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CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis

26 Jun 2021

Abstract excerpt

BACKGROUND: . METHODS: After conducting preclinical in vitro and in vivo studies, we evaluated the safety and pharmacodynamic effects of single escalating doses of NTLA-2001 in six patients with hereditary ATTR amyloidosis with polyneuropathy, three in each of the two initial dose groups (0.1 mg per kilogram and 0.3 mg per kilogram), within an ongoing phase 1 clinical study. RESULTS: after a single dose. Serial...

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PMID: 34215024
DOI: 10.1056/nejmoa2107454

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