CRISPR gene-editing breakthrough opens door to treating broad array of diseases
npr.org
npr.org
this is one of the coolest paragraphs I've read in a while. if it's accurate, serves as a great reminder that we're in the future as we speak
What isn’t clear to me is that if this is targeted at the liver cells in particular, or splicing it for every cell in the body. In other words, this could be a germline mutation (passed to children) or it could be a somatic mutation (no change to future children), it depends on how targeted the treatment was.
See: https://blogs.sciencemag.org/pipeline/archives/2021/05/20/cr...
Delivery of Cas9, sgRNA, and associated complexes into cells can occur via viral and non-viral systems. Electroporation of DNA, RNA, or ribonucleocomplexes is a common technique, though it can result in harmful effects on the target cells.[43] Chemical transfection techniques utilizing lipids and peptides have also been used to introduce sgRNAs in complex with Cas9 into cells.[44][45] Types of cells that are more difficult to transfect (e.g. stem cells, neurons, and hematopoietic cells) require more efficient delivery systems, such as those based on lentivirus (LVs), adenovirus (AdV), and adeno-associated virus (AAV).[46][47][48]
Mind you, this is just the beginning. First we would have all kinds of diseases being cured using this, 1. It would change the whole medical diagnostics industry to correctly diagnose the pathogen, 2. Then destroy the pathogen using DNA cutting 3. It would cure cancers of many kind by enabling the T-cells to fight the cancer cells effectively
Then we would start with reprogramming cells to do what we want. For instance, we can cure baldness, completely regenerate cardiac cells after heart attack.
However, it would not stop at this. We would have bio-machines which can capture carbon dioxide, generate medicines, make clothes/cotton and food. We would have bio solar cells. We would bio light bulbs which ingest light during the day and glow at night. We would have artificial meat produced using this technology. We would have carbohydrates produced using this. Plastic and sewage eating bacteria would be a reality soon.
Next 30-40 years is going to be biotechnology as last 30-40 were computing.
But like all decisions, you weigh the good vs the bad and you make a decision. In many cases it will be the best choice by far. Additionally, the more we use it the more we will understand the down side and we can plan for it.
Yup, it has a down side but we'll be able to balance the good and bad parts. Techies love to focus on the good of technology but I think it's good to point out the bad as loud as possible so we can plan for it. The fact is that the genie is out of the bottle and it won't go back in. So CRISP is part of our future.
It seems like the scientists interviewed (some of them leaders in CRISPR) were of the opinion that we should stay the hell away from editing the human germ line (not sure if I used the right term, but basically stuff that would be passed to offspring).
Why would you manufacture anything if you can grow it? This’ll probably remove great deal of the maintenance too.
I'm not anti-GMO but i've been more wary ever since I realized it will inevitably be used to make better citizens (and not by making them better humans).
GE is a double edged sword just like nuclear energy. With great power comes great responsability.
This is often said in defense of GMO, but surely cross-breeding and hybridization - a farmer or gardener attempting to combine the entire genetic entity of one plant with another one (which must therefore be naturally compatible), to increase the potential utility of the result - is totally different to inserting a single gene from a totally arbitrary source (which can therefore be a completely separate life-form)?
... Or is that a logical fallacy?
If your program won’t compile, it has a bug somewhere.
If your program will compile, it almost certainly still has a bug somewhere.
Apparently the edit made him totally lactose tolerant for over a year and now he has very mild lactose intolerance. He says he hasn’t redosed because he wants to improve the plasmid (I think that’s the right term, BUGFIX probably isn’t right) before he takes another run at it.
Here’s his update video from last year on that endeavor. https://www.youtube.com/watch?v=aoczYXJeMY4
He really is doing some neat stuff though. He’s inspired me to do some small bio hacking experiments and it ALMOST makes me want to go back to college.
Amazing and alarming what a smart and dedicated person can do with limited funds.
Imagine if we had the scientific process about literally every aspect in human life since the dawn of civilization? We'd still be making bricks with straw, manure, sand, and water because there wasn't a government sponsored test of the quality of "Roman Cement" therefore we should not use it. Some of these people who criticize a lot of science do so just for the sake of disputing it. It's a good thing this stuff happens. The more anecdotal evidence we have, the more likely it can be subject to legitimate scrutiny and understand certain pitfalls that these individuals encountered and why. Just telling these people "you're stupid, it's bad for you!" is an ultimate waste of time and just stagnates data collection.
