherited diseases alters the DNA of all subsequent generations, but fears over its safety, and the prospect of so-called “designer babies”, has led to it being made illegal in Britain and many other countries.

The new gene-editing technique could address many of the safety concerns because it is so accurate. Some scientists now believe it is only a matter of time before IVF doctors suggest that it could be used to eliminate genetic diseases from affected families by changing an embryo’s DNA before implanting it into the womb.

“If this new technique succeeds in allowing perfectly targeted correction of abnormal genes, eliminating safety concerns, then the exciting prospect is that treatments could be developed and applied to the germline, ridding families and all their descendants of devastating inherited disorders,” said Dagan Wells, an IVF scientist at Oxford University.

“It would be difficult to argue against using it if it can be shown to be as safe, reliable and effective as it appears to be. Who would condemn a child to terrible suffering and perhaps an early death when a therapy exists, capable of repairing the problem?” Dr Wells said.

Here's hoping that the Crispr technique lives up to the hype. Now if we can only keep the bioethicists ("science is outrunning our regulations") from interfering.

The whole article is worth reading.

H/T Marian Tupy.

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  • Hugh Akston||

    I was on Crispr for a while, but like most platforms it was soon overrun by pathetic weeaboos and Sonic pr0n.

  • DJF||

    How soon before New Jersey passes a law against “fixing” homosexuality in embryos?

  • Orion||

    While I find homosexuality in men thoroughly disgusting, it's well documented that it is present in nature; in animals. It doesn't appear to be an aberration. Judging whether it is or not is irrelevant.

    The greatest threat of genetic engineering is on the institution of religion. Religious groups are strongly pro-life, for example. I'm a Libertarian. People should have the freedom to choose what they want to do with their body, even if it's "the wrong thing".

  • LynchPin1477||

    Bring on the Eugenics Wars!

  • db||

  • LynchPin1477||

    The real question is, can this finally be used to deliver a penis enlargement that actually works? And will anyone on the internet actually believe it?

  • Orion||

    They'll believe it by word of mouth, not because of internet advertisement.

    But I wouldn't consider it particularly important. It would be better to correct an abnormally small size in embryos. Or to improve sexual compatibility in a relationship between adults.

    There are all kinds of people though, so maybe with the advent of genetic engineering it could also make genetic comparisons within a population, and list the number of ideal mates. Instead of having to go on hundreds of blind dates, you could make a more educated guess. Instead of having to change yourself, you just find the right people that can appreciate you for who you are.

  • Hyperion||

    Can they edit out the control freak gene in politicians so that the rest of us can get on with our lives without their destructive meddling?

  • PD Scott||

    Who would condemn a child to terrible suffering and perhaps an early death when a therapy exists, capable of repairing the problem?” Dr Wells said.

    Hand-wringing bioethicists on line 1, anti-GMO activists on line 2…

  • Hyperion||

    Good news is that the luddites lose in the end.

  • Free Society||

    Superstitious conservatives, progressives, socialists, bioethicists and bureaucrats.

  • Drake||

    Sounds like the same technology could be used to eliminate dna replication errors - which would reverse many effects of aging.

  • Hyperion||

    Yes, and can you imagine the nanny staters freak out on that one? If you think printing plastic guns make them freak out, wait until they find out about that. Popluation bomb! They're using all of OUR resources!

  • LynchPin1477||

    But no way can we touch Social Security. Hands off!

  • DJK||

    I'm not sure. From what I understand about the technology, it seems to work by unzipping DNA, cutting out a single nucleotide, and inserting a new one. It sort of requires that the base pair that you're altering be there in the first place. Much of aging occurs due to the irreversible loss of the ends of DNA (telomeres) during DNA replication. You're actually losing that DNA. I'm not sure that this method would work for getting it back. The DNA replication errors you're referring to are more often associated with the development of cancers and such. Clearly, treating that would be a breakthrough. Not quite ending aging though.

  • mr simple||

    What a fucking time to be alive.

  • Michael S. Langston||

    ^THIS^

    Though I may not make it 100 more years to when we reach immortality :)

  • Fluffy||

    I want to be able to edit my offspring's genetics so they don't need to sleep.

