CRISPR-Based Therapy for Hereditary Angioedema.
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| Title: | CRISPR-Based Therapy for Hereditary Angioedema. |
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| Authors: | Cohn DM; From Amsterdam Cardiovascular Sciences, Amsterdam University Medical Center, University of Amsterdam, Amsterdam (D.M.C., R.S.P); Cambridge University Hospitals, NHS Foundation Trust, Cambridge, United Kingdom (P.G.); the Institute of Allergology, Charité-Universitätsmedizin Berlin, Freie Universität Berlin and Humboldt-Universität zu Berlin, and Immunology and Allergology, Fraunhofer Institute for Translational Medicine and Pharmacology ITMP, Berlin (M.M.), and the Department of Children and Adolescents, University Hospital Frankfurt, Goethe University Frankfurt, Frankfurt (E.A.-P.) - all in Germany; the Department of Medicine, Campbelltown Hospital and Western Sydney University, Sydney (C.H.K.); University of Lille, U1286-INFINITE-Institute for Translational Research in Inflammation, F-59000, INSERM, and the Department of Internal Medicine and Clinical Immunology, CHU Lille, National Reference Center for Angioedema (CREAK), Lille (D.L.), and CREAK, Angioedema Center of Reference and Excellence (ACARE), Grenoble Alpes University Hospital, and the Translational Research in Autoimmunity and Inflammation Arm (T-RAIG), French National Center for Scientific Research (CNRS), Grenoble (L.B.) - all in France; Intellia Therapeutics, Cambridge, MA (D.M., J.S.B., M.Y.S., A.G., Y.X., A.M.A., D.L.); and the Department of Immunology, Auckland City Hospital, and the Department of Medicine, University of Auckland - both in Auckland, New Zealand (K.L., H.J.L.)., Gurugama P; From Amsterdam Cardiovascular Sciences, Amsterdam University Medical Center, University of Amsterdam, Amsterdam (D.M.C., R.S.P); Cambridge University Hospitals, NHS Foundation Trust, Cambridge, United Kingdom (P.G.); the Institute of Allergology, Charité-Universitätsmedizin Berlin, Freie Universität Berlin and Humboldt-Universität zu Berlin, and Immunology and Allergology, Fraunhofer Institute for Translational Medicine and Pharmacology ITMP, Berlin (M.M.), and the Department of Children and Adolescents, University Hospital Frankfurt, Goethe University Frankfurt, Frankfurt (E.A.-P.) - all in Germany; the Department of Medicine, Campbelltown Hospital and Western Sydney University, Sydney (C.H.K.); University of Lille, U1286-INFINITE-Institute for Translational Research in Inflammation, F-59000, INSERM, and the Department of Internal Medicine and Clinical Immunology, CHU Lille, National Reference Center for Angioedema (CREAK), Lille (D.L.), and CREAK, Angioedema Center of Reference and Excellence (ACARE), Grenoble Alpes University Hospital, and the Translational Research in Autoimmunity and Inflammation Arm (T-RAIG), French National Center for Scientific Research (CNRS), Grenoble (L.B.) - all in France; Intellia Therapeutics, Cambridge, MA (D.M., J.S.B., M.Y.S., A.G., Y.X., A.M.A., D.L.); and the Department of Immunology, Auckland City Hospital, and the Department of Medicine, University of Auckland - both in Auckland, New Zealand (K.L., H.J.L.)., Magerl M; From Amsterdam Cardiovascular Sciences, Amsterdam University Medical Center, University of Amsterdam, Amsterdam (D.M.C., R.S.P); Cambridge University Hospitals, NHS Foundation Trust, Cambridge, United Kingdom (P.G.); the Institute of Allergology, Charité-Universitätsmedizin Berlin, Freie Universität Berlin and Humboldt-Universität zu Berlin, and Immunology and Allergology, Fraunhofer Institute for Translational Medicine and Pharmacology ITMP, Berlin (M.M.), and the Department of Children and Adolescents, University Hospital Frankfurt, Goethe University Frankfurt, Frankfurt (E.A.