Hacker News Board Hacker News, browsed like a classic message board
Live data from Hacker News

Baby is healed with first personalized gene-editing treatment

nytimes.com

Post reply on HN
Print view View original

361–370 of 570 posts

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

#361

Post by rkangel »

palisade wrote: post #322

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.

> know this is an issue with caesarean section. It is becoming more prevalent because those who require it are surviving

You state this as a fact and I've heard it as a strong hypothesis, but I wasn't aware of much evidence to confirm it?

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

#362

Post by cnity »

alexey-salmin wrote: post #310
gr_norm wrote: post #243

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.

That community is basically the "r/iamverysmart" types bringing their baggage into adulthood. Almost everything I've read in that sphere is basically Dunning–Kruger to the nth degree.

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

#363

Post by cnity »

rob74 wrote: post #277
tuna-piano wrote: post #224

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…

That's all very cool, but there are also articles like this one: https://www.theguardian.com/us-news/2025/feb/20/trump-nih-cu... - I'm not able to read the Times article because it's paywalled, but as other commenters have mentioned, this research was funded by the NIH, which the Trump administration is currently in the process of defunding. So, if further progress along this road will be made, it'll probably be much…

Or, it means that funding will be secured in the private sector. Basically by investors that focus on revenue streams (read: extremely expensive private healthcare).

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

#364

Post by rubidium »

j16sdiz wrote: post #316
dclowd9901 wrote: post #204

Earlier quoted context omitted.

I literally said the same thing out loud. I had heard about CRISPR a while back but most reporting on it kind of hand waved over the mechanisms of how it actually accomplishes its work. What these researchers have figured out to make this work absolutely blows my mind.

AFAICT, CRISPR still make many bad edits. We relies on the fact that most of those bad edit won't survive.

It can make “bad edits” eg off target effects. But in this case there were, as far as is known, none. It’s aided that this was a single nucleotide defect.

They specifically tested for off target edits in the mouse study and found no harmful edits (and very rare off target ones). That plus the specific targeting of the liver cells (no germ line effect expected), makes this a low risk approach and certainly better than doing nothing.

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

#365

Post by nopakos »

declan_roberts wrote: post #101
jmcgough wrote: post #68

Earlier quoted context omitted.

Yep, this effort is the culmination of 50 years of research. Could be the last harrah of the NIH with the amount of cuts we've had and the scientists who are taking jobs in other countries.

Unfortunately a staggering amount of research in other countries is largely funded by the NIH/USA.

That may be partially true, but it's also important to understand that the US benefited a lot from that. Scientists from all over the world moved to work in the US, students looked forward to studying there and working in US companies, etc.

That is changing. Children in my country are moving from learning English to French and German in order to study in European universities. This started after Brexit and will accelerate now.

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

#366

Post by cm2187 »

Balgair wrote: post #83
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…

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

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

#367

Post by junon »

Teever wrote: post #99
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…

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…

RNA is a byproduct, not a "source of truth" in technical terms. The DNA is. DNA is converted to RNA and then executed and then discarded, per my understanding. The DNA is still AGCT.

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

#368

Post by GenshoTikamura »

kubb wrote: post #355
GenshoTikamura wrote: post #153

Earlier quoted context omitted.

One can not simply raise valid concerns about gene editing technologies in the hands of the entities that don't hesitate sending people en masse to kill and die and otherwise manifest their fascist cravings in the open, here on HN and walk away undownvoted

Science fiction pattern matching is a problem nobody talks about. People see something that vaguely resembles a movie that they saw and act like that movie is reality now.

Another problem nobody talks about is the cognitive dissonance of living in the world today but dismissing all matching patterns as science fiction or conspiracy theories. Maybe narrow focus and short attention span are to blame

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

#369

Post by aucisson_masque »

raldi wrote: post #331
mondaygreens wrote: post #328

Earlier quoted context omitted.

How can they pass it on when they don't have the defect any more?

The DNA was only edited in the liver. But by the time this baby grows up and starts a family, we'll probably be able to fix that, too.

That's a bold claim. The baby is 9 month old, there are many things that can still go wrong with this experimental treatment.

Hopefully not, but even then no one can say what progress will make science in the next 25 years.

Back in the 50's people thought we would be driving in flying car in 2000.

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

#370

Post by rubidium »

0x1ceb00da wrote: post #248
TheJoeMan wrote: post #207

Earlier quoted context omitted.

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

So you create a molecule that binds to a certain location in the dna, and then deploy a billion of them?

More or less, yes.

An interesting part of the study was determining what a clinical dose _should_ be. You need enough to edit enough liver cells. But don’t really want to completely overdo it to limit potentially negative side effects. Seems like they got it right enough here, with the first dose having some effect and the subsequent dose having more.

Post reply on HN