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Baby is healed with first personalized gene-editing treatment

(www.nytimes.com)
1167 points jbredeche | 5 comments | 15 May 25 18:06 UTC | HN request time: 0.4s | source
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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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1. maxerickson ◴[16 May 25 00:06 UTC] No.44000573[source]▶
Is this a troll? Pseudouridine mRNA isn't gene editing.
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2. VierScar ◴[16 May 25 00:53 UTC] No.44000815[source]▶
What do you mean? Is mRNA not used to produce the enzyme that these comments mentioned? I don't think they were saying mRNA is gene editing itself. Just commenting on a modified mRNA helping the process compared to normal mRNA. Might be misunderstanding though so correct me if I am
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3. maxerickson ◴[16 May 25 01:08 UTC] No.44000895[source]▶
I dunno, I think they are being sloppy and conflating things. We can induce manufacture of proteins and can design proteins that carry out gene editing, so we can stack that knowledge together to induce cells to manufacture proteins that carry out gene edits, but it's the payload that is the gene editing, not the instruction to make the protein.

Given the merry movement to call the COVID vaccines gene editing, it rankles.

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4. Balgair ◴[16 May 25 02:05 UTC] No.44001210{3}[source]▶
Hey, yeah, I'm not the most up to date on the current methods. Most of my knowhow is a bit out of date here. So thanks for piping up to correct things.

Do you know of any good resources that I can use to get up to speed on the exact methods they used for the baby?

My understanding, outdated as it is, is that we're using the mRNA to go in and create CRISPR-CAS9 slicers/dicers and additionally to that, the correct genes (not mRNA) to get stitched in. I would love to know more about how I am wrong here, as I am sure I'm not even close to really understanding it.

Thanks!

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5. ionwake ◴[16 May 25 10:11 UTC] No.44003604{4}[source]▶
I think you're replying to someone edgelord about covid who got confused about some mrna statement and then back pedalled re-affirming what the article was about.