CRISPR-Cas9 Gene Editing with Nexiguran Ziclumeran for ATTR Cardiomyopathy.

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Bibliographic Details
Title: CRISPR-Cas9 Gene Editing with Nexiguran Ziclumeran for ATTR Cardiomyopathy.
Authors: Fontana M; From the National Amyloidosis Centre, University College London, Royal Free Hospital (M.F., Y.R., J.D.G.), and Richmond Pharmacology (J.T.) - both in London; Brigham and Women's Hospital, Boston (S.D.S.), and Intellia Therapeutics, Cambridge (J.K., L.W., R.R., D.L., D.S., J.O., A.H., P.Z., Y.X., A.L., A.S.) - both in Massachusetts; the University of Auckland, Auckland, New Zealand (E.J.G.); Umea University, Umea, Sweden (B.P.); Centre Hospitalo-Universitaire de Bicêtre, Assistance Publique-Hôpitaux de Paris, University Paris-Saclay, Le Kremlin-Bicêtre, France (D.A.); and Regeneron Pharmaceuticals, Tarrytown, NY (D.E.G.)., Solomon SD; From the National Amyloidosis Centre, University College London, Royal Free Hospital (M.F., Y.R., J.D.G.), and Richmond Pharmacology (J.T.) - both in London; Brigham and Women's Hospital, Boston (S.D.S.), and Intellia Therapeutics, Cambridge (J.K., L.W., R.R., D.L., D.S., J.O., A.H., P.Z., Y.X., A.L., A.S.) - both in Massachusetts; the University of Auckland, Auckland, New Zealand (E.J.G.); Umea University, Umea, Sweden (B.P.); Centre Hospitalo-Universitaire de Bicêtre, Assistance Publique-Hôpitaux de Paris, University Paris-Saclay, Le Kremlin-Bicêtre, France (D.A.); and Regeneron Pharmaceuticals, Tarrytown, NY (D.E.G.)., Kachadourian J; From the National Amyloidosis Centre, University College London, Royal Free Hospital (M.F., Y.R., J.D.G.), and Richmond Pharmacology (J.T.) - both in London; Brigham and Women's Hospital, Boston (S.D.S.), and Intellia Therapeutics, Cambridge (J.K., L.W., R.R., D.L., D.S., J.O., A.H., P.Z., Y.X., A.L., A.S.) - both in Massachusetts; the University of Auckland, Auckland, New Zealand (E.J.G.); Umea University, Umea, Sweden (B.P.); Centre Hospitalo-Universitaire de Bicêtre, Assistance Publique-Hôpitaux de Paris, University Paris-Saclay, Le Kremlin-Bicêtre, France (D.A.); and Regeneron Pharmaceuticals, Tarrytown, NY (D.E.G.)., Walsh L; From the National Amyloidosis Centre, University College London, Royal Free Hospital (M.F., Y.R., J.D.G.), and Richmond Pharmacology (J.T.) - both in London; Brigham and Women's Hospital, Boston (S.D.S.), and Intellia Therapeutics, Cambridge (J.K., L.W., R.R., D.L., D.S., J.O., A.H., P.Z., Y.X., A.L., A.S.) - both in Massachusetts; the University of Auckland, Auckland, New Zealand (E.J.G.); Umea University, Umea, Sweden (B.P.); Centre Hospitalo-Universitaire de Bicêtre, Assistance Publique-Hôpitaux de Paris, University Paris-Saclay, Le Kremlin-Bicêtre, France (D.A.); and Regeneron Pharmaceuticals, Tarrytown, NY (D.E.G.)., Rocha R; From the National Amyloidosis Centre, University College London, Royal Free Hospital (M.F., Y.R., J.D.G.), and Richmond Pharmacology (J.T.) - both in London; Brigham and Women's Hospital, Boston (S.D.S.), and Intellia Therapeutics, Cambridge (J.K., L.W., R.R., D.L., D.S., J.O., A.H., P.Z., Y.X., A.L., A.S.) - both in Massachusetts; the University of Auckland, Auckland, New Zealand (E.J.G.); Umea University, Umea, Sweden (B.P.); Centre Hospitalo-Universitaire de Bicêtre, Assistance Publique-Hôpitaux de Paris, University Paris-Saclay, Le Kremlin-Bicêtre, France (D.A.); and Regeneron Pharmaceuticals, Tarrytown, NY (D.E.G.)