-P.) - all in Germany; the Department of Medicine, Campbelltown Hospital and Western Sydney University, Sydney (C.H.K.); University of Lille, U1286-INFINITE-Institute for Translational Research in Inflammation, F-59000, INSERM, and the Department of Internal Medicine and Clinical Immunology, CHU Lille, National Reference Center for Angioedema (CREAK), Lille (D.L.), and CREAK, Angioedema Center of Reference and Excellence (ACARE), Grenoble Alpes University Hospital, and the Translational Research in Autoimmunity and Inflammation Arm (T-RAIG), French National Center for Scientific Research (CNRS), Grenoble (L.B.) - all in France; Intellia Therapeutics, Cambridge, MA (D.M., J.S.B., M.Y.S., A.G., Y.X., A.M.A., D.L.); and the Department of Immunology, Auckland City Hospital, and the Department of Medicine, University of Auckland - both in Auckland, New Zealand (K.L., H.J.L.)., Katelaris CH; From Amsterdam Cardiovascular Sciences, Amsterdam University Medical Center, University of Amsterdam, Amsterdam (D.M.C., R.S.P); Cambridge University Hospitals, NHS Foundation Trust, Cambridge, United Kingdom (P.G.); the Institute of Allergology, Charité-Universitätsmedizin Berlin, Freie Universität Berlin and Humboldt-Universität zu Berlin, and Immunology and Allergology, Fraunhofer Institute for Translational Medicine and Pharmacology ITMP, Berlin (M.M.), and the Department of Children and Adolescents, University Hospital Frankfurt, Goethe University Frankfurt, Frankfurt (E.A.-P.) - all in Germany; the Department of Medicine, Campbelltown Hospital and Western Sydney University, Sydney (C.H.K.); University of Lille, U1286-INFINITE-Institute for Translational Research in Inflammation, F-59000, INSERM, and the Department of Internal Medicine and Clinical Immunology, CHU Lille, National Reference Center for Angioedema (CREAK), Lille (D.L.), and CREAK, Angioedema Center of Reference and Excellence (ACARE), Grenoble Alpes University Hospital, and the Translational Research in Autoimmunity and Inflammation Arm (T-RAIG), French National Center for Scientific Research (CNRS), Grenoble (L.B.) - all in France; Intellia Therapeutics, Cambridge, MA (D.M., J.S.B., M.Y.S., A.G., Y.X., A.M.A., D.L.); and the Department of Immunology, Auckland City Hospital, and the Department of Medicine, University of Auckland - both in Auckland, New Zealand (K.L., H.J.L.)., Launay D; From Amsterdam Cardiovascular Sciences, Amsterdam University Medical Center, University of Amsterdam, Amsterdam (D.M.C., R.S.P); Cambridge University Hospitals, NHS Foundation Trust, Cambridge, United Kingdom (P.G.); the Institute of Allergology, Charité-Universitätsmedizin Berlin, Freie Universität Berlin and Humboldt-Universität zu Berlin, and Immunology and Allergology, Fraunhofer Institute for Translational Medicine and Pharmacology ITMP, Berlin (M.M.), and the Department of Children and Adolescents, University Hospital Frankfurt, Goethe University Frankfurt, Frankfurt (E.A.-P.) - all in Germany; the Department of Medicine, Campbelltown Hospital and Western Sydney University, Sydney (C.H.K.); University of Lille, U1286-INFINITE-Institute for Translational Research in Inflammation, F-59000, INSERM, and the Department of Internal Medicine and Clinical Immunology, CHU Lille, National Reference Center for Angioedema (CREAK), Lille (D.L.), and CREAK, Angioedema Center of Reference and Excellence (ACARE), Grenoble Alpes University Hospital, and the Translational Research in Autoimmunity and Inflammation Arm (T-RAIG), French National Center for Scientific Research (CNRS), Grenoble (L.B.) - all in France; Intellia Therapeutics, Cambridge, MA (D.M., J.S.B., M.Y.S., A.G., Y.X., A.M.A., D.L.); and the Department of Immunology, Auckland City Hospital, and the Department of Medicine, University of Auckland - both in Auckland, New Zealand (K.L., H.J.L.)