    Then I will fill their heads with pseudo-Objectivism and unleash them upon you to destroy your weak, decadent society.

  • Brett L||

    Wait, what? Can you set that to activate after they are old enough to work the night shift?

  • Drake||

    Would that make me a beggar or chooser?

  • ||

    Reading Beggars and Choosers?

  • Mr Whipple||

    Meh. In 20 years we'll all have nanobots in our bodies that can be activated and controlled by your iPhone.

    But it'll cost you $0.99 per illness for the download. Of course, Android downloads will be free, but the free version will only cure half of the illness.

  • DJF||

    “””and controlled by your iPhone.””’

    And the NSA will control your IPhone.

  • Hyperion||

    But it'll cost you $0.99 per illness for the download

    Plus the $30,000 federal tax. And don't forget to get in line for the download and wait 20 years before the government panel approves your download. IOW, die serf! Because FYTW!

  • Floridian||

    I don't get the designer baby concern. I mean it is not like I can't alter the type of child I want to some degree now. I can choose to reproduce with a women is who is taller, shorter, darker skinned, lighter skinned, etc. to influence what my child would look like if I so desired.

  • Brett L||

    I would hope that we keep the "wild" strain alive. Which is no problem because most first time parents wanted to fuck more than they wanted to make a baby.

    So yes, there will be some people who try to tweak their genomic inheritance to maximize certain factors. But its too simplistic for many of the "success" factors like intelligence and personality and even actual height. Everything is so environmentally pressured. I would like to make sure that the genetic heart condition that affects an uncle and cousin don't pass on, if possible.

  • John||

    It will be expensive. So there will be billions of people who don't do it. Also, once you are pregnant, I am not sure you could alter the baby without a lot of expense. That would be worth it if the kid has a disability but not if he or she has the wrong eye color.

    The danger of it is that people design their kids based on fashion and in the macro you end up losing genetic diversity or producing all kinds of strange and unwanted effects. But as long as there are still millions of people out there having kids the normal way, I don't see how that happens.

    What this will do is make our upper classes even more inbreed and bizarre than they already are. Think about what celebrity and Ivy league twits have done with baby names and then imagine giving them access to designer babies.

    It will not be pretty. But the idiot sons and daughters of the elite have always been idiots and twits. They will just be weirder now.

  • Hyperion||

    Can you imagine a world in which every woman looks exactly like Britney Spears?

    Fortunately, John, a lot of people still have imagination.

    I think it will turn into more of an art form, where people might want to enhance certain of their features to be more unique. At least I hope so. An entire planet of Britney Spears is not where I want to live.

  • John||

    Fashion would change like it does with names. You would have a generation of Brittney Spears like you had a generation of Jennifers and then a generation of Emilys and Emmas.

    But I can't see this ever being cheap enough and safe enough where many people would do it for anything other than to avoid a disability.

    Insurance isn't going to cover it to make your kid look like Brittany Spears. So you will have to pay for it yourself. And how many people are really going to do that? Unless it is really cheap, not many and not everyone even then.

  • Hyperion||

    The only thing that will slow it down from becoming relatively cheap is the government and their cronies. But eventually, it will be available to everyone.

    The luddites already lost, that was a long time ago. They are still with us, but they will never be powerful enough to stop technological process, only slow it down to some degree.

  • John||

    I am not sure about that. Full IVF costs over $20,000 per attempt today. And that is in a pay per service market. Insurance won't cover IVF. So people save and finance and buy it themselves. And it still costs 20K. This stuff is just expensive. Labs and the skills of doctors are just not cheap and they are not going to be cheap.

    I bet it costs about the same as IVF. And in that case most people won't do it unless it is to avoid a disability. Brave New World is not quite here yet.

  • Hyperion||

    Brave New World is not quite here yet

    Oh man, here we go.

    How many people owned a car in 1910?

    How many people had ever flown in an airplane in 1920?

    How many people owned a cell phone in 1975?

    How many people had a TV in their house in 1940?

    How many people had a computer in their home in 1970?

    How many people could have envisioned the internet in 1960?

