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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

#51

Post by xiphias2 »

pacoWebConsult wrote: post #13
chewbacha wrote: post #3

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

From a purely utilitarian perspective, funding research like this is not an effective use of dollars at the margin. How many people could we save if an equivalent amount was put into reducing obesity, smoking, and drinking? How many people could we save if we stopped spending money we don't have to do things that the government isn't competent at allocating anyways? That's not to say the research itself is not impres…

It's super effective funding.

There are known DNA changes that would probably help all people with chronic diseases, but it's ethically more accepted to go for the more fatal diseases and cleaner cases first, like a rare mutation with a high fix rate.

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

#52

Post by scotty79 »

vessenes wrote: post #40

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.

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

#53

Post by caycep »

poyu wrote: post #31
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…

Made it sound like it's a computer, is it Turing complete?

I think I recall reading at least some papers or at least exercises trying to draw analogies between Turing machines and ribosome/proteonsome and other type of cellular proteins, but I can't remember back to that class some 20 years ago...

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

#54

Post by caycep »

forgotpwagain wrote: post #36

Detailed New England Journal of Medicine article about this case: https://www.nejm.org/doi/full/10.1056/NEJMoa2504747 And an Editorial piece (more technical than the NYT): https://www.nejm.org/doi/full/10.1056/NEJMe2505721

thanks for this, I think all these lay articles on biomedical news should definitely be accompanied by the paper

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

#57

Post by caycep »

colechristensen wrote: post #5

>KJ has made medical history. The baby, now 9 ½ months old, became the first patient of any age to have a custom gene-editing treatment, according to his doctors. This is _not_ the first human to be treated with a treatment under the wide umbrella of gene therapy based on their own edited genes. There probably is a more narrow first here but the technical details get lost in journalism which is a shame.

I want to say, maybe it's better to say first human under proper IRB/regulatory compliance. Some rogue academic in China tried it a few years ago, if I recall, but with absolutely no oversight and he was pilloried. Also I don't think there is much details about what he actually did...

https://www.npr.org/2023/06/08/1178695152/china-scientist-he...

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

#58

Post by psychoslave »

pacoWebConsult wrote: post #13
chewbacha wrote: post #3

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

From a purely utilitarian perspective, funding research like this is not an effective use of dollars at the margin. How many people could we save if an equivalent amount was put into reducing obesity, smoking, and drinking? How many people could we save if we stopped spending money we don't have to do things that the government isn't competent at allocating anyways? That's not to say the research itself is not impres…

That is not comparable at all. To save people from obesity, smoking and drinking, you don't need more resources on fundamental research. You need different education, and socio-economical programs, possibly even less funds on the overall: if no resources is spent anymore in promoting bad habits, you end up with more financial resources and a healthier population.

Instead if no resources is allocated on developing all the technical requirements to do such a thing, humanity ends up with less tools to heal itself, and that's it.

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

#59

Post by WorkerBee28474 »

os2warpman wrote: post #41
pacoWebConsult wrote: post #13

Earlier quoted context omitted.

From a purely utilitarian perspective, funding research like this is not an effective use of dollars at the margin. How many people could we save if an equivalent amount was put into reducing obesity, smoking, and drinking? How many people could we save if we stopped spending money we don't have to do things that the government isn't competent at allocating anyways? That's not to say the research itself is not impres…

I think you may be operating under the assumption that the extremely expensive price tag will need to be repeated for each patient. In reality, as this process becomes more mature it is going to become inexpensive. The reduction in cost will almost certainly be similar to reduction in cost needed to sequence an individual's genome, which has fallen from tens of millions to hundreds of dollars. The only catch is that…

> 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.

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

#60

Post by [deleted] »

delfinom wrote: post #46
pacoWebConsult wrote: post #13

Earlier quoted context omitted.

From a purely utilitarian perspective, funding research like this is not an effective use of dollars at the margin. How many people could we save if an equivalent amount was put into reducing obesity, smoking, and drinking? How many people could we save if we stopped spending money we don't have to do things that the government isn't competent at allocating anyways? That's not to say the research itself is not impres…

Because reducing obesity, smoking and drinking is not a money problem in the slightest.

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