., Lebwohl D; From the National Amyloidosis Centre, University College London, Royal Free Hospital (M.F., Y.R., J.D.G.), and Richmond Pharmacology (J.T.) - both in London; Brigham and Women's Hospital, Boston (S.D.S.), and Intellia Therapeutics, Cambridge (J.K., L.W., R.R., D.L., D.S., J.O., A.H., P.Z., Y.X., A.L., A.S.) - both in Massachusetts; the University of Auckland, Auckland, New Zealand (E.J.G.); Umea University, Umea, Sweden (B.P.); Centre Hospitalo-Universitaire de Bicêtre, Assistance Publique-Hôpitaux de Paris, University Paris-Saclay, Le Kremlin-Bicêtre, France (D.A.); and Regeneron Pharmaceuticals, Tarrytown, NY (D.E.G.)., Smith D; From the National Amyloidosis Centre, University College London, Royal Free Hospital (M.F., Y.R., J.D.G.), and Richmond Pharmacology (J.T.) - both in London; Brigham and Women's Hospital, Boston (S.D.S.), and Intellia Therapeutics, Cambridge (J.K., L.W., R.R., D.L., D.S., J.O., A.H., P.Z., Y.X., A.L., A.S.) - both in Massachusetts; the University of Auckland, Auckland, New Zealand (E.J.G.); Umea University, Umea, Sweden (B.P.); Centre Hospitalo-Universitaire de Bicêtre, Assistance Publique-Hôpitaux de Paris, University Paris-Saclay, Le Kremlin-Bicêtre, France (D.A.); and Regeneron Pharmaceuticals, Tarrytown, NY (D.E.G.)., Täubel J; From the National Amyloidosis Centre, University College London, Royal Free Hospital (M.F., Y.R., J.D.G.), and Richmond Pharmacology (J.T.) - both in London; Brigham and Women's Hospital, Boston (S.D.S.), and Intellia Therapeutics, Cambridge (J.K., L.W., R.R., D.L., D.S., J.O., A.H., P.Z., Y.X., A.L., A.S.) - both in Massachusetts; the University of Auckland, Auckland, New Zealand (E.J.G.); Umea University, Umea, Sweden (B.P.); Centre Hospitalo-Universitaire de Bicêtre, Assistance Publique-Hôpitaux de Paris, University Paris-Saclay, Le Kremlin-Bicêtre, France (D.A.); and Regeneron Pharmaceuticals, Tarrytown, NY (D.E.G.)., Gane EJ; From the National Amyloidosis Centre, University College London, Royal Free Hospital (M.F., Y.R., J.D.G.), and Richmond Pharmacology (J.T.) - both in London; Brigham and Women's Hospital, Boston (S.D.S.), and Intellia Therapeutics, Cambridge (J.K., L.W., R.R., D.L., D.S., J.O., A.H., P.Z., Y.X., A.L., A.S.) - both in Massachusetts; the University of Auckland, Auckland, New Zealand (E.J.G.); Umea University, Umea, Sweden (B.P.); Centre Hospitalo-Universitaire de Bicêtre, Assistance Publique-Hôpitaux de Paris, University Paris-Saclay, Le Kremlin-Bicêtre, France (D.A.); and Regeneron Pharmaceuticals, Tarrytown, NY (D.E.G.)., Pilebro B; From the National Amyloidosis Centre, University College London, Royal Free Hospital (M.F., Y.R., J.D.G.), and Richmond Pharmacology (J.T.) - both in London; Brigham and Women's Hospital, Boston (S.D.S.), and Intellia Therapeutics, Cambridge (J.K., L.W., R.R., D.L., D.S., J.O., A.H., P.Z., Y.X., A.L., A.S.) - both in Massachusetts; the University of Auckland, Auckland, New Zealand (E.J.G.); Umea University, Umea, Sweden (B.P.); Centre Hospitalo-Universitaire de Bicêtre, Assistance Publique-Hôpitaux de Paris, University Paris-Saclay, Le Kremlin-Bicêtre, France (D.A.); and Regeneron Pharmaceuticals, Tarrytown, NY (D.E.G.)