., Bouillet L; From Amsterdam Cardiovascular Sciences, Amsterdam University Medical Center, University of Amsterdam, Amsterdam (D.M.C., R.S.P); Cambridge University Hospitals, NHS Foundation Trust, Cambridge, United Kingdom (P.G.); the Institute of Allergology, Charité-Universitätsmedizin Berlin, Freie Universität Berlin and Humboldt-Universität zu Berlin, and Immunology and Allergology, Fraunhofer Institute for Translational Medicine and Pharmacology ITMP, Berlin (M.M.), and the Department of Children and Adolescents, University Hospital Frankfurt, Goethe University Frankfurt, Frankfurt (E.A.-P.) - all in Germany; the Department of Medicine, Campbelltown Hospital and Western Sydney University, Sydney (C.H.K.); University of Lille, U1286-INFINITE-Institute for Translational Research in Inflammation, F-59000, INSERM, and the Department of Internal Medicine and Clinical Immunology, CHU Lille, National Reference Center for Angioedema (CREAK), Lille (D.L.), and CREAK, Angioedema Center of Reference and Excellence (ACARE), Grenoble Alpes University Hospital, and the Translational Research in Autoimmunity and Inflammation Arm (T-RAIG), French National Center for Scientific Research (CNRS), Grenoble (L.B.) - all in France; Intellia Therapeutics, Cambridge, MA (D.M., J.S.B., M.Y.S., A.G., Y.X., A.M.A., D.L.); and the Department of Immunology, Auckland City Hospital, and the Department of Medicine, University of Auckland - both in Auckland, New Zealand (K.L., H.J.L.)., Petersen RS; From Amsterdam Cardiovascular Sciences, Amsterdam University Medical Center, University of Amsterdam, Amsterdam (D.M.C., R.S.P); Cambridge University Hospitals, NHS Foundation Trust, Cambridge, United Kingdom (P.G.); the Institute of Allergology, Charité-Universitätsmedizin Berlin, Freie Universität Berlin and Humboldt-Universität zu Berlin, and Immunology and Allergology, Fraunhofer Institute for Translational Medicine and Pharmacology ITMP, Berlin (M.M.), and the Department of Children and Adolescents, University Hospital Frankfurt, Goethe University Frankfurt, Frankfurt (E.A.-P.) - all in Germany; the Department of Medicine, Campbelltown Hospital and Western Sydney University, Sydney (C.H.K.); University of Lille, U1286-INFINITE-Institute for Translational Research in Inflammation, F-59000, INSERM, and the Department of Internal Medicine and Clinical Immunology, CHU Lille, National Reference Center for Angioedema (CREAK), Lille (D.L.), and CREAK, Angioedema Center of Reference and Excellence (ACARE), Grenoble Alpes University Hospital, and the Translational Research in Autoimmunity and Inflammation Arm (T-RAIG), French National Center for Scientific Research (CNRS), Grenoble (L.B.) - all in France; Intellia Therapeutics, Cambridge, MA (D.M., J.S.B., M.Y.S., A.G., Y.X., A.M.A., D.L.); and the Department of Immunology, Auckland City Hospital, and the Department of Medicine, University of Auckland - both in Auckland, New Zealand (K.L., H.J.L.)., Lindsay K; From Amsterdam Cardiovascular Sciences, Amsterdam University Medical Center, University of Amsterdam, Amsterdam (D.M.C., R.S.P); Cambridge University Hospitals, NHS Foundation Trust, Cambridge, United Kingdom (P.G.); the Institute of Allergology, Charité-Universitätsmedizin Berlin, Freie Universität Berlin and Humboldt-Universität zu Berlin, and Immunology and Allergology, Fraunhofer Institute for Translational Medicine and Pharmacology ITMP, Berlin (M.M.), and the Department of Children and Adolescents, University Hospital Frankfurt, Goethe University Frankfurt, Frankfurt (E.A.