If you make germline edits, this does potentially impact others - you can have children who inherit the edit. They don't have any choice about that (depending on the edit they might randomly not receive a copy of the edited material, but that's luck) and its consequences in them may be undesirable even if the outcome for you, an adult, was fine and anyway your choice.
EDIT: and preventing young women from choosing sterilization.
For abortion, you can claim it impacts others both ways, are you impacting a new born from having its life, or are you preventing a yet to be born child from having a troubled life, and saving family, parents and society its burden.
For a vaccine, are you putting others at risk of a disease by not taking the vaccine?
For bio-hacking, are you possibly creating dangerous virus or bacterias that could harm others, are you making modifications to yourself which will put a tole on the medical system if you later need assistance or special treatment if you mess up, etc.
This is the same reasoning with drugs often, will your modified behavior cause harm to others if you consume them?
Etc.
None of this is black and white, but I think having these discussions on "what harm to others it can cause" is justified.
The first problem is that you are claiming that the child's body is the woman's body. It isn't and you know that. You know that is it an entirely distinct human being with it's own DNA, etc, etc. In order to justify abortion, you would need to argue that murder (which is the direct, intentional killing of an innocent human being) is justifiable. The logical consequences are devastating. For example, if murder of the most innocent, powerless, dependent, and vulnerable, by those with power over them no less, becomes justifable, then it becomes impossible to argue for the legitimacy any human rights whatsoever. You can't just make up moral principles out of thin air or assign arbitrary boundaries to them according to taste.
The second problem is that this logic seems to put the cart before the horse. In other words, you've decided that abortion is a right axiomatically and you've decided to dismiss anything that would undermine that belief. But the moral acceptability of abortion is not axiomatic and must be judged according to prior moral principles.
"This is exactly the logic conservatives use with the Covid Vaccines but because liberals "know what's good for everyone," people shouldn't be allowed to make the "my body my choice" when it comes to a vaccine."
See my first point above. Here conservatives are indeed speaking of just their own bodies, so this is a false equivalence.
What makes us human beings is our personalities, our brains. Chop off my hand, I'm still me. Destroy my brain, I'm deceased regardless of how long you keep my other organs going.
A fetus does not become human, IMO, until it can have a personality and sentience. Women have the power to create without consent of the created; the power to destroy their not-yet-human equally lacks the consent of the created. If the latter is prohibited than so should the former.
From a societal view, we have enough people. Every way to support people consensually choosing to not have children should be encouraged.
You know that it isn't entirely distinct, it is inside and completely dependent upon another human.
> "In order to justify abortion, you would need to argue that murder (which is the direct, intentional killing of an innocent human being) is justifiable."
How do you feel when a woman has a miscarriage? The child is dead, the mother's body deliberately killed it. Is she culpable? Is it different if her brain cells are involved in the rejection of the child? Isn't that you assigning arbitrary boundaries according to taste?
There have to be arbitrary boundaries, the universe is a single thing, the idea of there being separate things inside it is a human convenient fiction, it's not real. The boundaries between things are fuzzy and disappear the closer you look at them. There's "just" atoms and energy, everything else is concept, if we want morals in the universe we have to make them up, and if we want them to apply to people, we have to make those up too.
I think it's pretty obvious that right implies morally acceptability. You can't claim to have a right to something evil. (The reverse is not true. It is morally acceptable to own a Tesla, but you don't have the right to one.) Alterations can be harmful and any harm that is not a justifiable side effect is by definition immoral. That it's your body does not somehow negate the immorality of self-harm. Indeed, direct self-harm is even worse morally than harm of others because it is in direct opposition to one's own good (arguably, harm of others is also a form of self-harm, specifically harm of one's character and will and habitus, of one's intellect because of the harm of one's reasoning faculties it entails, and harm of the common good). So you cannot say you have the right to self-harm. This is just a strange consequence of the incoherent ideology of absolute autonomy.
Now, if you have, say, a defect in your body that is preventing the proper functioning of your body, then we may correct this defect through intervention. This is restorative, that is, it seeks to restore the function due to the body by virtue of how it ought to function. This is the basis of medicine.
Another false view that can nudge people toward the view that any modification is acceptable is metaphysical materialism, also incoherent. In that case, crude metaphors between artifacts and living things are elevated to the status of the truth and since it doesn't matter what we do to an artifact per se. We can modify a computer any way we wish because there is no fact of the matter about what a computer is or how it should be apart from human intent. It is just a collection of things arranged in a particular way incidentally that puts them in a series of incidental causal relations, but there is no inherent tendency for these things to exist in these relations and no accounting for one in terms of the others except through the lens of human intent. Living things aren't like that.
