{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,29]],"date-time":"2026-09-29T08:45:51Z","timestamp":1790671551972,"version":"4.1.0"},"reference-count":0,"publisher":"Global Cardiology Science and Practice","issue":"1","license":[{"start":{"date-parts":[[2023,2,6]],"date-time":"2023-02-06T00:00:00Z","timestamp":1675641600000},"content-version":"unspecified","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["gcsp"],"abstract":"<jats:p>Study and results: The first-in-human in vivo CRISPR\/Cas9 trial of TTR Gene editing by NTLA-2001 in patients with Transthyretin Amyloidosis and cardiomyopathy\u00a0was\u00a0designed to evaluate the safety, tolerability, efficacy, and pharmacokinetic and pharmacodynamic responses to IV NTLA-2001 administration and its effect on serum transthyretin (TTR) levels in patients with ATTR amyloidosis and cardiomyopathy.\u00a0\u00a0Twelve subjects received NTLA-2001 (three NYHA I\/II subjects at 0.7 mg\/kg, three subjects at 1.0 mg\/kg, and six NYHA III subjects at 0.7 mg\/kg). Serum TTR levels were reduced from the baseline in all subjects (mean&gt;90% after 28 days). Mean % reductions (+\/-SEM) from baseline to day 28 were: NYHA I\/II at 0.7 mg\/kg = 92% (1%), at 1.0 mg\/kg = 92% (2%), and for NYHA III at 0.7 mg\/kg = 94% (1%) maintained through 4-6 months. Two of the 12 patients (16.7 %) reported a transient infusion reaction. One patient experienced a grade 3 infusion-related reaction that resolved without any clinical sequelae.\u00a0\u00a0<\/jats:p>","DOI":"10.21542\/gcsp.2023.4","type":"journal-article","created":{"date-parts":[[2023,2,6]],"date-time":"2023-02-06T12:24:46Z","timestamp":1675686286000},"source":"Crossref","is-referenced-by-count":16,"title":["Lessons from the first-in-human in vivo CRISPR\/Cas9 editing of the TTR gene by NTLA-2001 trial in patients with transthyretin amyloidosis with cardiomyopathy"],"prefix":"10.21542","volume":"2023","author":[{"given":"Susy","family":"Kotit","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"8990","published-online":{"date-parts":[[2023,2,6]]},"container-title":["Global Cardiology Science and Practice"],"original-title":[],"link":[{"URL":"https:\/\/globalcardiologyscienceandpractice.com\/index.php\/gcsp\/article\/download\/597\/525","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/globalcardiologyscienceandpractice.com\/index.php\/gcsp\/article\/download\/597\/525","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2023,9,14]],"date-time":"2023-09-14T12:46:36Z","timestamp":1694695596000},"score":1,"resource":{"primary":{"URL":"https:\/\/globalcardiologyscienceandpractice.com\/index.php\/gcsp\/article\/view\/597"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2023,2,6]]},"references-count":0,"journal-issue":{"issue":"1","published-online":{"date-parts":[[2023,2,6]]}},"URL":"https:\/\/doi.org\/10.21542\/gcsp.2023.4","relation":{},"ISSN":["2305-7823"],"issn-type":[{"value":"2305-7823","type":"electronic"}],"subject":[],"published":{"date-parts":[[2023,2,6]]}}}