←back to thread

Baby is healed with first personalized gene-editing treatment

(www.nytimes.com)
1168 points jbredeche | 6 comments | 15 May 25 18:06 UTC | HN request time: 0.262s | source | bottom
Show context
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.

replies(4): >>43998742 #>>43998906 #>>43999140 #>>43999563 #
1. 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.
replies(2): >>43999711 #>>43999719 #
2. 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

3. XorNot ◴[15 May 25 21:54 UTC] No.43999719[source]▶
No they were not. A vaccine triggers an immune response, not a functional change.

mRNA vaccines are highly localized: you get a sore arm because most of it only gets taken up by muscle cells around the injection site, which spend some time producing the antigen and triggering a primary immune response (the inflammation aka the sore arm).

replies(2): >>44000936 #>>44006415 #
4. scotty79 ◴[16 May 25 01:15 UTC] No.44000936[source]▶
Still it needs to enter the cells all the same.

As for being localized it's true however after vaccine dose S proteins have been detected also in remote locations in the body because you can't make something 100% localized.

If you had an infusion that doesn't trigger immune system you could just increase the dose significantly, put it in the blood and most likely it would have reached all cells that blood reaches.

replies(1): >>44001649 #
5. im3w1l ◴[16 May 25 03:43 UTC] No.44001649{3}[source]▶
Last I heard those gene editing things lead to so called of-target edits, so they were basically corrupting random dna. Now in this case the baby would have died without this treatment so clearly benefits outweight the risks. But even then they probably want to have the dose be as low as possible.

But I'm speculating a bit here.

6. xrhobo ◴[16 May 25 15:08 UTC] No.44006415[source]▶
What I find interesting about the covid mRNA vaccine is I remember being sick in March 2020 and I can't remember being sick since.

I can remember getting a sniffle at night and waking up fine the next morning a few times.

I think I had two doses of covid mRNA vaccine.

I have actually forgot what it is like to be sick. It almost feels like the covid vaccine gave me some kind of super immunity. I never get the flu shot either. I have not had the flu in 5 years for sure.