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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

#401

Post by amelius »

jjtheblunt wrote: post #112
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…

> That is one of the most incredible things I have ever read. This is even more great reading behind the above: https://en.wikipedia.org/wiki/Jennifer_Doudna

A darker passage in the history of gene editing, but still an interesting story and something not to forget:

https://www.sciencehistory.org/stories/magazine/the-death-of...

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

#402

Post by something098 »

Traubenfuchs wrote: post #295
thrance wrote: post #106

Earlier quoted context omitted.

Is the global gene pool actually degrading though? I only ever hear that in thinly veiled attempts at advocating for eugenicism. And it never comes substantiated by any research. Anyway, this baby proves we can fix hereditary diseases now.

> eugenicism That comes in many forms: Black/dark one, nazi style, where you outright sterilise or even kill those with unhealthy/bad genes. And white/peaceful one, where you‘d appeal to those with unhealthy/bad genes not to procreate and encourage those with healthy/good ones to do. You can‘t seriously tell me it‘s not extremely unethical for people with huntington‘s disease or cystic fibrosis to have children.

The issue here is that with this approach we have to ask who has to be limited next. Especially if you get older...

>>>You can‘t seriously tell me it‘s not extremely unethical for people with huntington‘s disease or cystic fibrosis to have children. Don't flatter yourself - your genes are basic and ridddled with bad genes. You do not know what time bomb you are carying in your DNA.

The solution that you are offering is quite simple - procreate early as possible and die not in old age and voila - there are no issues in more than 99.99% cases. But something tells me that you are already older than healthy monkey and do not plan to live in a tree - your bones are too old for that and thanks to evolution are not meant for that.

Evolution of humans in future includes even longer lifespan which naturally comes with children produced at much later age than we do now and that comes with diseases to be dealt with, as that is part of evolution. We do not know much about mutations in DNA - they are never good or bad - they are combinations of something. For example - diabetes type 2 seems to be from genes, that allowed humans to survive hunger for long period of time - are those genes bad, because people are obese nowadays? As for mentioned diseases - we value other humans not by DNA, but what they are to us. You would sing a different song, when their offspring would have any of such disease and you are in luck and not planning to have any.

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

#403

Post by Tuna-Fish »

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?

You need billions to cover multiple cells, you don't need many for a cell.

The counterintuitive part is how fast thermal motion is relative to the size of dna.

In body temperature water, the thermal velocity of water molecules jostling around everything is ~600m/s. The nucleus of a human cell is ~6µm in diameter. That is, your average water molecule bounces around at a speed that makes it move from one end of the nucleus to another roughly 100 million times per second.

Larger molecules move more slowly, but they still zip around fast enough that nothing needs to "seek" to a specific position in a cell to get there, everything will touch everything just from thermal random walk in a very short time. So how biology works is that inside the cell there might be just one messenger, which will have to hit a specific piece of dna just right in order to do anything, but that's still nearly instantaneous from our perspective.

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

#404

Post by Cthulhu_ »

deadbabe wrote: post #396

Do you think someday soon we will have the equivalent of an LLM coding assistant, but for gene editing?

Definitely not soon, there's a lot of conditions at the moment for this to be applicable (from what I gather, I'm no expert, just a HN commenter), and well before wholesale gene editing is a thing there will be the legal aspect.

I mean the precursor to gene editing is selective breeding, which on humans quickly leads to eugenics.

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

#405

Post by dagurp »

anthk wrote: post #179
koeng wrote: post #65

Earlier quoted context omitted.

It's fundamentally different than a computer and arguably more complete. The talk of "crawling along the genome" is kinda fundamentally wrong though and is a bit irking - CRISPR kinda just bumps around until it hits a PAM site, in which case it starts checking against sgRNA. Much more random than they make it seem

This is crazier: https://www.sciencealert.com/are-we-all-quantum-computers-wi... About CRISP, it's like the ultimate Perl+Regex for the body.

sounds more like sed

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

#406

Post by Cthulhu_ »

adamredwoods wrote: post #298

Let's be specific, they don't know if he is fully cured. They need more time to conclude efficacy.

While true, so far so good - the baby is 9 1/2 months old now, whereas they had a life expectancy of a week earlier without intervention.

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

#407

Post by snalty »

vanderZwan wrote: post #381
cm2187 wrote: post #366

Earlier quoted context omitted.

That also sounds like a recipe for a terrifying virus

I would be surprised if viruses using U instead of T didn't already exist. After all, don't all viruses work by doing gene editing in vivo, except just localized to one cell? EDIT: well, I suppose the question is whether cells of living beings could produce the U required for the viruses. But if not, then a wild virus using U instead of T to bypass our immunity also would not be a threat for that very reason.

It’s not the use of Uracil/Urimidine that bypasses the immune system. RNA uses Uracil instead of thymine in all organisms afaik, and RNA viruses certainly exist. It’s pseudouridine that’s the magic stuff.

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

#408

Post by thijson »

Sammi wrote: post #115
thijson wrote: post #87

[flagged]

Delete your comment. You're minimising the death of children. Yeah I'm sensitive about this. I like many other parents of children who have been lost to genetic disease have good reason to be.

I think it's a tool that can be used for great good. But also can be used for eugenics. That's why I pasted the link. It's a slippery slope. What a parent will do for their kids knows no bound.

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

#409

Post by Jugurtha »

mike_hearn wrote: post #379
monkeycantype wrote: post #171

Earlier quoted context omitted.

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 somet…

What was the success of other means, such as sugars and proteins? Something like glycocalyx or polysaccharide capsules? Or HIV like deployment gp41/gp120?

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

#410

Post by Cthulhu_ »

Traubenfuchs wrote: post #295
thrance wrote: post #106

Earlier quoted context omitted.

Is the global gene pool actually degrading though? I only ever hear that in thinly veiled attempts at advocating for eugenicism. And it never comes substantiated by any research. Anyway, this baby proves we can fix hereditary diseases now.

> eugenicism That comes in many forms: Black/dark one, nazi style, where you outright sterilise or even kill those with unhealthy/bad genes. And white/peaceful one, where you‘d appeal to those with unhealthy/bad genes not to procreate and encourage those with healthy/good ones to do. You can‘t seriously tell me it‘s not extremely unethical for people with huntington‘s disease or cystic fibrosis to have children.

Most genetic diseases only occur when two parents happen to have it, but they won't necessarily be aware of it; would it be unethical for people who are unaware of their genetic defects to have children?

Second, according to a quick search, 10% of cases of Huntington's Disease are due to new mutations; I suspect (but I'm a HN commenter, no geneticist) this is the case for many other genetic conditions.

So the other ethics question to ask: should people be able to get DNA tests for genetic conditions (voluntary)? I'd say yes. Should people be mandated to get DNA tests and be forbidden to procreate if there's something in there? No, that's eugenics. Should people who know they have a genetic condition and there's a chance their child has it too have children? That'd be their choice. I don't think it's fair for people to intentionally place a burden on health care systems like that, but thing is, there's very, very few people that have children with that as the intent.

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