141–150 of 570 posts
> To accomplish that feat, the treatment is wrapped in fatty lipid molecules to protect it from degradation in the blood on its way to the liver, where the edit will be made. Inside the lipids are instructions that command the cells to produce an enzyme that edits the gene. They also carry a molecular GPS — CRISPR — which was altered to crawl along a person’s DNA until it finds the exact DNA letter that needs to be c…
Earlier quoted context omitted.
> In reality, as this process becomes more mature it is going to become inexpensive. There's no evidence to support that gene therapy will ever be inexpensive. We can merely say that the process may become less shockingly expensive.
What should we as humanity, as society, spend most of our wealth and resources doing? Sending robber barrons and their girlfriends into space?
When a Utopian dies, of anything, the cause is marked and not forgotten until solved. A fall? They rebuild the site to make it safe. A criminal? They do not rest until he is rendered harmless. An illness? It is researched until cured, regardless of the time, the cost, over generations if need be. A car crash? They create their separate system, slower, less efficient, costing hours, but which has never cost a single life. Even for suicide they track the cause, and so, patiently, blade by blade, disarm Death. Death, of course, has many weapons, and, if they have deprived him of a hundred million, he still has enough at hand to keep them mortal. For now.
I think it was here a few years ago that I read a comment saying that sick children will be the Trojan horse for normalizing gene editing of humans, because who could say no to sick children, right? Well, guess it's here now, so how long utill the eugenics wars start?
It's not a slippery slope. Fixing defects is rather straightforward, since it's usually a single gene that needs to be edited. If you want make your baby smarter, taller, or more handsome, it's not so easy because these traits involve 1000s of genes. For this reason I do not think that curying diseases will lead to designer babies.
Earlier quoted context omitted.
One other fun part of gene editing in vivo is that we don't actually use GACU (T in DNA). It turns out that if you use Pseudouridine (Ψ) instead of uridine (U) then the body's immune system doesn't nearly alarm as much, as it doesn't really see that mRNA as quite so dangerous. But , the RNA -> Protein equipment will just make protiens it without any problems. Which, yeah, that's a miraculous discovery. And it was wel…
> [...] then the body's immune system doesn't nearly alarm as much, as it doesn't really see that mRNA as quite so dangerous Please tell me there are measures to prevent this going into the wild. Please tell me this won't be used in large-scale industrial farming.
NYT isn’t super specific here, but they made it sound like the disease treated is liver related. My understanding is that the liver is a good place to start with CRISPR-type gene treatments, in that the liver normally deals with anomalous shit in your bloodstream, say, like CRISPR type edits. So anywhere outside the liver is going to be significantly harder to get really broad uptake of gene edits. It’s crazy encoura…
I don't think it's gonna be that hard. All cells that blood reaches were happily taking mRNA vaccine.
Right off the bat, the liver receives roughly a quarter of all cardiac output, either directly or second hand from the digestive organs. Additionally, the liver has a fenestrated endothelium which, while not completely unique in the body, uniquely allows molecules like lipid nanoparticles (LNPs) to access liver cells. Finally, the liver is the site of most lipoprotein processing, and LNPs can be designed to take advantage of the existing pathways to get the gene editing mRNA into the hepatocytes. All this is to say that if you have a genetic condition that primarily effects the liver, there's a lot more hope for treatment in the near term than for others.
Good lecture on the difficulties of finding appropriate platforms for delivering gene therapies to cells for anyone interested [1]
> To accomplish that feat, the treatment is wrapped in fatty lipid molecules to protect it from degradation in the blood on its way to the liver, where the edit will be made. Inside the lipids are instructions that command the cells to produce an enzyme that edits the gene. They also carry a molecular GPS — CRISPR — which was altered to crawl along a person’s DNA until it finds the exact DNA letter that needs to be c…
> That is one of the most incredible things I have ever read. This is even more great reading behind the above: https://en.wikipedia.org/wiki/Jennifer_Doudna
NYT isn’t super specific here, but they made it sound like the disease treated is liver related. My understanding is that the liver is a good place to start with CRISPR-type gene treatments, in that the liver normally deals with anomalous shit in your bloodstream, say, like CRISPR type edits. So anywhere outside the liver is going to be significantly harder to get really broad uptake of gene edits. It’s crazy encoura…
I don't think it's gonna be that hard. All cells that blood reaches were happily taking mRNA vaccine.
mRNA vaccines are highly localized: you get a sore arm because most of it only gets taken up by muscle cells around the injection site, which spend some time producing the antigen and triggering a primary immune response (the inflammation aka the sore arm).
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Earlier quoted context omitted.
One other fun part of gene editing in vivo is that we don't actually use GACU (T in DNA). It turns out that if you use Pseudouridine (Ψ) instead of uridine (U) then the body's immune system doesn't nearly alarm as much, as it doesn't really see that mRNA as quite so dangerous. But , the RNA -> Protein equipment will just make protiens it without any problems. Which, yeah, that's a miraculous discovery. And it was wel…
I suppose a downside (depending on your perspective) of this is that it will make people who are genetically modified in this fashion trivial to detect. That's good if your goals are to detect genetic modification which may be considered cheating in competitive sports. That's bad if your goals are to detect genetically modified people and discriminate against them. I see a near future where the kind of people who loa…
I'm not arguing about whether the risks of the attenuated virus outweigh the benefits. I think the data are very clear there. (Heh -- and I'm sure the vast majority of people will agree with that statement, even if they disagree on what the clear answer is....)
It's just that one shouldn't mock a belief without including the necessary qualifiers, as otherwise you're setting up an argument that can be invalidated by being shown to be factually incorrect.
As for genetic modification of humans, IMO there are a lot of very good reasons to be wary, most of them social. Fatal hereditary conditions are obviously an easy call. What about autism (not saying there's a genetic link there to use, just a what if)? Or other neurodivergence? Like being a troublemaker in class? Or voting for the party that doesn't control the medical incentive structure? Heck, let's stick with the fatal hereditary conditions, and say the editing does not affect germ cells. Is it ok if the human race gradually becomes dependent on gene editing to produce viable offspring? Or let's say it does extend to germ cells. The population with resources becomes genetically superior (eg in the sense of natural lifespan and lower medical costs) to those without, creating a solid scientific rationale for eugenics. Think of it as redlining carved into our blood.
I don't think discrimination against the genetically modified is the only potential problem here.
As humans, we'll deal with these problems the way we've dealt with everything else transformational. Namely: very, very badly.
Earlier quoted context omitted.
> That is one of the most incredible things I have ever read. This is even more great reading behind the above: https://en.wikipedia.org/wiki/Jennifer_Doudna
A rare case where the list of awards she's received is so long it needs a separate Wikipedia page https://en.wikipedia.org/wiki/List_of_awards_and_honors_rece...