CRISPR-Cas9 In Vivo Gene Editing of KLKB1 for Hereditary Angioedema.
Hilary J LonghurstKaren LindsayRemy S PetersenLauré M FijenPadmalal GurugamaDavid MaagJames S ButlerMrinal Y ShahAdele GoldenYuanxin XuCarri BoiselleJoseph D VogelAhmed M AbdelhadyMichael L MaitlandMark D McKeeJessica SeitzerBo W HanSamantha SoukamneuthJohn LeonardLaura Sepp-LorenzinoEliana D ClarkDavid LebwohlDanny M CohnPublished in: The New England journal of medicine (2024)
In this small study, a single dose of NTLA-2002 led to robust, dose-dependent, and durable reductions in total plasma kallikrein levels, and no severe adverse events were observed. In exploratory analyses, reductions in the number of angioedema attacks per month were observed at all dose levels. (Funded by Intellia Therapeutics; ClinicalTrials.gov number, NCT05120830.).