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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

#81
post #45

Earlier quoted context omitted.

How do you know it's not effective? The cost per life saved is extremely high now , but this stuff gets better over time. How much did penicillin cost to produce originally?

Isn’t penicillin just bread mold? So probably not a great example.

And yet, the first patient treated with mass-produced penicillin used half the total supply, and the stuff was so rare that it was extracted from patients' urine for reuse.

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

#82

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…

30 million people in the US are affected by "rare" genetic conditions.

Yes, but the cures here aren't general. They're highly specific, and the rare conditions have a long tail- large numbers of different conditions, each with a very small population of affected individuals, and likely, the treatments will be somewhat customized for each type of disease.

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

#83

> 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 well worth the 2023 Nobel in Medicine.

Like, the whole system for gene editing in vivo that we've developed is just crazy little discovery after crazy little discovery. It's all sooooo freakin' cool.

https://en.wikipedia.org/wiki/Pseudouridine

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

#84

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…

This argument could be used to stop absolutely any research that isn't dirt cheap. Maybe even the dirt cheap one, because even 100 dollars could go longer way somewhere in the Sahel. It is good that the humanity does not have a one-track mind.

I’ve thought about this recently as well and I don’t know if I have a fully developed view. What is the moral responsibility of all people to pay for medical research or operations that would affect a small number of people. Is it ethical to compel others to pay for the research deemed valuable by some, but not by others. Who is the arbiter of that research’s value?

I could say I believe the government should fund research into fixing people who think cilantro tastes like soap because for most of us it is delicious and promotes healthy diets. Should I be able to compel (tax) you to pay for that research?

Where that line is drawn will always be wrong to someone. How research is prioritized will always be wrong to someone. Is there an ethical way to determine the best use of collective resources and what portion of one’s property must be taken from them to fund that research.

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

#85

Earlier quoted context omitted.

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.

>There's no evidence to support that gene therapy will ever be inexpensive.

My prediction is based on the number of efforts, too numerous to list here, being undertaken to develop lab equipment to automate the extremely labor-intensive workflow and the accumulation of vast libraries of CRISPR-Cas9 screens and dependency maps, the creation of which are also expensive and labor-intensive.

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

#86

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.

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

#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.

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

#89
post #57

>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...

The major difference is that was a hereditary change. So those changes could now diffuse throughout the species over time. As I recall it was a change that reduces vulnerability to HIV infection.

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

#90

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?

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