On: CRISPR gene-editing breakthrough opens door to treating broad array of diseases

[iandanforth]

Please do familial hypercholesterolemia soon! I know it would be harder but getting started on targeting specific mutations could be fruitful.

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3681164/

[DrAwdeOccarim → iandanforth]

It's coming very, very soon. Verve just IPO'ed and you check out their S1 to see their data on using the exact same idea (N1mΨ mRNA + gRNA inside LNP) to treat hypercholesterolemia! The data look amazing in non-human primates, which based on the Intellia data, suggest 10-fold lower dose in humans should work just fine.

https://www.biospace.com/article/releases/verve-therapeutics...

[iandanforth → DrAwdeOccarim]

That's amazing! How does one register as a potential patient for the trials?

[DrAwdeOccarim → iandanforth]

www.clinicaltrials.gov once their IND is open, you'll be able to find it there! My guess is it's a year or so away before they start recruiting.

[bigmattystyles → iandanforth]

I want the CRISPR research that focuses on CIP accelerated. I have a minor chronic pain issue that will get worse over time (think sciatica). By the time I’m 60 in 25 years, it’d be great if we had that. No more need for opiates or drugs that barely make a dent. https://en.m.wikipedia.org/wiki/Congenital_insensitivity_to_...