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Baby is healed with first personalized gene-editing treatment

(www.nytimes.com)
1162 points jbredeche | 3 comments | 15 May 25 18:06 UTC | HN request time: 0.662s | 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. dclowd9901 ◴[15 May 25 23:57 UTC] No.44000524[source]▶
I literally said the same thing out loud.

I had heard about CRISPR a while back but most reporting on it kind of hand waved over the mechanisms of how it actually accomplishes its work. What these researchers have figured out to make this work absolutely blows my mind.

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2. j16sdiz ◴[16 May 25 05:18 UTC] No.44002049[source]▶
AFAICT, CRISPR still make many bad edits. We relies on the fact that most of those bad edit won't survive.
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3. rubidium ◴[16 May 25 08:32 UTC] No.44003044[source]▶
It can make “bad edits” eg off target effects. But in this case there were, as far as is known, none. It’s aided that this was a single nucleotide defect.

They specifically tested for off target edits in the mouse study and found no harmful edits (and very rare off target ones). That plus the specific targeting of the liver cells (no germ line effect expected), makes this a low risk approach and certainly better than doing nothing.