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Baby is healed with first personalized gene-editing treatment

(www.nytimes.com)
1168 points jbredeche | 1 comments | 15 May 25 18:06 UTC | HN request time: 0s | 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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1. Thebroser ◴[16 May 25 00:20 UTC] No.44000649[source]▶
Add gene has a great guide as to what goes on at the molecular level: https://www.addgene.org/guides/crispr/

Essentially you can design an rna molecular that contains a 20 nucleotide long sequence that can target your region of interest, with the caveat that there is a standard recognition sequence proximal to your sequence of interest (PAM sequence)