6 Deaf Children Can Now Hear After a Single Injection | Hacker News Reader

6 Deaf Children Can Now Hear After a Single Injection

wired.com

55 pointsBrajeshwar11 comments

Recent and related:

Gene therapy allows an 11-year-old boy to hear - https://news.ycombinator.com/item?id=39106464 - Jan 2024 (238 comments)

That's quite an injection if it can cure six children at once.

It must have been a pretty terrifying needle.

That's nothing, one small kid with one remaining cowpox lesion was the source of vaccination material for Spanish America.

https://en.wikipedia.org/wiki/Balmis_Expedition

(They took off from Europe with 22 kids and did an "infectious relay" in pairs. By the time they got to South American mainland, precisely 21 kids already resolved their cowpox. One last kid was still barely infectious, thus becoming the sole source of the vaccination material.)

Our science fiction theorization is starting to be realized

How would it feel for someone who was born deaf to suddenly start hearing?

Do they start getting irritated by everyday sounds? I’m really curious ..

They absolutely love it. One of the kids who got this said he felt there are "no bad sounds."

Six(6) Deaf Children Can Now... (I'd guess that HN auto-truncated the title)

It's experimental gene therapy, with very small n, and only looks applicable for certain types of inherited deafness.

"Small n" is not always a problem. They are not trying to tease out a small effect size between two treatments. This is a strong signal -- literally children can now hear when they couldn't before. A small sample size does not allow statistical noise to produce that effect.

Yeah the FDA is also being pretty lenient about allowing single-arm trials for gene therapy for this reason. The effect size needs to be obvious, but there's no reason to subject 3/6 deaf children to being a control if the risk is relatively low and the odds of success are high.

(also, for many of theses diseases, there's no plan B -- if it's not fixed in early childhood, you can never interpret sound).

All gene therapy is only applicable to very specific types of disorders.

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