-P.) - all in Germany; the Department of Medicine, Campbelltown Hospital and Western Sydney University, Sydney (C.H.K.); University of Lille, U1286-INFINITE-Institute for Translational Research in Inflammation, F-59000, INSERM, and the Department of Internal Medicine and Clinical Immunology, CHU Lille, National Reference Center for Angioedema (CREAK), Lille (D.L.), and CREAK, Angioedema Center of Reference and Excellence (ACARE), Grenoble Alpes University Hospital, and the Translational Research in Autoimmunity and Inflammation Arm (T-RAIG), French National Center for Scientific Research (CNRS), Grenoble (L.B.) - all in France; Intellia Therapeutics, Cambridge, MA (D.M., J.S.B., M.Y.S., A.G., Y.X., A.M.A., D.L.); and the Department of Immunology, Auckland City Hospital, and the Department of Medicine, University of Auckland - both in Auckland, New Zealand (K.L., H.J.L.)., Aygören-Pürsün E; From Amsterdam Cardiovascular Sciences, Amsterdam University Medical Center, University of Amsterdam, Amsterdam (D.M.C., R.S.P); Cambridge University Hospitals, NHS Foundation Trust, Cambridge, United Kingdom (P.G.); the Institute of Allergology, Charité-Universitätsmedizin Berlin, Freie Universität Berlin and Humboldt-Universität zu Berlin, and Immunology and Allergology, Fraunhofer Institute for Translational Medicine and Pharmacology ITMP, Berlin (M.M.), and the Department of Children and Adolescents, University Hospital Frankfurt, Goethe University Frankfurt, Frankfurt (E.A.-P.) - all in Germany; the Department of Medicine, Campbelltown Hospital and Western Sydney University, Sydney (C.H.K.); University of Lille, U1286-INFINITE-Institute for Translational Research in Inflammation, F-59000, INSERM, and the Department of Internal Medicine and Clinical Immunology, CHU Lille, National Reference Center for Angioedema (CREAK), Lille (D.L.), and CREAK, Angioedema Center of Reference and Excellence (ACARE), Grenoble Alpes University Hospital, and the Translational Research in Autoimmunity and Inflammation Arm (T-RAIG), French National Center for Scientific Research (CNRS), Grenoble (L.B.) - all in France; Intellia Therapeutics, Cambridge, MA (D.M., J.S.B., M.Y.S., A.G., Y.X., A.M.A., D.L.); and the Department of Immunology, Auckland City Hospital, and the Department of Medicine, University of Auckland - both in Auckland, New Zealand (K.L., H.J.L.)., Maag D; From Amsterdam Cardiovascular Sciences, Amsterdam University Medical Center, University of Amsterdam, Amsterdam (D.M.C., R.S.P); Cambridge University Hospitals, NHS Foundation Trust, Cambridge, United Kingdom (P.G.); the Institute of Allergology, Charité-Universitätsmedizin Berlin, Freie Universität Berlin and Humboldt-Universität zu Berlin, and Immunology and Allergology, Fraunhofer Institute for Translational Medicine and Pharmacology ITMP, Berlin (M.M.), and the Department of Children and Adolescents, University Hospital Frankfurt, Goethe University Frankfurt, Frankfurt (E.A.-P.) - all in Germany; the Department of Medicine, Campbelltown Hospital and Western Sydney University, Sydney (C.H.K.); University of Lille, U1286-INFINITE-Institute for Translational Research in Inflammation, F-59000, INSERM, and the Department of Internal Medicine and Clinical Immunology, CHU Lille, National Reference Center for Angioedema (CREAK), Lille (D.L.), and CREAK, Angioedema Center of Reference and Excellence (ACARE), Grenoble Alpes University Hospital, and the Translational Research in Autoimmunity and Inflammation Arm (T-RAIG), French National Center for Scientific Research (CNRS), Grenoble (L.B.) - all in France; Intellia Therapeutics, Cambridge, MA (D.M., J.S.B., M.Y.S., A.G., Y.X., A.M.A., D.L.); and the Department of Immunology, Auckland City Hospital, and the Department of Medicine, University of Auckland - both in Auckland, New Zealand (K.L., H.J.L.)