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trollbridgeyesterday at 9:52 PM4 repliesview on HN

I’m very puzzled why this wasn’t replicated in eg a study in rats first (where you duplicate the mutation, and then attempt to cure it).


Replies

austin-schickyesterday at 9:55 PM

The article says that they did do this:

> Qiu’s team had engineered mice to have a human version of the CHD3 gene with their daughter’s mutation, R1025W, which results in a protein with the amino acid tryptophan where there should be an arginine. The mutant pups developed autismlike traits and didn’t squeak as much as normal mice when separated from their mothers. When the researchers repaired that mutation, the pups developed normally.

fabian2kyesterday at 9:56 PM

They did animal experiments first. But according to the article they didn't really look at the serious side effects observed in monkeys, or ignored them. But animal experiments can only provide warning signs here, they can't tell you what will really happen in a human.