    20 years from now there will be technology available that will be almost unimaginable now, for people who are now saying things like 'Man will never fly!'. I'm not saying that Kurzweil is right about the singularity, but he's a lot closer than what many think.

  • John||

    Dude,

    IVF has been around for 30 years. Some things are just hard and will always be expensive. Technology gets cheaper but skilled labor doesn't. Not everything is a computer.

  • Hyperion||

    30 years? And now it can never change?

    The horse was the main means of faster transportation for thousands of years. That doesn't mean anything.

  • John||

    I am not saying it will never change. I am saying it will not get to the point that anyone can do it any time soon.

    Again, Moore's law is great. But it is not universal to all progress.

  • Cytotoxic||

    You're wrong John. Crispr is so simple that even a novice biochemist could do it. Even I am competent to possibly do it. Right now it can be used on embryos but it totally can be used on adults in theory. And it will be easy and cheap.

  • Mendelism||

    I think the same could be said for IVF, perhaps even more strongly. I suspect the relative costs for these procedures will depend more on how the IP is structured and obviously the consumers' willingness to pay. My understanding is that most of the cost of IVF is due to the cost of fertility drugs (which are more easily protected by patent than procedures per se), and frequent clinic visits (which would probably be no different for Crispr-related genomic editing in embryos).

    Crispr being owned by Harvard, I'm not sure how it will play out, but often universities will grant multiple licenses to different companies which should result in lower costs to the consumer relative to an exclusive license or patent.

    Overall I tend to agree with John, this won't actually be a mainstream issue for a while.

  • Orion||

    "All that I am, is what I am."

    If the world was filled with women that looked exactly like Britney Spears, then it would be natural. And be no more an issue than it is for sextuplet girls to look alike. *You* wouldn't want to live in that world and therefore you become extinct. You would not reproduce.

    But what we're talking about now is really the conflict between thousands of years of evolution in the form of instinct and genetic preferences, with weeks of artificial evolution.

    I think at first genetic engineering would be a "fad", but eventually people would choose to make changes based on who they are. Of course, who they are then, would be determined by genetic changes made previously.

    In actuality, being more alike genetically would be better. The greater the genetic differences, the greater the risk of rejecting those traits. Even in a world of women that look identical, the question is still the same.. are they *attractive*? Variety is highly overrated. Love only requires one ideal person, not a million different people.

  • Michael S. Langston||

    I am not sure you could alter the baby without a lot of expense.

    Not in the very near term - but so long as governments don't kill the progress, there's no reason to think DNA alterations won't take the same tack as all other new technologies - which is get less expensive and have more widespread use over time.

    I would think given the trends in human history - it should be easy to see a day when almost all pregnancies get "DNA alterations" as a normal matter of their prenatal care - sure, it might not be Gattaca and be about height and such, but to know instantly you're significantly dropping chances of things like cancer - it's a no-brainer.

    Disclaimer: While the trends show the likelihood of lower costs over time combined with stabilization of use, when it happens and exactly how is anyone's guess - and no matter what we guess, we're likely to be off by 1000 fold (assuming altering DNA can do the things we think).

    As an example: contemplate the big predictive blunders in history - like Bill Gates "No one will need a computer in their homes."

  • Hyperion||

    Here's hoping that the Crispr technique lives up to the hype. Now if we can only keep the bioethicists ("science is outrunning our regulations") from interfering.

    You won't be able to keep the government nannies from interfering. Because science is irrelevant, big government is our savior!

    Anyone remember Leon Kass? Because there are a thousand more of him in government just waiting for their chance to come out of the closet railing against people living longer than what they have decided we can. They only have so much money and resources to spend on us peasants, you know.

  • LynchPin1477||

    I don't remember Leon Kass. History lesson?

  • Hyperion||

    He's the statist asshole who is always railing against any type of technology which will allow people to live longer. He's the ultimate malthusian control freak.

    Leon Kass, the talking horses ass

  • LynchPin1477||

    Just read the article, or the 30% of it Bailey didn't re-print. Pretty remarkable, awesome stuff. I understand why it makes people uneasy...this is uncharted territory. But the potential good something like this can do is just outstanding.

