Baby is healed with first personalized gene-editing treatment

(www.nytimes.com)

1163 points jbredeche | 4 comments | 15 May 25 18:06 UTC | HN request time: 0.842s | 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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1. DrScientist ◴[16 May 25 09:45 UTC] No.44003457[source]▶

>>43998447 #

Bear in mind that they intentionally choose something that was soluble - ie the easiest thing possible. So it's doesn't mean everything is now solvable.

For example it's no coincidence this is a liver disease as basically almost everything you inject in the bloodstream ends up concentrating in the liver by default - if you needed to target another organ with your LNP it would be much harder. Most of the time people are trying to stop stuff accumulating in the liver!

The liver has other special properties that are helpful as well.

Having said all that - it is still a massive achievement.

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

Biology is incredible - and you can do incredible things if you leverage it.

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2. abcd_f ◴[16 May 25 11:45 UTC] No.44004211[source]▶

>>44003457 (TP) #

> that was soluble

solvable

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3. DrScientist ◴[16 May 25 13:46 UTC] No.44005489[source]▶

>>44004211 #