., Adams D; From the National Amyloidosis Centre, University College London, Royal Free Hospital (M.F., Y.R., J.D.G.), and Richmond Pharmacology (J.T.) - both in London; Brigham and Women's Hospital, Boston (S.D.S.), and Intellia Therapeutics, Cambridge (J.K., L.W., R.R., D.L., D.S., J.O., A.H., P.Z., Y.X., A.L., A.S.) - both in Massachusetts; the University of Auckland, Auckland, New Zealand (E.J.G.); Umea University, Umea, Sweden (B.P.); Centre Hospitalo-Universitaire de Bicêtre, Assistance Publique-Hôpitaux de Paris, University Paris-Saclay, Le Kremlin-Bicêtre, France (D.A.); and Regeneron Pharmaceuticals, Tarrytown, NY (D.E.G.)., Razvi Y; From the National Amyloidosis Centre, University College London, Royal Free Hospital (M.F., Y.R., J.D.G.), and Richmond Pharmacology (J.T.) - both in London; Brigham and Women's Hospital, Boston (S.D.S.), and Intellia Therapeutics, Cambridge (J.K., L.W., R.R., D.L., D.S., J.O., A.H., P.Z., Y.X., A.L., A.S.) - both in Massachusetts; the University of Auckland, Auckland, New Zealand (E.J.G.); Umea University, Umea, Sweden (B.P.); Centre Hospitalo-Universitaire de Bicêtre, Assistance Publique-Hôpitaux de Paris, University Paris-Saclay, Le Kremlin-Bicêtre, France (D.A.); and Regeneron Pharmaceuticals, Tarrytown, NY (D.E.G.)., Olbertz J; From the National Amyloidosis Centre, University College London, Royal Free Hospital (M.F., Y.R., J.D.G.), and Richmond Pharmacology (J.T.) - both in London; Brigham and Women's Hospital, Boston (S.D.S.), and Intellia Therapeutics, Cambridge (J.K., L.W., R.R., D.L., D.S., J.O., A.H., P.Z., Y.X., A.L., A.S.) - both in Massachusetts; the University of Auckland, Auckland, New Zealand (E.J.G.); Umea University, Umea, Sweden (B.P.); Centre Hospitalo-Universitaire de Bicêtre, Assistance Publique-Hôpitaux de Paris, University Paris-Saclay, Le Kremlin-Bicêtre, France (D.A.); and Regeneron Pharmaceuticals, Tarrytown, NY (D.E.G.)., Haagensen A; From the National Amyloidosis Centre, University College London, Royal Free Hospital (M.F., Y.R., J.D.G.), and Richmond Pharmacology (J.T.) - both in London; Brigham and Women's Hospital, Boston (S.D.S.), and Intellia Therapeutics, Cambridge (J.K., L.W., R.R., D.L., D.S., J.O., A.H., P.Z., Y.X., A.L., A.S.) - both in Massachusetts; the University of Auckland, Auckland, New Zealand (E.J.G.); Umea University, Umea, Sweden (B.P.); Centre Hospitalo-Universitaire de Bicêtre, Assistance Publique-Hôpitaux de Paris, University Paris-Saclay, Le Kremlin-Bicêtre, France (D.A.); and Regeneron Pharmaceuticals, Tarrytown, NY (D.E.G.)., Zhu P; From the National Amyloidosis Centre, University College London, Royal Free Hospital (M.F., Y.R., J.D.G.), and Richmond Pharmacology (J.T.) - both in London; Brigham and Women's Hospital, Boston (S.D.S.), and Intellia Therapeutics, Cambridge (J.K., L.W., R.R., D.L., D.S., J.O., A.H., P.Z., Y.X., A.L., A.S.) - both in Massachusetts; the University of Auckland, Auckland, New Zealand (E.J.G.); Umea University, Umea, Sweden (B.P.); Centre Hospitalo-Universitaire de Bicêtre, Assistance Publique-Hôpitaux de Paris, University Paris-Saclay, Le Kremlin-Bicêtre, France (D.A.); and Regeneron Pharmaceuticals, Tarrytown, NY (D.E.G.)., Xu Y; From the National Amyloidosis Centre, University College London, Royal Free Hospital (M.F., Y.R., J.D.G.), and Richmond Pharmacology (J.T.) - both in London; Brigham and Women's Hospital, Boston (S.D.S.), and Intellia Therapeutics, Cambridge (J.K., L.W., R.R., D.L., D.S., J.O., A.H., P.Z., Y.X., A.L., A.S.) - both in Massachusetts; the University of Auckland, Auckland, New Zealand (E.J.G.); Umea University, Umea, Sweden (B.P.); Centre Hospitalo-Universitaire de Bicêtre, Assistance Publique-Hôpitaux de Paris, University Paris-Saclay, Le Kremlin-Bicêtre, France (D.A.); and Regeneron Pharmaceuticals, Tarrytown, NY (D.E.G.)