    Anyone on here knowledgable enough to say whether this can be used to treat diseases in adults, and not just in early stages of development. This is very far outside my area of expertise.

  • Hyperion||

    It's great. The singularity is near! Ha.

    Our government will regulate this technology out of existence in the USA.

    But no worry, you'll be able to go out of the country to get the treatments, until they put up the big invisible fence to keep us all in.

  • LynchPin1477||

    There is a chance of that, of course. But if you can cure cancer, AIDs, Parkinsons, Alzheimers, Downs, autism, MS, etc., it's going to be pretty hard for politicians to say "no" because they don't want parents choosing between green or blue eyes.

    Now if you can somehow increase intelligence, athleticism, etc., then I can see people being more willing to ban it, or at least very heavily restrict it. But how many of those things are actually rooted in DNA, and how much in development? Is there really any risk of creating a race of supermen?

    I think a more likely outcome is to regulate it so heavily and put up so many barriers to entry that it becomes unaffordable for most. And then to force health insurance companies to cover the procedure and subsidize the hell out of it.

  • Hyperion||

    it's going to be pretty hard for politicians to say "no" because they don't want parents choosing between green or blue eyes.

    It's going to be impossible to stop people from getting life saving treatments when they are available somewhere. Can you imagine having a child who is dying from some disease, and the cure is available, but some government panel of bureaucrats says no? This is what Obamacare will do, but people will start going elsewhere for treatments. Medical tourism is going to become a huge thing for some countries who will be willing to take advantage of this.

    It will be the same with radical life extension, which will eventually be available. People are going to want that and there is no stopping it, but the government will try because control is more important to them than anything.

    This is where the progressives are so wrong. Government is not progress, it is anti-progress.

  • DJK||

    I disagree. Opening up organ sales would allow hundreds of thousands of people to survive. There are enough people willing to sell their organs and enough surgeons okay with the concept in the abstract to allow this. Yet you'll never find a surgeon willing to perform such a procedure due to the regulatory environment. Government can create just as many financial and criminal penalties in the way as it needs and that will kill the development of the technology.

  • DJK||

    If, somehow, the technology advanced enough that you could do DIY genetic engineering, I might be convinced. However, I think that it will require big money research for quite awhile. And that is precisely the type that governments are capable of crushing.

  • John||

    How many people who are really poor would sell a kidney and use the money to get out of poverty? You would save thousands of people and give thousands of others a leg up in life. It is a shame we can't do it.

  • DJK||

    Agreed. This is my point. The health and social benefits are obvious. The health risks are absurdly low. Yet this procedure has been regulated out of existence. I wouldn't be surprised to see governments do the same with this technology.

  • Hyperion||

    How many people who are really poor would sell a kidney and use the money to get out of poverty?

    Before long, it will be a question of if a donor provided kidney will be cheaper than a printed one. Eventually, there will be no need for donors at all, because 3D printed organs will be readily available and cheap.

  • John||

    Hopefully hyperion. That would be even better.

  • Hyperion||

    The government is not going to stop these technologies. They are going to try, and fail.

  • DJK||

    The research on this is not at all clear. It's well-known that there are at least 20 point mutations that are correlated with intelligence. Taken together, though, they account for about 1% of all variation in IQ scores. There is a Nature Genetics paper from last year that claims to have found a set of genetic markets which amplify the effects of these variations. Even if you're convinced by the authors' claims, they still only account for about a 2 point increase in IQ. So, it's pretty evident that intelligence has at least some basis in genetics. So far, it seems to be pretty insubstantial. The research regarding that may or may not change.

  • John||

    It seems to me that if the effect were substantial or even statistically significant, people would have long since segregated themselves into intelligence castes.

  • Hyperion||

    A lot of it is environment. Some is genetics, though.

  • DJK||

    Perhaps. I think that a decent argument could be made that we have, to some extent, done so. Not "castes" as you say, but there is definitely at least some level of social stratification based on intelligence.

    If there is a genetic basis to very high intelligence, which is what I think LP is getting at, then it's going to be very hard to study. People of extremely high intelligence are, by necessity statistical anomalies. This makes finding statistical significance in this realm very hard to come by.