., Butler JS; From Amsterdam Cardiovascular Sciences, Amsterdam University Medical Center, University of Amsterdam, Amsterdam (D.M.C., R.S.P); Cambridge University Hospitals, NHS Foundation Trust, Cambridge, United Kingdom (P.G.); the Institute of Allergology, Charité-Universitätsmedizin Berlin, Freie Universität Berlin and Humboldt-Universität zu Berlin, and Immunology and Allergology, Fraunhofer Institute for Translational Medicine and Pharmacology ITMP, Berlin (M.M.), and the Department of Children and Adolescents, University Hospital Frankfurt, Goethe University Frankfurt, Frankfurt (E.A.-P.) - all in Germany; the Department of Medicine, Campbelltown Hospital and Western Sydney University, Sydney (C.H.K.); University of Lille, U1286-INFINITE-Institute for Translational Research in Inflammation, F-59000, INSERM, and the Department of Internal Medicine and Clinical Immunology, CHU Lille, National Reference Center for Angioedema (CREAK), Lille (D.L.), and CREAK, Angioedema Center of Reference and Excellence (ACARE), Grenoble Alpes University Hospital, and the Translational Research in Autoimmunity and Inflammation Arm (T-RAIG), French National Center for Scientific Research (CNRS), Grenoble (L.B.) - all in France; Intellia Therapeutics, Cambridge, MA (D.M., J.S.B., M.Y.S., A.G., Y.X., A.M.A., D.L.); and the Department of Immunology, Auckland City Hospital, and the Department of Medicine, University of Auckland - both in Auckland, New Zealand (K.L., H.J.L.)., Shah MY; From Amsterdam Cardiovascular Sciences, Amsterdam University Medical Center, University of Amsterdam, Amsterdam (D.M.C., R.S.P); Cambridge University Hospitals, NHS Foundation Trust, Cambridge, United Kingdom (P.G.); the Institute of Allergology, Charité-Universitätsmedizin Berlin, Freie Universität Berlin and Humboldt-Universität zu Berlin, and Immunology and Allergology, Fraunhofer Institute for Translational Medicine and Pharmacology ITMP, Berlin (M.M.), and the Department of Children and Adolescents, University Hospital Frankfurt, Goethe University Frankfurt, Frankfurt (E.A.-P.) - all in Germany; the Department of Medicine, Campbelltown Hospital and Western Sydney University, Sydney (C.H.K.); University of Lille, U1286-INFINITE-Institute for Translational Research in Inflammation, F-59000, INSERM, and the Department of Internal Medicine and Clinical Immunology, CHU Lille, National Reference Center for Angioedema (CREAK), Lille (D.L.), and CREAK, Angioedema Center of Reference and Excellence (ACARE), Grenoble Alpes University Hospital, and the Translational Research in Autoimmunity and Inflammation Arm (T-RAIG), French National Center for Scientific Research (CNRS), Grenoble (L.B.) - all in France; Intellia Therapeutics, Cambridge, MA (D.M., J.S.B., M.Y.S., A.G., Y.X., A.M.A., D.L.); and the Department of Immunology, Auckland City Hospital, and the Department of Medicine, University of Auckland - both in Auckland, New Zealand (K.L., H.J.L.)., Golden A; From Amsterdam Cardiovascular Sciences, Amsterdam University Medical Center, University of Amsterdam, Amsterdam (D.M.C., R.S.P); Cambridge University Hospitals, NHS Foundation Trust, Cambridge, United Kingdom (P.G.); the Institute of Allergology, Charité-Universitätsmedizin Berlin, Freie Universität Berlin and Humboldt-Universität zu Berlin, and Immunology and Allergology, Fraunhofer Institute for Translational Medicine and Pharmacology ITMP, Berlin (M.M.), and the Department of Children and Adolescents, University Hospital Frankfurt, Goethe University Frankfurt, Frankfurt (E.A.