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CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis.
N Engl J Med
August 2021
From the National Amyloidosis Centre, Division of Medicine, University College London, Royal Free Hospital (J.D.G., M.F.) and Richmond Pharmacology, St. George's University of London (J.T.) - both in London; New Zealand Clinical Research (E.G.), University of Auckland (E.G.), and the Department of Neurology, Auckland City Hospital (J.K.) - all in Auckland, New Zealand; Intellia Therapeutics, Cambridge, MA (M.L.M., J.S., D.O., K.R.W., K.W., J.P., Y.X., A.A., A.P.B., J.E.C., M.D.M., A.S., J.L., L.S.-L., D.L.); and Regeneron Pharmaceuticals, Tarrytown, NY (O.H., A.M., C.A.K., B.Z., R.S., D.E.G.).
Background: Transthyretin amyloidosis, also called ATTR amyloidosis, is a life-threatening disease characterized by progressive accumulation of misfolded transthyretin (TTR) protein in tissues, predominantly the nerves and heart. NTLA-2001 is an in vivo gene-editing therapeutic agent that is designed to treat ATTR amyloidosis by reducing the concentration of TTR in serum. It is based on the clustered regularly interspaced short palindromic repeats and associated Cas9 endonuclease (CRISPR-Cas9) system and comprises a lipid nanoparticle encapsulating messenger RNA for Cas9 protein and a single guide RNA targeting .
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