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Baby is healed with first personalized gene-editing treatment

nytimes.com

371–380 of 570 posts

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

#371

Earlier quoted context omitted.

"Effective Altruism", something I find myself aligned with but not to the extremes taken by others.

Technically lesswrong is about rationalists not effective altruists, but you're right in a sense that it's the same breed. They think that the key to scientific thinking is to forego the moral limitations, not to study and learn. As soon as you're free from the shackles of tradition you become 100% rational and therefore 100% correct.

Approximately no one in the community thinks this. If you can go two days in a rationalist space without hearing about "Chesterton's Fence", I'll be impressed. No one thinks they're 100% rational nor that this is a reasonable aspiration. Traditions are generally regarded as sufficiently important that a not small amount of effort has gone into trying to build new ones. Not only is the case that no one thinks that anyone including themselves is 100% correct, but the community norm is to express credence in probabilities and convert those probabilities into bets when possible. People in the rationalist community constantly, loudly, and proudly disagree with each other, to the point that this can make it difficult to coordinate on anything. And everyone is obsessed with studying and learning, and constantly trying to come up with ways to do this more effectively.

Like, I'm sure there are people who approximately match the description you're giving here. But I've spent a lot of time around flesh-and-blood rationalists and EAs, and they violently diverge from the account you give here.

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

#372

> 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…

Thanks to DOGE you might read less and less about this kind of things.

Unfortunately, this is true. From the article:

> The implications of the treatment go far beyond treating KJ, said Dr. Peter Marks, who was the Food and Drug Administration official overseeing gene-therapy regulation until he recently resigned over disagreements with Robert F. Kennedy Jr., the secretary of health and human services.

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

#373

This is incredible work. Its jaw dropping to learn that something like this is possible at all. Sometimes I wish I could work for a company whose products make a meaningful positive contribution to the work. Do companies like this have a need for SWEs? Are there opportunities for a backend SWE without any background in hardware or biology?

Yes. Aldevron and IDT are two companies (owned by danaher) that collaborated to make this happen. They have multiple authors listed in the NEJM article.

https://jobs.danaher.com/global/en/search-results?keywords=S...

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

#374

If someone in the year 2050 was to pick out the most important news article from 2025, I won't be surprised if they choose this one. For those who don't understand this stuff - we are now capable of editing some of a body's DNA in ways that predictably change their attributes. The baby's liver now has different (and better) DNA than the rest of its body. We still are struggling in most cases with how to deliver the D…

Easiest way to do this stuff is before fertilization when you have one egg and one sperm to work with. Delivering change through a multicellular organism is very challenging. All this stuff like transgenic mice are set up in mutant crosses before this stage, before mating really. Eventually this will be the outcome of our species to edit the gametes themselves. The issue to overcome for this again won’t be technologi…

>>>Easiest way to do this stuff is before fertilization when you have one egg and one sperm to work with. Yes. But it seems, that nature so far is still better than we at picking better quality cells in laboratory environment. Not all eggs and sperms are equal - the difference in DNA quality varies.

>>>Eventually this will be the outcome of our species to edit the gametes themselves. The issue to overcome for this again won’t be technological as that is pretty much solved but getting people over their own “ick” factor. This is a new fear unlocked, as this will be like another cosmetic surgery procedure, which from my minimal understanding does not affect DNA that is delivered to offsprings - that could be changed but require a lot more work, but like you mentioned - it is easier to do before fertilization :). It is catch22 situation rn.

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

#375

Does this mean when they grow up, their own offspring will also have this defect and require a correction? And, if so, does this mean it is now introducing this defective gene into our gene pool? I know this is an issue with caesarean section. It is becoming more prevalent because those who require it are surviving, making it more likely to happen in their offspring.

they could CRISPR his relevant reproductive cells, this general topic is an important subject of discussion

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

#376

Good thing RFK pushed out the official overseeing this financing and the current administration is actively defunding the organizations that produced this. Better to have more disabled or dead babies instead of science. /s

The current administration doesn't care about kids. They only want you to not terminate a new kid from being born. That they care lots about. What happens after birth is not their concern. Also, I think when they say they want more babies, they want a specific subset of babies to increase.

> The current administration doesn't care about kids.

Of course they do. But untold amounts spent on very few kids could be spent elsewhere on many more. Federal budgets are a zero-sum game.

> Also, I think when they say they want more babies, they want a specific subset of babies to increase.

I've seen quite a few conservative commentators celebrate that the massively disproportionate levels of African-American abortion have been reduced, resulting in more African-American people being born, and zero bemoaning it. So maybe you're right.

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

#377

Earlier quoted context omitted.

"Effective Altruism", something I find myself aligned with but not to the extremes taken by others.

Technically lesswrong is about rationalists not effective altruists, but you're right in a sense that it's the same breed. They think that the key to scientific thinking is to forego the moral limitations, not to study and learn. As soon as you're free from the shackles of tradition you become 100% rational and therefore 100% correct.

So much vitriol. I understand it's cool to hate on EA after the SBF fiasco, but this is just smearing.

The key to scientific thinking is empiricism and rationalism. Some people in EA and lesswrong extend this to moral reasoning, but utilitarianism is not a pillar of these communities.

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

#378
post #366
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's almost exactly the premise of the movie "I am Legend," But it uses CRISPR instead of a Virus as the delivery mechanism.

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

#379
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 remember from a few few years back that the lipid coating may have caused problems for the liver, when treating people for diseases that needed to target a lot of tissue, such as muscle disorders. Is that still the case?

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 something you only need to take once so the toxicity issue could be dodged. Doh. That was in 2017.

https://www.statnews.com/2017/01/10/moderna-trouble-mrna/

By the time COVID vaccines came around a few years later there was no evidence they had fixed the problems with lipid nanoparticle delivery. I looked for such evidence extensively at the time, for example, announcements by Moderna of breakthroughs or trials of new drugs. Today the situation seems not much different. Note that Moderna's wikipedia article has a section on "rare disease therapeutics" but it's literally empty:

https://en.wikipedia.org/wiki/Moderna#Rare_disease_therapeut...

Because of their failure to progress beyond COVID vaccines Moderna's share price got slaughtered, falling from a peak of ~$450 to ~$25 today.

I don't know if other companies were able to find breakthroughs here, after COVID I stopped following the topic. Unfortunately, although mRNA tech has great potential, when normal safety standards were reimposed it appears that Moderna went back to being unable to make anything safe enough to launch.

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

#380

Earlier quoted context omitted.

How does it know how to gps around? From what I know everything down there is a chemical reaction with some minimal physical motion, but how do you program it to know where to change and what and how.

It’s more like a “ctrl+F” for DNA. Hopefully there’s only 1 match (the target site).

Well its more like search and replace, where you cross your fingers that it only replaces the words you are trying replace without impacting the rest of the text in the document.
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