I'm in favor of drug use (at least almost all), tattoos, body modifications, trying experimental medicine, everything that affects your body. I can see drawing the line at "doing this thing could become a contagion so you can only do it in BSL-4 isolation". But it's your body, your life, you want to spend it stoned or a voluntary amputee or whatever so long as don't take from me, then you should be free to do so.
If you couldn't gouge out your right eye and cast it from thee (Matthew 5:29), would you still have freedom of religion?
> "So you cannot say you have the right to self-harm. Now, if you have, say, a defect in your body that is preventing the proper functioning of your body, then we may correct this defect through intervention. This is restorative, that is, it seeks to restore the function due to the body by virtue of how it ought to function. This is the basis of medicine."
What about vaccination and such where the intent is to cause a small amount of harm now, to prevent a problem you don't have - and may have never faced in any future. This is not restoring a defect, it's augmentative and preventative. Do you not have the right to cause that small harm? And yet that's in opposition to your "direct self-harm is even worse morally than harm of others because it is in direct opposition to one's own good". It's in alignment with one's own good. So, for example, are the torn muscle fibres when weight lifting, or the buildup of lactic acid causing muscle burn when running, or working through the night with no sleep in an emergency.
Less seriously, piercings for jewellery are self-harm and for one's greater happiness in appearance. Do people have no right to wear small pointy uncomfortable high-heel shoes? Is that immoral?
> "I think it's pretty obvious that right implies morally acceptability."
I think it's pretty obvious that there isn't a single "moral acceptability" which everyone agrees on. For example some have the right to eat animals and others consider that morally inacceptable. Some US states have different rules on public nudity than others, and some groups of people have different opinions on the morality of it.
> "You can't claim to have a right to something evil"
Not sure I follow this one; you can't really have a right to murder others, but in the sense of self-harm is it really "evil" to hurt yourself? What is evil and also doesn't involve harm to anyone else?
> "For example, do you have the right to lobotomize a perfectly healthy brain?"
One could argue whether a "perfectly healthy" brain would choose to lobotomize itself, but let's say it does - why do you say that's "very problematic"? The main objection I have - assuming the person is considered sane and healthy - is who is going to pay for the care afterwards, i.e. the harm rolls over onto others and is not purely self-harm. If you could sign that away - it's basically a slow and horrible suicide method.
I think it's pretty obvious that right does NOT imply moral acceptability, and that you can have a right to something evil. In our societies, we have a great deal of disagreement about what things are morally acceptable/permissible. For a good liberal society, we may allow many things that we think are actually immoral, even evil. You need look no further than, say, the religious arena (including atheists) for such disagreement. People disagree about the moral acceptability of worshiping different gods (or about worshiping any 'gods'), calling someone an asshole, teaching your children one thing or another about sex, about engaging in particular sexual acts, about giving charity (or refraining from giving charity) to certain causes, and so on.
We grant people rights to do certain things (and I'm glad we do), but that does not entail that those things are therefore morally acceptable/permissible. You have a right to do them, but maybe you ought not.
The technical requirements to this kind of limit the number of people that are going to really mess themselves up. I would be more worried about people accidentally releasing edited bacteria or viruses.
From what I understand it’s hard to order the sequences of DNA or RNA you’d need for this without someone noticing. It’s probably not impossible, but not something anyone is going to bungle into.
Anyway, Maybe a few years ago the barrier to entry was too high for most people but that is so not the case anymore. Even just this year a friend sent me a link to a $2000 gizmo that was about the size of a pack of gum that could do desktop sequencing. Generating your own custom DNA in your bedroom is considerably harder (but not impossible) but it is not at all difficult to order a custom sequence from a half dozen different retailers. The file you send them is just a big chunk of a few letters. It’s assembly code with no comments (yes I know folks do put in comments in text sometimes and sometimes biologically but just like always comments lie more than not), they functionally cannot be checking for what it could do if what you’re working on is even remotely novel. For the price of a playstation you can have it delivered to your door next day air.
There are very real risks here for our entire planet. Possibly one of our largest risks. To hide behind “it’s too difficult for a malicious actor” is not helpful. I think genetic literacy is maybe the only thing we all can do as we might be entering very weird territory as a closed system that has a bent towards global narcissism and self harm.