  • John||

    The other thing is that withing the normal range IQ really isn't that important to success. Yes, you are the one in a million 170 IQ, you have a big leg up on the world just like you have a real problem if you are the one in a million at the lower end. But if you are in the big middle, between say 100 and 120, your IQ is probably not going to do you as much good as you think. Your character and luck of where you were born is going to make the difference.

    The thing people never seem to understand is that the IQ is only valuable insofar as it produces extra value. How many jobs really require a 150 IQ? Not that many. Okay, I have a 140 IQ, that doesn't necessarily mean I will do my job any better than someone with a 130 or a 120. My extra IQ only matters if they somehow are required for the job.

    It sounds counter intuitive. You would think that you would always want "the smartest people for the job". Sure, but that doesn't mean it is worth paying extra for them.

  • Brett L||

    It sounds counter intuitive. You would think that you would always want "the smartest people for the job". Sure, but that doesn't mean it is worth paying extra for them.

    Except cops who want everybody to be between about 90 and 105, lest the smart ones start trying to change things.

  • Brett L||

    Well, the problem is that there seems to be a genetic maxima. The smarter you are, the less effect the genetics of breeding with another smart person are going to have. Regression to the mean makes breeding on the edges expensive from a time/resource/people standpoint. You need, say, 5000 smart people to breed 10000 kids to get 1000 genetically smarter people who have to have 3000 kids to get 300 genetically smart people and by the fourth generation its inbreeding or finding the best non-specifically bred stock.

    So even if we could cut and paste to raise the breeding results, it still takes several generations of people dedicated to doing this over the next century to break through the "wild" barrier.

  • Auric Demonocles||

    No man, we just need like 3 generations of this kind of program before we produce people capable of living for centuries and traveling through time to bang their mom.

  • Brett L||

    I don't love my mother like that. Does that make me a bad libertarian?

  • Brett L||

    Although I do qualify for Howard Family membership, so I am eagerly awaiting my initiation.

  • Auric Demonocles||

    Sorry, we're already past gen 1. So if you're not already in, there's no getting in.

  • Brett L||

    Sorry, we're already past gen 1. So if you're not already in, there's no getting in.

    Can you send someone else's hot, red-headed mom around as a consolation prize?

  • Auric Demonocles||

    You can't make any kids though. We don't want to waste time with any genetic weaklings who are gonna die after 100 years.

  • Hyperion||

    When everyone has neural implants to enhance their intelligence 1000x, genetics won't matter as much.

  • Orion||

    Creating a race of superhumans, or "alphas", is not a "risk", it's an ideal. It's what defines our drive to reproduce. To seek out who we perceive as an alpha, and pass on superior genetics.

  • Cytotoxic||

    Our government will regulate this technology out of existence in the USA.

    No it won't. Engineering this kind of stuff is actually not that hard. ssRNA is delicate but it can be stored and stabilized especially if it's made into dsRNA. Any idiot can express and purify to some degree the vast majority of recombinant proteins and I'm sure CAS9 is one of those.

  • OldMexican||

    The Next Gene Engineering Revolution: Curing Genetic Disease and "Designer Babies" Using Crispr


    And how would you like your baby, Mrs. Zislaff? Original or Extra Crispr?

  • OldMexican||

    It might also be used controversially to correct gene defects in human IVF embryos, scientists said...


    I'm guessing that at one point, the technology will be so advanced and reliable that it will be capable of being used uncontroversially. Right?

    Because, otherwise, I don't understand the reason for adding such qualifier, except maybe ridiculous Political Correctness.

  • The Late P Brooks||

    I just want to know when I can get an eye drop which will enable my damaged optic nerve to repair itself.

  • Hyperion||

    Optic nerve repairing nano-bots in each drop.

  • Hyperion||

    For anyone here who enjoys this type of topic, might want to check out this site:

    H+

  • Robert||

    Acid?

  • Cytotoxic||

    This technology is giving me a huge boner, straight up. Not only is this so versatile that it can modify just about ANY organism, it can probably be used on adults AND it's very easy. A novice can do it. We don't have to tolerate imperfections like CF or Huntington's. WE ARE GOD.

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