3 ms·

You guys are freaking out about apples, but I'm excited for the application of (the concept of) silencing towards humans. I don't think* it quite works this way

You guys are freaking out about apples, but I'm excited for the application of (the concept of) silencing towards humans. I don't think* it quite works this way & I'm sure biologics are similar, but it would be amazing if you could do the same thing for humans. Imagine doing that for people who have psoriasis. Would be amazing if the body took to that & could continue producing cells that react properly.

jfarlow 9y ago
It is happening. Hemophilia being cured at Marin Bio [1]. There are a bunch of therapies where Cas9 is being used by to therapeutically knock out or repair particular proteins. Indications currently targeted at Intellia: ATTR, Hepatitis B, AATD, PH-1, etc. [2]. [1] http://investors.biomarin.com/2017-07-11-BioMarins-Investigational-Gene-Therapy-for-Hemophilia-A-at-6e13-vg-kg-Dose-Maintains-Average-Factor-VIII-Levels-within-Normal-Range-for-over-One-Year http://investors.biomarin.com/2017-07-11-BioMarins-Investiga... [2] http://www.intelliatx.com/pipeline/ http://www.intelliatx.com/pipeline/