Something that gives me some comfort is that to be good at it you really have to do a gross and difficult deep dive into how all life works and I think there are very few people that could do that and still feel like it’s ok to do harm at even a small scale. The things we are going to see in the next few years will be pretty fucking weird. I guess when you get down to this level, the only thing to have is something like faith. Eeeek.
What makes me feel alright is that I think to do some damage is not a technical or money issue but an issue with taking a dive on the closest thing we have to the meaning of life and I'd hope that that study would be the sort of thing that keeps someone from being the worst kind of monster. I mean, we have a ton of tools out there to be awful, why this?
I guess what I'm saying is that to look at this set of problems and say "someone else probably has it handled" or assume anything we know is even slightly predictive of the future is silly. I think it's important for those that care to get an understanding of biology as we are now at an unprecedented point in existence where there's just not a way to feel too comfortable. Added benefit, the more people that get that understanding the closer we might get to yeast that eats plastic or mushrooms that make insulin.
If writing software required being a licensed engineer then you would without a doubt see reactions to open source, calling it deeply irresponsible and dangerous.
Cystic fibrosis significantly impacts gut function and many people with it do poorly with dairy products. I could see this and/or some variation of this being valuable therapy for the CF community while we wait for better answers.
Although best known as a deadly lung disorder, the impact on gut function is a major problem that just gets less press but is something people with CF and their loved ones fret about a whole lot. I think if you could do something to improve gut function fairly quickly and semi permanently, you would likely reduce a lot of their other issues as well because people with CF are typically underweight, sometimes severely underweight, and their chronic malnourishment is a major underlying source of their inability to fight off infection.
That's because it has barely begun to become viable IRL while science fiction writers have been using it as a convenient punching bag for decades -- without ever stopping to ask if they should.
https://en.m.wikipedia.org/wiki/Tuskegee_Syphilis_Study
https://encyclopedia.ushmm.org/content/en/article/nazi-medic...
https://pediatricsnationwide.org/2019/11/25/a-major-mileston...
And this source agrees with your statement:
The triple therapy, known as Trikafta, could effectively treat 90% of people with cystic fibrosis.
https://pediatricsnationwide.org/2019/11/25/a-major-mileston...
When I was on CF lists, I was hearing that the first drug in this class that had recently come out only helped five percent of people with CF (I was specifically hearing that it helped people who were double F508del and that was only five percent of all CF patients, IIRC) at a cost of at least $250k annually, so "if you can afford it or live in a country that covers that for you."
I was also hearing people say that some of the stats were dummied up and exaggerated because so much money was at stake.
I left all the CF lists years ago. The figures I'm seeing today don't fit with what's in my head and I don't know how to sort out what's true and what's PR nonsense and it doesn't really matter to me personally. I and my son with CF are both off all drugs and have lived on very little money for years.
Good for them. I'm sure this makes me even more irrelevant, "crazy" and stupid in the eyes of the world than I was already. Which is no big deal. I don't really care. I'm just leaving this comment because I asked you for a citation and then found my own and deleted my comment.
I don't want people to think I'm being a weasel or anything. The articles I'm finding seem to agree with you. I don't know what to make of that and it's not really relevant to my life. I will never have the money for these drugs and don't really care.
Have a good day.
Like an actual breakthrough with an actual cure for a specific disease. It's remarkable.
Here's to more cures.
God bless.
A modified tree that drops nuts of (nearly) pure graphite or some other form of nondecomposable carbon which we can easily harvest and sequester.
Photosynthesis in humans to supplement our energy consumption. Shoutout to Knights of Sidonia.
I understand the ethical issues surrounding the second one.
Well, we sort of have that in the form of specifically engineering bacteria to create some drugs, notably insulin. Though I’m sure graphene require additional biological structure and that is a much harder problem them adding/removing a few genes.
To match the IEAs projections for carbon capture (7.5 gigatonnes CO2 captured per year in 2050) you would need around 3*10^12 trees. That's about the same as the total number of trees in the world. So you need to replace all of the worlds trees, with the corresponding destruction of many of the biggest ecosystems on earth.
An added benefit would be if the wood after harvested lasted longer than a cedar.
I'm guessing the reason this isn't possible is more due to the fact that it's just not financially worth the investment than it being too difficult.
But I'd love to hear from someone who knows more about this stuff.
The success of this should mean that many genetic liver conditions can soon be treated this way.
For other organs, I expect a longer wait.
