3 ms·
Please do familial hypercholesterolemia soon! I know it would be harder but getting started on targeting specific mutations could be fruitful. https://www.ncbi
by iandanforth 5y ago
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/ https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3681164/
- bigmattystyles 5y agoI 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_pain https://en.m.wikipedia.org/wiki/Congenital_insensitivity_to_...
- DrAwdeOccarim 5y agoIt'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-to-present-preclinical-data-from-gene-editing-programs-for-the-treatment-of-atherosclerotic-cardiovascular-disease-at-asgct/ https://www.biospace.com/article/releases/verve-therapeutics...
- iandanforth 5y agoThat's amazing! How does one register as a potential patient for the trials?
- DrAwdeOccarim 5y agowww.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.