-P.) - all in Germany; the Department of Medicine, Campbelltown Hospital and Western Sydney University, Sydney (C.H.K.); University of Lille, U1286-INFINITE-Institute for Translational Research in Inflammation, F-59000, INSERM, and the Department of Internal Medicine and Clinical Immunology, CHU Lille, National Reference Center for Angioedema (CREAK), Lille (D.L.), and CREAK, Angioedema Center of Reference and Excellence (ACARE), Grenoble Alpes University Hospital, and the Translational Research in Autoimmunity and Inflammation Arm (T-RAIG), French National Center for Scientific Research (CNRS), Grenoble (L.B.) - all in France; Intellia Therapeutics, Cambridge, MA (D.M., J.S.B., M.Y.S., A.G., Y.X., A.M.A., D.L.); and the Department of Immunology, Auckland City Hospital, and the Department of Medicine, University of Auckland - both in Auckland, New Zealand (K.L., H.J.L.)., Xu Y; From Amsterdam Cardiovascular Sciences, Amsterdam University Medical Center, University of Amsterdam, Amsterdam (D.M.C., R.S.P); Cambridge University Hospitals, NHS Foundation Trust, Cambridge, United Kingdom (P.G.); the Institute of Allergology, Charité-Universitätsmedizin Berlin, Freie Universität Berlin and Humboldt-Universität zu Berlin, and Immunology and Allergology, Fraunhofer Institute for Translational Medicine and Pharmacology ITMP, Berlin (M.M.), and the Department of Children and Adolescents, University Hospital Frankfurt, Goethe University Frankfurt, Frankfurt (E.A.-P.) - all in Germany; the Department of Medicine, Campbelltown Hospital and Western Sydney University, Sydney (C.H.K.); University of Lille, U1286-INFINITE-Institute for Translational Research in Inflammation, F-59000, INSERM, and the Department of Internal Medicine and Clinical Immunology, CHU Lille, National Reference Center for Angioedema (CREAK), Lille (D.L.), and CREAK, Angioedema Center of Reference and Excellence (ACARE), Grenoble Alpes University Hospital, and the Translational Research in Autoimmunity and Inflammation Arm (T-RAIG), French National Center for Scientific Research (CNRS), Grenoble (L.B.) - all in France; Intellia Therapeutics, Cambridge, MA (D.M., J.S.B., M.Y.S., A.G., Y.X., A.M.A., D.L.); and the Department of Immunology, Auckland City Hospital, and the Department of Medicine, University of Auckland - both in Auckland, New Zealand (K.L., H.J.L.)., Abdelhady AM; From Amsterdam Cardiovascular Sciences, Amsterdam University Medical Center, University of Amsterdam, Amsterdam (D.M.C., R.S.P); Cambridge University Hospitals, NHS Foundation Trust, Cambridge, United Kingdom (P.G.); the Institute of Allergology, Charité-Universitätsmedizin Berlin, Freie Universität Berlin and Humboldt-Universität zu Berlin, and Immunology and Allergology, Fraunhofer Institute for Translational Medicine and Pharmacology ITMP, Berlin (M.M.), and the Department of Children and Adolescents, University Hospital Frankfurt, Goethe University Frankfurt, Frankfurt (E.A.-P.) - all in Germany; the Department of Medicine, Campbelltown Hospital and Western Sydney University, Sydney (C.H.K.); University of Lille, U1286-INFINITE-Institute for Translational Research in Inflammation, F-59000, INSERM, and the Department of Internal Medicine and Clinical Immunology, CHU Lille, National Reference Center for Angioedema (CREAK), Lille (D.L.), and CREAK, Angioedema Center of Reference and Excellence (ACARE), Grenoble Alpes University Hospital, and the Translational Research in Autoimmunity and Inflammation Arm (T-RAIG), French National Center for Scientific Research (CNRS), Grenoble (L.B.) - all in France; Intellia Therapeutics, Cambridge, MA (D.M., J.S.B., M.Y.S., A.G., Y.X., A.M.A., D.L.); and the Department of Immunology, Auckland City Hospital, and the Department of Medicine, University of Auckland - both in Auckland, New Zealand (K.L., H.J.L.)