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A comment on First UK child to receive gene therapy for fatal genetic disorder is now healthy. Parent comment

Re:First UK child to receive gene therapy for fatal genetic ... by piyh on Friday February 17, 2023 @05:48PM (#34841691)
Most recipients don't need to take immunosuppressants at all if they get PBSC or bone marrow transplants. Even if they do, it's short term. Additionally, the allo grafts need to be matched, which if you're non white is not a good success rate. 85%ish of whites get matched, that number gets depressingly low for minorites. On the US registry, only 1 in 400 donors get called. I happen to be one of those donors and a system where I'm not needed is a better one. The best part is no part.

https://ashpublications.org/blood/article/104/12/3501/89040/...

>Previous studies have shown that 30% to 70% of all patients surviving beyond 100 days after HCT require treatment for chronic GHVD,2,4-6 often for more than 2 years.

Re: a better system is where you don't need donors:

https://www.cbsnews.com/newyork/news/l-i-woman-dies-after-ma...

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Re:First UK child to receive gene therapy for fatal genetic ... by meese712 on Friday February 17, 2023 @05:55PM (#34841749)
I was getting at the article giving the impression this somehow cured the disease vs a BMT just slowing progression. I know the whole transplant thing sucks. I've had two transplants, thank you for being on the list :).
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Re:First UK child to receive gene therapy for fatal genetic ... by tialaramex on Friday February 17, 2023 @08:00PM (#34842739)
The article indicates they don't know. "it is not yet clear whether it will persist life-long, and extended follow-up is needed" - So they can be optimistic without knowing for sure because they don't have a bunch of kids they treated say ten years ago to check on. It seems like this might stick, they hope it will, they can't know yet.

One of my friends gave his wife a kidney which allowed her to get off dialysis, so yeah, thanks to anybody who is willing to do this for someone they don't even know.

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Re:First UK child to receive gene therapy for fatal genetic ... by jimbob45 on Friday February 17, 2023 @07:31PM (#34842563)
What’s the story with minorities? Just a smaller pool of donors or something?
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Re:First UK child to receive gene therapy for fatal genetic ... by jonfromsf on Friday February 17, 2023 @08:27PM (#34842894)
Yes, a smaller pool of donors. Worst still for finding matches is multi-racial people.
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Re:First UK child to receive gene therapy for fatal genetic ... by concordDance on Friday February 17, 2023 @07:42PM (#34842645)
> 85%ish of whites get matched, that number gets depressingly low for minorites.

Hmm...

How does this translate to other places? E.g. can you only match Han in China?

Do Italians and Scots match? Turks and Egyptians?

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Re:First UK child to receive gene therapy for fatal genetic ... by mlyle on Friday February 17, 2023 @08:37PM (#34842958)
A big part of the problem is that minorities are not reached/enrolled onto the registry at a high rate.

What matters isn't really the proportion of the subpopulations that are registered, but the absolute numbers.

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Re:First UK child to receive gene therapy for fatal genetic ... by inciampati on Saturday February 18, 2023 @10:50AM (#34847750)
Matching involves the now simple (if costly) task of comparing diplotypes at immune system loci involved in self recognition, like the MHC.

What's horrific to me is that matching by race is still a thing! These genes are under diversifying selection. That means that they often do not match the surrounding genetic background. That means that racially driven matching is bonkers.

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