dclowd9901 23 hours ago

I literally said the same thing out loud.

I had heard about CRISPR a while back but most reporting on it kind of hand waved over the mechanisms of how it actually accomplishes its work. What these researchers have figured out to make this work absolutely blows my mind.

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j16sdiz 17 hours ago

AFAICT, CRISPR still make many bad edits. We relies on the fact that most of those bad edit won't survive.

rubidium 14 hours ago

It can make “bad edits” eg off target effects. But in this case there were, as far as is known, none. It’s aided that this was a single nucleotide defect.

They specifically tested for off target edits in the mouse study and found no harmful edits (and very rare off target ones). That plus the specific targeting of the liver cells (no germ line effect expected), makes this a low risk approach and certainly better than doing nothing.