CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis.
Julian D GillmoreEd GaneJorg TaubelJustin KaoMarianna FontanaMichael L MaitlandJessica SeitzerDaniel O'ConnellKathryn R WalshKristy WoodJonathan A PhillipsYuanxin XuAdam AmaralAdam P BoydJeffrey E CehelskyMark D McKeeAndrew SchiermeierOlivier HarariAndrew MurphyChristos A KyratsousBrian ZambrowiczRandy SoltysDavid E GutsteinJohn LeonardLaura Sepp-LorenzinoDavid LebwohlPublished in: The New England journal of medicine (2021)
In a small group of patients with hereditary ATTR amyloidosis with polyneuropathy, administration of NTLA-2001 was associated with only mild adverse events and led to decreases in serum TTR protein concentrations through targeted knockout of TTR. (Funded by Intellia Therapeutics and Regeneron Pharmaceuticals; ClinicalTrials.gov number, NCT04601051.).