“Just”.

Most shit doesn’t even make it to clinical trials, much less show such striking efficacy so quickly.

This is exactly where this sort of thing needs to start. Super rare stuff we don’t have a real cure for that makes lives miserable and can be fatal. And so far, the results are incredible.

If this was a normal pharmacological therapy, I wouldn’t be super excited about stage 1 trials showing striking results, because it would actually be limited to that one therapy. But it isn’t. It’s “magic bullet” therapy that we should be able to modify for nearly any problem related to bad genes. If it works as effectively in further trials, it could very well change medical treatment as we know it for a whole range of stuff.