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Baby is healed with first personalized gene-editing treatment

(www.nytimes.com)
1167 points jbredeche | 6 comments | 15 May 25 18:06 UTC | HN request time: 0.69s | source | bottom
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MrZander ◴[15 May 25 19:30 UTC] No.43998447[source]▶
> 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 changed.

That is one of the most incredible things I have ever read.

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Balgair ◴[15 May 25 20:31 UTC] No.43999018[source]▶
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

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alecco ◴[15 May 25 20:53 UTC] No.43999243[source]▶
> [...] then the body's immune system doesn't nearly alarm as much, as it doesn't really see that mRNA as quite so dangerous

Please tell me there are measures to prevent this going into the wild. Please tell me this won't be used in large-scale industrial farming.

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imcritic ◴[15 May 25 21:52 UTC] No.43999695[source]▶
Farming? This will be used in warfare.
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1. Muromec ◴[15 May 25 22:55 UTC] No.44000169[source]▶
That would be less effective than bio and chemical weapons are. Which are not used because they just suck
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2. kulahan ◴[16 May 25 00:31 UTC] No.44000716[source]▶
I’m not sure of by “they just suck” you meant to imply that they’re ineffective. If that’s the case, I strongly disagree. They are not used because somehow all countries pretty much agreed they’re way TOO effective and horrific. Nobody wants it used on them, so nobody uses it on anyone else.

I cannot imagine a more effective weapon than an invisible gas that melts you alive, and there are MANY chemical and bio examples of these types of weapons.

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3. beeflet ◴[16 May 25 01:12 UTC] No.44000918[source]▶
The ceiling for the destruction caused by biological weapons is far greater than chemical weapons. There is no chemical weapon that can hijack the victim to make more of it.
4. wffurr ◴[16 May 25 02:56 UTC] No.44001437[source]▶
>> They are not used because somehow all countries pretty much agreed they’re way TOO effective and horrific

That’s the story but it doesn’t hold up. Chemical weapons were used as recently as the Syrian civil war. I also think if they were really effective in modern warfare, Russia would have long ago deployed them in Ukraine.

More here: https://acoup.blog/2020/03/20/collections-why-dont-we-use-ch...

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5. kulahan ◴[16 May 25 19:35 UTC] No.44009038{3}[source]▶
What do you mean “if they were really effective”? We still hand out CBRN gear and train in how to put necessary parts on in seconds, because that’s often how little time you get before you’re permanently incapacitated. Mustard gas alone should prove this, and that’s an OLD chemical weapon.

Nowadays we have riot control agents that can be tailored to demographics, react more violently in the presence of sweat, or contain psychoactive ingredients. Nanoparticle dispersion bypasses common gas masks and clothing protection. Even if you’re completely geared up, they can be engineered to last on surfaces for a long time, or react only in the presence of certain triggers. Imagine thinking you’re safe until someone turns on a certain light bulb and you cook inside your protective gear because you were actually exposed 12 hours earlier in an undetectable manner.

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6. wffurr ◴[17 May 25 00:06 UTC] No.44010908{4}[source]▶
I'd encourage you to read the article. Chemical weapons are effectively useless against a well-trained "modern system" army. Part of that is the chemical warfare equipment and vehicles, but mostly it's cover-and-concealment. If you can actually find the enemy, it's much faster and simpler to use the other vastly destructive munitions that modern militaries have.