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Baby is healed with first personalized gene-editing treatment

nytimes.com

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

#421

Post by abcd_f »

DrScientist wrote: post #384
MrZander wrote: post #11

> 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…

Bear in mind that they intentionally choose something that was soluble - ie the easiest thing possible. So it's doesn't mean everything is now solvable. For example it's no coincidence this is a liver disease as basically almost everything you inject in the bloodstream ends up concentrating in the liver by default - if you needed to target another organ with your LNP it would be much harder. Most of the time people a…

> that was soluble

solvable

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

#422

Post by Symmetry »

cm2187 wrote: post #366
Balgair wrote: 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…

That also sounds like a recipe for a terrifying virus

It would allow a synthetic virus to get a foothold in your cells more easily, but our cells don't make Pseudouridine naturally which throws a big wrench in the ability of a virus to copy itself. And without replication you don't have a serious infection.

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

#423

Post by nomadpenguin »

philsnow wrote: post #420
nomadpenguin wrote: post #163

Earlier quoted context omitted.

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 he…

I thought the evolutionary impetus for fetal hemoglobin was because it greatly increases the efficiency of fetal oxygen uptake across the placental interface? From shadowgovt: > I have seen no literature on whether having fetal RBCs in adulthood has any benefits or drawbacks (besides changing the affinity ratio for their fetus if the patient gets pregnant This was exactly the question that popped into my mind when I…

Yes, that is true. I phrased that badly -- it's more that we didn't take the evolutionary branch where we retain the fetal hemoglobin because it is maladaptive in adults.

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

#424

Post by morsecodist »

kmmlng wrote: post #339
morsecodist wrote: post #317

Earlier quoted context omitted.

Effective Altruism is such an interesting title. Almost no one views their Altruism as ineffective. The differentiator is what makes their flavor of Altruism effective, but that's not in the title. It would be like calling the movement "real Altruism" or "good Altruism". A good name might be rational Altruism because in practice these people are from the rationalist movement and doing Altruism, or what they feel is A…

I suppose in the beginning, it was about finding ways to measure how effective different altruistic approaches actually are and focusing your efforts on the most effective ones. Effective then essentially means how much impact you are achieving per dollar spent. One of the more convincing ways of doing this is looking at different charitable foundations and determining how much of each dollar you donate to them actua…

This is a super fair summary and has shifted my thinking on this a bit thanks.

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

#425

Post by jillyboel »

_bin_ wrote: post #278
jillyboel wrote: post #187

Earlier quoted context omitted.

Don't be silly, the rich will want their babies to be perfect so gene editing will be legal and considered OK.

Can you explain why this is a bad thing, or is it just “”the rich” bad”?

I'm explaining that gene modification will not be considered illegal or bad because the rich will have a vested interest in it being legal. This is a reply to GP saying:

> use legal mechanisms to discriminate and persecute people who are genetically modified

I believe there is no way this will happen, because legal mechanisms are driven by the whims of the rich, and they will want gene editing to be legal. So there will beno legal mechanisms to discriminate against those who have been edited.

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

#426

Post by squigz »

palisade wrote: post #419
squigz wrote: post #412

Earlier quoted context omitted.

> Another Note: Also, ironically WHO's article I've linked is lamenting that Sub-saharan Africa only had 5% cesarean due to less availability of the procedure. It is their perspective that the increase in percentages is a good thing and indicates progress, instead of being concerning. And, they find Sub-saharan Africa's low numbers concerning, instead. Pretty sure their perspective is that "saving the lives of mother…

Yes, that's what they're indicating. And, it is saving lives. I myself was cesarean section, as was my mother. I wouldn't be here without it. That's the potential conundrum, if it turns out to be vastly increasing the need to save those lives than in the past due to a evolutionary pressure on the gene pool. If the WHO is right and we're going to start seeing 50 - 63% increases by 2030, what's in store for the human r…

Are c-sections replacing 'natural' births, or are they simply becoming more common because we have the expertise? There is a difference

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

#427

Post by mike_hearn »

Gareth321 wrote: post #415
mike_hearn wrote: post #379

Earlier quoted context omitted.

