CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis

D. Gillmore, Julian; Gane, Ed; Täubel, Jörg; Kao, Justin; Fontana, Marianna; L. Maitland, Michael; Seitzer, Jessica; J. O’Connell, Daniel; Walsh, Kathryn; Wood, Kristy; A. Phillips, Jonathan; Xu, Yuanxin; Amaral, Adam; P. Boyd, Adam; Cehelsky, Jeffrey; D. McKee, Mark; Schiermeier, Andrew; Harari, Olivier; Murphy, Andrew; A. Kyratsous, Christos; Zambrowicz, Brian; Soltys, Randy; E. Gutstein, David; P. Leonard, John; Sepp‐Lorenzino, Laura; Lebwohl, David

doi:10.1056/nejmoa2107454

Public

CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis

Published in New England Journal of Medicine • Jun 26, 2021

Authors:

Julian D. Gillmore

Ed Gane

Jörg Täubel

Abstract

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...

Subject

Transthyretin

CRISPR

Genome editing

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