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Baby is healed with first personalized gene-editing treatment

(www.nytimes.com)
1168 points jbredeche | 2 comments | 15 May 25 18:06 UTC | HN request time: 0.001s | 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. verisimi ◴[16 May 25 06:02 UTC] No.44002243[source]▶
Once the gene has been edited, things will work. But at some point that cell will die. Why would the replacement cell also have the edit? The DNA in the rest of the body's cells will still not be correct.
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2. riffraff ◴[16 May 25 06:25 UTC] No.44002346[source]▶
When cells duplicate they have the same (altered) DNA so the mutated cells survive.

You'll end up with mosaicism (cells with different DNA) but presumably you have enough of the new cells to fix the problem the original ones had.

You don't need to fix all the body, you just need to fix some of the, say, liver, and you're good.