You remember correctly. Moderna had a lot of problems with their drug trials due to the lipid nanoparticles they were using to transport mRNA. They were toxic to the liver upon repeat dosings. Unfortunately, it appears they never found a fix for the problem. Instead they gave up and found a "business solution" by pivoting from drugs to the (at the time) less profitable vaccines, on the grounds that vaccines are somet…

> on the grounds that vaccines are something you only need to take once so the toxicity issue could be dodged But we didn't take these vaccines once. We took many of them. Am I to understand a known side effect is liver toxicity for multiple doses?

You have successfully read between the lines, yes.

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

#428

Post by popol12 »

Gareth321 wrote: post #415
mike_hearn wrote: post #379

Earlier quoted context omitted.

You remember correctly. Moderna had a lot of problems with their drug trials due to the lipid nanoparticles they were using to transport mRNA. They were toxic to the liver upon repeat dosings. Unfortunately, it appears they never found a fix for the problem. Instead they gave up and found a "business solution" by pivoting from drugs to the (at the time) less profitable vaccines, on the grounds that vaccines are somet…

> on the grounds that vaccines are something you only need to take once so the toxicity issue could be dodged But we didn't take these vaccines once. We took many of them. Am I to understand a known side effect is liver toxicity for multiple doses?

I guess the issue is not about taking the medicine once vs twice, but rather « a few times » vs « daily »

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

#429

Post by squigz »

palisade wrote: post #419
squigz wrote: post #412

Earlier quoted context omitted.

> Another Note: Also, ironically WHO's article I've linked is lamenting that Sub-saharan Africa only had 5% cesarean due to less availability of the procedure. It is their perspective that the increase in percentages is a good thing and indicates progress, instead of being concerning. And, they find Sub-saharan Africa's low numbers concerning, instead. Pretty sure their perspective is that "saving the lives of mother…

Yes, that's what they're indicating. And, it is saving lives. I myself was cesarean section, as was my mother. I wouldn't be here without it. That's the potential conundrum, if it turns out to be vastly increasing the need to save those lives than in the past due to a evolutionary pressure on the gene pool. If the WHO is right and we're going to start seeing 50 - 63% increases by 2030, what's in store for the human r…

> Edit Edit: I can't reply to your comment below I think we've hit the leaf end of this post. But, to reply to your question are c-sections replacing natural births or are they just becoming more common? The research I've cited has indicated this is a genetic transfer among female-to-female births of a need for more cesareans.

To reply after a certain number of child comments, you have to open the comment by clicking the timestamp thing

I'm also afraid I don't understand your response. Can you elaborate?

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

#430

Post by palisade »

squigz wrote: post #426
palisade wrote: post #419

Earlier quoted context omitted.

Yes, that's what they're indicating. And, it is saving lives. I myself was cesarean section, as was my mother. I wouldn't be here without it. That's the potential conundrum, if it turns out to be vastly increasing the need to save those lives than in the past due to a evolutionary pressure on the gene pool. If the WHO is right and we're going to start seeing 50 - 63% increases by 2030, what's in store for the human r…

Are c-sections replacing 'natural' births, or are they simply becoming more common because we have the expertise? There is a difference

The research I've cited has indicated this is a genetic transfer among female-to-female births of a need for more cesareans.

"A female-to-female familial predisposition to caesarean section was observed. It could be caused by biologic inheritance, primarily working through maternal alleles and/or environmental factors. The results imply that both mechanisms could be important."

https://pubmed.ncbi.nlm.nih.gov/18540028/

"Large-scale epidemiological studies indeed evidence that women born by C-section are more likely to deliver by Caesarean than women born vaginally, owing primarily to genetic rather than social factors."

https://www.pnas.org/doi/10.1073/pnas.1712203114

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