>

This is a game changer level of technology. To me this is as big as the polio vaccination.
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. >

This is 2 for 2 as of recently. LFG >

I feel like we always read these groundbreaking stories but never see the light of day for availability for the masses. >

They’re available, but right now generally limited to very rare diseases as those tend to be the easiest to fix (usually just single edit). They are also prohibitively expensive to research and manufacture, and thus purchase. Some gene therapies are over a million dollars per dose. It’s happening, but slowly. >

Also those million dollar drugs can be one time treatment only with a lifetime of benefit. 
The level of benefit over the course of lifetime is over a million dollars worth. Justifying the price. Yes, insurers and/or government will pay it. Much better to have someone cured than suffering for a lifetime. >

A human life is worth 10,000 to a few million so I'm not sure about that, from an strictly economical purpose >

Can we do one that gives you “a new face”? Cause I could really usr that