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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 >

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

It’s gonna be bigger. It’ll usher in a complete change in treatment of disease and maybe aging too. Think of a personalized drug just for your body/disease interaction. Imagine a drug that monitors and adjusts itself on the bio-molecular level to relieve symptoms, repair damaged tissue and quantify ts efficacy with a simple blood test. All of these building blocks are within reach. We need massive computing power, engineers and scientists specializing in molecular biology and proteomics, and the list goes on. This technology will change life on earth, the way the industrial age and the digital/electronic age did. Imagine an inoculation for the precise type of cancer that only you have. And you’re cured for the rest of your life. We have the technology. It’s like we’re building a sand castle, but we can only do it one grain of sand at a time. This is where we are, but we’re moving incredibly fast. Source: Medical Technolgy Design Control Engineer here. >

My daughter is in middle school but this is her goal. She is talented enough that I think it is realistic for her to achieve. She wants to go into genetics. >

Maybe give her the book “Code Breaker” by Walter Isaacson about Jennifer Doudna, if she doesn’t already have it. Bet that’ll light a fire. Your daughter is exactly who we need. Look at Harvard and Stanford - Ph.D. center of the universe right now.