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Baby is healed with first personalized gene-editing treatment

(www.nytimes.com)
1165 points jbredeche | 1 comments | 15 May 25 18:06 UTC | HN request time: 0.221s | 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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scotty79 ◴[15 May 25 20:01 UTC] No.43998742[source]▶
I don't think it's gonna be that hard. All cells that blood reaches were happily taking mRNA vaccine.
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1. derektank ◴[15 May 25 21:53 UTC] No.43999711[source]▶
I hate to break it to you, but it will be substantially more difficult to target other organ systems. The liver is uniquely easy to target with our current vectors.

Right off the bat, the liver receives roughly a quarter of all cardiac output, either directly or second hand from the digestive organs. Additionally, the liver has a fenestrated endothelium which, while not completely unique in the body, uniquely allows molecules like lipid nanoparticles (LNPs) to access liver cells. Finally, the liver is the site of most lipoprotein processing, and LNPs can be designed to take advantage of the existing pathways to get the gene editing mRNA into the hepatocytes. All this is to say that if you have a genetic condition that primarily effects the liver, there's a lot more hope for treatment in the near term than for others.

Good lecture on the difficulties of finding appropriate platforms for delivering gene therapies to cells for anyone interested [1]

[1] https://youtu.be/6URTjoK58Yc