CRISPR-Cas9 Gene Editing with Nexiguran Ziclumeran for ATTR Cardiomyopathy.
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| Title: | CRISPR-Cas9 Gene Editing with Nexiguran Ziclumeran for ATTR Cardiomyopathy. |
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| 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 |
| FullText | Text: Availability: 0 |
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| Header | DbId: mdl DbLabel: MEDLINE Ultimate An: 39555828 AccessLevel: 2 PubType: Academic Journal PubTypeId: academicJournal PreciseRelevancyScore: 0 |
| IllustrationInfo | |
| Items | – Name: Title Label: Title Group: Ti Data: CRISPR-Cas9 Gene Editing with Nexiguran Ziclumeran for ATTR Cardiomyopathy. – Name: Author Label: Authors Group: Au Data: <searchLink fieldCode="AU" term="%22Fontana+M%22">Fontana M</searchLink>; 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.).<br /><searchLink fieldCode="AU" term="%22Solomon+SD%22">Solomon SD</searchLink>; 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.).<br /><searchLink fieldCode="AU" term="%22Kachadourian+J%22">Kachadourian J</searchLink>; 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.).<br /><searchLink fieldCode="AU" term="%22Walsh+L%22">Walsh L</searchLink>; 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.).<br /><searchLink fieldCode="AU" term="%22Rocha+R%22">Rocha R</searchLink>; 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.).<br /><searchLink fieldCode="AU" term="%22Lebwohl+D%22">Lebwohl D</searchLink>; 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.).<br /><searchLink fieldCode="AU" term="%22Smith+D%22">Smith D</searchLink>; 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.).<br /><searchLink fieldCode="AU" term="%22Täubel+J%22">Täubel J</searchLink>; 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.).<br /><searchLink fieldCode="AU" term="%22Gane+EJ%22">Gane EJ</searchLink>; 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.).<br /><searchLink fieldCode="AU" term="%22Pilebro+B%22">Pilebro B</searchLink>; 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.).<br /><searchLink fieldCode="AU" term="%22Adams+D%22">Adams D</searchLink>; 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.).<br /><searchLink fieldCode="AU" term="%22Razvi+Y%22">Razvi Y</searchLink>; 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.).<br /><searchLink fieldCode="AU" term="%22Olbertz+J%22">Olbertz J</searchLink>; 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.).<br /><searchLink fieldCode="AU" term="%22Haagensen+A%22">Haagensen A</searchLink>; 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.).<br /><searchLink fieldCode="AU" term="%22Zhu+P%22">Zhu P</searchLink>; 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.).<br /><searchLink fieldCode="AU" term="%22Xu+Y%22">Xu Y</searchLink>; 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.).<br /><searchLink fieldCode="AU" term="%22Leung+A%22">Leung A</searchLink>; 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.).<br /><searchLink fieldCode="AU" term="%22Sonderfan+A%22">Sonderfan A</searchLink>; 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.).<br /><searchLink fieldCode="AU" term="%22Gutstein+DE%22">Gutstein DE</searchLink>; 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.).<br /><searchLink fieldCode="AU" term="%22Gillmore+JD%22">Gillmore JD</searchLink>; 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.). – Name: TitleSource Label: Source Group: Src Data: <searchLink fieldCode="JN" term="%220255562%22">The New England journal of medicine</searchLink> [N Engl J Med] 2024 Dec 12; Vol. 391 (23), pp. 2231-2241. <i>Date of Electronic Publication: </i>2024 Nov 16. – Name: TypePub Label: Publication Type Group: TypPub Data: Clinical Trial, Phase I; Journal Article – Name: TitleSource Label: Journal Info Group: Src Data: <i>Publisher: </i><searchLink fieldCode="PB" term="%22Massachusetts+Medical+Society%22">Massachusetts Medical Society </searchLink><i>Country of Publication: </i>United States <i>NLM ID: </i>0255562 <i>Publication Model: </i>Print-Electronic <i>Cited Medium: </i>Internet <i>ISSN: </i>1533-4406 (Electronic) <i>Linking ISSN: </i><searchLink fieldCode="IS" term="%2200284793%22">00284793 </searchLink><i>NLM ISO Abbreviation: </i>N Engl J Med <i>Subsets: </i>MEDLINE |
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| RecordInfo | BibRecord: BibEntity: Identifiers: – Type: doi Value: 10.1056/NEJMoa2412309 Languages: – Code: eng Text: English PhysicalDescription: Pagination: StartPage: 2231 Titles: – TitleFull: CRISPR-Cas9 Gene Editing with Nexiguran Ziclumeran for ATTR Cardiomyopathy. Type: main BibRelationships: HasContributorRelationships: – PersonEntity: Name: NameFull: Fontana M – PersonEntity: Name: NameFull: Solomon SD – PersonEntity: Name: NameFull: Kachadourian J – PersonEntity: Name: NameFull: Walsh L – PersonEntity: Name: NameFull: Rocha R – PersonEntity: Name: NameFull: Lebwohl D – PersonEntity: Name: NameFull: Smith D – PersonEntity: Name: NameFull: Täubel J – PersonEntity: Name: NameFull: Gane EJ – PersonEntity: Name: NameFull: Pilebro B – PersonEntity: Name: NameFull: Adams D – PersonEntity: Name: NameFull: Razvi Y – PersonEntity: Name: NameFull: Olbertz J – PersonEntity: Name: NameFull: Haagensen A – PersonEntity: Name: NameFull: Zhu P – PersonEntity: Name: NameFull: Xu Y – PersonEntity: Name: NameFull: Leung A – PersonEntity: Name: NameFull: Sonderfan A – PersonEntity: Name: NameFull: Gutstein DE – PersonEntity: Name: NameFull: Gillmore JD IsPartOfRelationships: – BibEntity: Dates: – D: 12 M: 12 Text: 2024 Dec 12 Type: published Y: 2024 Identifiers: – Type: issn-electronic Value: 1533-4406 Numbering: – Type: volume Value: 391 – Type: issue Value: 23 Titles: – TitleFull: The New England journal of medicine Type: main |
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