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Baby is healed with first personalized gene-editing treatment

(www.nytimes.com)
1165 points jbredeche | 1 comments | 15 May 25 18:06 UTC | HN request time: 0.383s | 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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cryptoegorophy ◴[16 May 25 00:01 UTC] No.44000545[source]▶
How does it know how to gps around? From what I know everything down there is a chemical reaction with some minimal physical motion, but how do you program it to know where to change and what and how.
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TheJoeMan ◴[16 May 25 00:04 UTC] No.44000557[source]▶
It’s more like a “ctrl+F” for DNA. Hopefully there’s only 1 match (the target site).
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0x1ceb00da ◴[16 May 25 01:15 UTC] No.44000942[source]▶
So you create a molecule that binds to a certain location in the dna, and then deploy a billion of them?
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1. rubidium ◴[16 May 25 08:38 UTC] No.44003082[source]▶
More or less, yes.

An interesting part of the study was determining what a clinical dose _should_ be. You need enough to edit enough liver cells. But don’t really want to completely overdo it to limit potentially negative side effects. Seems like they got it right enough here, with the first dose having some effect and the subsequent dose having more.