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 par

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/