., Leung A; From the National Amyloidosis Centre, University College London, Royal Free Hospital (M.F., Y.R., J.D.G.), and Richmond Pharmacology (J.T.) - both in London; Brigham and Women's Hospital, Boston (S.D.S.), and Intellia Therapeutics, Cambridge (J.K., L.W., R.R., D.L., D.S., J.O., A.H., P.Z., Y.X., A.L., A.S.) - both in Massachusetts; the University of Auckland, Auckland, New Zealand (E.J.G.); Umea University, Umea, Sweden (B.P.); Centre Hospitalo-Universitaire de Bicêtre, Assistance Publique-Hôpitaux de Paris, University Paris-Saclay, Le Kremlin-Bicêtre, France (D.A.); and Regeneron Pharmaceuticals, Tarrytown, NY (D.E.G.)., Sonderfan A; From the National Amyloidosis Centre, University College London, Royal Free Hospital (M.F., Y.R., J.D.G.), and Richmond Pharmacology (J.T.) - both in London; Brigham and Women's Hospital, Boston (S.D.S.), and Intellia Therapeutics, Cambridge (J.K., L.W., R.R., D.L., D.S., J.O., A.H., P.Z., Y.X., A.L., A.S.) - both in Massachusetts; the University of Auckland, Auckland, New Zealand (E.J.G.); Umea University, Umea, Sweden (B.P.); Centre Hospitalo-Universitaire de Bicêtre, Assistance Publique-Hôpitaux de Paris, University Paris-Saclay, Le Kremlin-Bicêtre, France (D.A.); and Regeneron Pharmaceuticals, Tarrytown, NY (D.E.G.)., Gutstein DE; From the National Amyloidosis Centre, University College London, Royal Free Hospital (M.F., Y.R., J.D.G.), and Richmond Pharmacology (J.T.) - both in London; Brigham and Women's Hospital, Boston (S.D.S.), and Intellia Therapeutics, Cambridge (J.K., L.W., R.R., D.L., D.S., J.O., A.H., P.Z., Y.X., A.L., A.S.) - both in Massachusetts; the University of Auckland, Auckland, New Zealand (E.J.G.); Umea University, Umea, Sweden (B.P.); Centre Hospitalo-Universitaire de Bicêtre, Assistance Publique-Hôpitaux de Paris, University Paris-Saclay, Le Kremlin-Bicêtre, France (D.A.); and Regeneron Pharmaceuticals, Tarrytown, NY (D.E.G.)., Gillmore JD; From the National Amyloidosis Centre, University College London, Royal Free Hospital (M.F., Y.R., J.D.G.), and Richmond Pharmacology (J.T.) - both in London; Brigham and Women's Hospital, Boston (S.D.S.), and Intellia Therapeutics, Cambridge (J.K., L.W., R.R., D.L., D.S., J.O., A.H., P.Z., Y.X., A.L., A.S.) - both in Massachusetts; the University of Auckland, Auckland, New Zealand (E.J.G.); Umea University, Umea, Sweden (B.P.); Centre Hospitalo-Universitaire de Bicêtre, Assistance Publique-Hôpitaux de Paris, University Paris-Saclay, Le Kremlin-Bicêtre, France (D.A.); and Regeneron Pharmaceuticals, Tarrytown, NY (D.E.G.).
Source: The New England journal of medicine [N Engl J Med] 2024 Dec 12; Vol. 391 (23), pp. 2231-2241. Date of Electronic Publication: 2024 Nov 16.
Publication Type: Clinical Trial, Phase I; Journal Article
Journal Info: Publisher: Massachusetts Medical Society Country of Publication: United States NLM ID: 0255562 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1533-4406 (Electronic) Linking ISSN: 00284793 NLM ISO Abbreviation: N Engl J Med Subsets: MEDLINE
Database: MEDLINE Ultimate
Description
ISSN:1533-4406
DOI:10.1056/NEJMoa2412309