>

This is 2 for 2 as of recently. LFG
I have a hereditary condition. I wonder if this sort of treatment can be modified to cure it >


They have a way to deliver to liver siRNA to modify gene expression.

Deliver?  More like reliver! >

I'll allow it. Overruled. >

That's a relief >

Thanks. I wonder how long it will take to have something along the lines of a CRISPR gene editing cure for some disease. >

Modifying only one particular target and no others is really hard. Then the additional challenge of getting it into humans and where it is needed. >

I'm just kinda surprised it hasn't been done already for something like expression of a missing clotting factor in the blood. >

Getting RNA into the body has been a real challenge. Delivery is always the technical problem.
How they actually get their stuff in via liver is their big thing.