What's wrong with "CRISPR gene editing shown to cure Transthyretin amyloidosis in patient study"
Maybe I'm now one of the old people who doesn't understand new usage of language. But I understand that usage dictates grammar rules, but I feel I missed something.
But it's a terrible title for an article meant for laypeople like us. The words "transthyretin amyloidosis" are utterly meaningless to me and most HN/NPR readers, but "He Inherited A Devastating Disease" tells me what kind of disease it is (devastating, heritable) and why CRISPR gene editing might be a big deal.
It also helps clue me into the contents of the article. Because it is as much a summary of a key research result, as it is a profile of Patrick Doherty, a man affected by the disease. That's entirely missing from your title.
None of this is new. "Write for your audience" is as old as writing itself.
I understand your asking a rhetorical question but in the interest of having a discussion about this I’ll try to give you an answer. Imagine a title like “Xonuicfixs shown in zenocttkike to cure Xclatngt”
Reading that you might thing “wrf are they talking about?” But imagine I just change the title to be more like:
“A breakthrough in Xonuicfixs shows promise as a cure for a range of decided.”
Have I lowered the information content of the title? Yes, certainly. However I have also made it very apparent to you an others who don’t know the subject matter why this is a worthwhile achievement, and this might therefore just become the first article that you read and learn about Xonuicfixs.
And really shouldn’t that be the job of a title? To convey to potential readers why the content might be with their time. And I would note that this is entirely different from clickbait which tires to lure you into content with no information about what the content is.
edit: Strike that, hadn't realized that the article title and the HN link differed so much.
https://www.biospace.com/article/releases/verve-therapeutics...
https://en.wikipedia.org/wiki/CRISPR_gene_editing
https://news.harvard.edu/gazette/story/2020/11/coated-nanopa...
Most people won't use it but there will be an active market , legal or illegal, for the modifications. The Sci-Fi future is here.
This is really great. Being born into the wrong set of genes seems like an incredibly silly thing to restrict humans to. Allowing humans to choose their appearance seems like it could resolve a bunch of psycho social issues and hopefully people can live more happy lives (although I am aware that these things often lead to as yet unknown problems of their own…)
I may soon be able to drink again after having my genes edited, but I'm not sure I would do it
Edit: Something not often discussed is that Bill Wilson was hoping to make money from this book, being dead broke in the middle of the Great Depression. The first people he helped sober up convinced him that he had to give it away freely.
This is the first published IV-administration of a gene-editing CRISPR payload. Interesting convergence with the nanoparticles used in mRNA vaccines. Sickle-cell and immune reprogramming edits have been made on cells removed from the body. Josiah Zayner infamously injected himself with CRISPR at a conference years back, but (1) unpublished, AFAIK and (2) he had been drinking which likely attenuated the payload [and (3) he wanted bigger muscles, not to treat a disease]
What are some good investment opportunities if you think this is going to revolutionize the world in the next 30-50 years?
Any more suggestions?
a) need to edit liver cells only, not the entire organism, b) the disease is deadly, so the patients do not have much to lose if anything goes wrong.
Ethical researchers are thus far being super-cautious about modifying the genome of someone who's likely to have children. While the article doesn't say, I wouldn't be surprised to learn that he has explicitly agreed not to have any (more?) children.
Straight from Wikipedia: https://en.wikipedia.org/wiki/LEAPER_gene_editing
I bet a dip into the specialized papers will show a multitude of these techniques.
[0] https://www.researchgate.net/post/How-does-the-CRISPR-guideR...
Eg selective fast twitch / slow twitch muscle fibres.
https://www.sciencehistory.org/distillations/the-death-of-je...
> Doudna expressed similar concerns about CRISPR.
> “I hope that we don’t get ahead of ourselves with this technology. As exciting as it is, I really would like to see . . . people take a very measured and responsible path forward, where there’s careful vetting along the way,” she said. “Of course, the challenge is that patients are waiting, so you don’t want to delay unduly. But you also want to be safe.”
Damn this sucks to read. To think of how much progress we might have made in a promising field if we could just get over one person dying. Yes it's awful, and we shouldn't be flippant, but we're talking about work to help _humanity as a whole_. This isn't the same as saying "the means justify the ends." This is just saying, "Shit, we fucked up there, let's not do that again, but let's keep learning." I don't know how you ever advance without making mistakes.
That could be useful in the future.
Yes genetic modification is another wonderful technology that humans will weaponize….like atomic energy, like the internet…