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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: 0.21s | source
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vessenes ◴[15 May 25 19:53 UTC] No.43998661[source]▶
NYT isn’t super specific here, but they made it sound like the disease treated is liver related. My understanding is that the liver is a good place to start with CRISPR-type gene treatments, in that the liver normally deals with anomalous shit in your bloodstream, say, like CRISPR type edits. So anywhere outside the liver is going to be significantly harder to get really broad uptake of gene edits.

It’s crazy encouraging that this worked out for this kid, and I’m somewhat shocked this treatment was approved in the US - I don’t think of us as very aggressive in areas like this. But to me, really hopeful and interesting.

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1. cdcox ◴[15 May 25 21:37 UTC] No.43999563[source]▶
You are right, current CRISPR systems tends to accumulate in the liver. Most CRISPR companies have shifted their focus to the liver over time because it's easiest to deliver there. Most viruses people use to target other organs are not large enough to carry CRISPR and lipid nanoparticles with CRISPR seem to like ending up in the liver and are hard to deliver at dose to hit other organ systems. It has been one of the big struggles of CRISPR companies. That being said, this is a huge deal and very encouraging.

As to the FDA stance, it tends to be more willing to go ahead with compassionate uses like this when it's clearly life or death.[1]

[1] https://www.statnews.com/2025/05/15/crispr-gene-editing-land... This discuss a little of the FDA stuff but not much more detail, it sounds like they did let them skip some testing.