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Baby is healed with first personalized gene-editing treatment

nytimes.com

161–170 of 570 posts

Re: Baby is healed with first personalized gene-editing treatment

#161

> 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…

> 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.

This isnt entirely unlike the method mRNA vaccines use. Through some clever biochemistry, mRNA vaccines get bits of code into cells where the cell's built in code compilers manufacture proteins that induce immunity.

We have developed software patches for our biology.

Re: Baby is healed with first personalized gene-editing treatment

#163

> 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…

Gene therapies are pretty incredible. Some of them are still making a button-hole with a machete, but that's relative to the previous medical intervention of a button-hole with a tank's main gun. One of the treatments for sickle-cell involves switching off the gene that makes the malfunctioning red blood cells, but of course that's not sufficient; you'd stop making red blood cells completely and you'd die. So it's co…

High affinity RBCs would actually be a disadvantage for athletics. You actually don't need very high affinity to pick up oxygen from the lungs -- your lungs are comparatively extremely high in oxygen. What matters more is being able to drop the oxygen off in peripheral tissues. Higher affinity means that it's harder to actually deliver the oxygen, which is why we evolutionarily developed the switch away from fetal hemoglobin.

Re: Baby is healed with first personalized gene-editing treatment

#164
post #88

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.

If you can affect germline cells, then I don't see how it's not a slippery slope. (I'm not arguing against doing it, just that it is a slope and the slope is slippery.) No designer babies necessary.

I'll steelman "fixing defects" by sticking to serious hereditary diseases (and yes, only those that correspond to one or a few known genes). As more and more conditions become treatable, the population with access to resources will have lower healthcare costs by being less susceptible to problems. (Which is a good thing, note!) Insurance companies will have more and more proxies for differentiating that don't involve direct genetic information. Societally, "those people" [the poor and therefore untreated] cost more to support medically and are an increasing burden on the system. Eugenics gains a scientific basis. Do you want your daughter marrying someone genetically substandard, if you don't have the resources to correct any issues that might show up? Probably not, you're more likely to want to build a wall between you and them. Then throw over anyone who falls behind the bleeding edge of corrections.

It'll be the latest form of redlining, but this time "red" refers literally to blood.

Re: Baby is healed with first personalized gene-editing treatment

#165

Earlier quoted context omitted.

Farming? This will be used in warfare.

Not under the current way we do things, I don't imagine. So the real trick here isn't the mRNA, it's the nanobubbles. Basically, you're putting these bits of mRNA into these little fat bubbles and then injecting those into the blood. Making those bubble shelf stable is really hard , hence the issues with temperature and the covid vaccine. To then make those little fat bubbles stable-ish in the blood is also a really…

I think it doesn't need to be a direct weapon to be used in warfare. You can genetically modify your own military.

Re: Baby is healed with first personalized gene-editing treatment

#166
post #99
post #83

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 less interested in detecting genetic modification for the purposes of discrimination than making sure it's available to everyone.

Assuming requisite safety of course.

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