Landmark results from the first in vivo CRISPR gene-editing therapy have been unveiled by Intellia Therapeutics and Regeneron, and could put them at the forefront of a medical revolution.
Six patients living with hereditary transthyretin amyloidosis with polyneuropathy (hATTRv-PN) were given in the CRISPR/Cas9-based therapy candidate NTLA-2001 which ‘edited...