., Lebwohl D; From Amsterdam Cardiovascular Sciences, Amsterdam University Medical Center, University of Amsterdam, Amsterdam (D.M.C., R.S.P); Cambridge University Hospitals, NHS Foundation Trust, Cambridge, United Kingdom (P.G.); the Institute of Allergology, Charité-Universitätsmedizin Berlin, Freie Universität Berlin and Humboldt-Universität zu Berlin, and Immunology and Allergology, Fraunhofer Institute for Translational Medicine and Pharmacology ITMP, Berlin (M.M.), and the Department of Children and Adolescents, University Hospital Frankfurt, Goethe University Frankfurt, Frankfurt (E.A.-P.) - all in Germany; the Department of Medicine, Campbelltown Hospital and Western Sydney University, Sydney (C.H.K.); University of Lille, U1286-INFINITE-Institute for Translational Research in Inflammation, F-59000, INSERM, and the Department of Internal Medicine and Clinical Immunology, CHU Lille, National Reference Center for Angioedema (CREAK), Lille (D.L.), and CREAK, Angioedema Center of Reference and Excellence (ACARE), Grenoble Alpes University Hospital, and the Translational Research in Autoimmunity and Inflammation Arm (T-RAIG), French National Center for Scientific Research (CNRS), Grenoble (L.B.) - all in France; Intellia Therapeutics, Cambridge, MA (D.M., J.S.B., M.Y.S., A.G., Y.X., A.M.A., D.L.); and the Department of Immunology, Auckland City Hospital, and the Department of Medicine, University of Auckland - both in Auckland, New Zealand (K.L., H.J.L.)., Longhurst HJ; From Amsterdam Cardiovascular Sciences, Amsterdam University Medical Center, University of Amsterdam, Amsterdam (D.M.C., R.S.P); Cambridge University Hospitals, NHS Foundation Trust, Cambridge, United Kingdom (P.G.); the Institute of Allergology, Charité-Universitätsmedizin Berlin, Freie Universität Berlin and Humboldt-Universität zu Berlin, and Immunology and Allergology, Fraunhofer Institute for Translational Medicine and Pharmacology ITMP, Berlin (M.M.), and the Department of Children and Adolescents, University Hospital Frankfurt, Goethe University Frankfurt, Frankfurt (E.A.-P.) - all in Germany; the Department of Medicine, Campbelltown Hospital and Western Sydney University, Sydney (C.H.K.); University of Lille, U1286-INFINITE-Institute for Translational Research in Inflammation, F-59000, INSERM, and the Department of Internal Medicine and Clinical Immunology, CHU Lille, National Reference Center for Angioedema (CREAK), Lille (D.L.), and CREAK, Angioedema Center of Reference and Excellence (ACARE), Grenoble Alpes University Hospital, and the Translational Research in Autoimmunity and Inflammation Arm (T-RAIG), French National Center for Scientific Research (CNRS), Grenoble (L.B.) - all in France; Intellia Therapeutics, Cambridge, MA (D.M., J.S.B., M.Y.S., A.G., Y.X., A.M.A., D.L.); and the Department of Immunology, Auckland City Hospital, and the Department of Medicine, University of Auckland - both in Auckland, New Zealand (K.L., H.J.L.). |
| Source: | The New England journal of medicine [N Engl J Med] 2025 Jan 30; Vol. 392 (5), pp. 458-467. Date of Electronic Publication: 2024 Oct 24. |
| Publication Type: | Clinical Trial, Phase II; Journal Article; Multicenter Study; Randomized Controlled Trial |
| 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: 39445704 AccessLevel: 2 PubType: Academic Journal PubTypeId: academicJournal PreciseRelevancyScore: 0 |
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| Items | – Name: Title Label: Title Group: Ti Data: CRISPR-Based Therapy for Hereditary Angioedema. – Name: Author Label: Authors Group: Au Data: <searchLink fieldCode="AU" term="%22Cohn+DM%22">Cohn DM</searchLink>; From Amsterdam Cardiovascular Sciences, Amsterdam University Medical Center, University of Amsterdam, Amsterdam (D.M.C., R.S.P); Cambridge University Hospitals, NHS Foundation Trust, Cambridge, United Kingdom (P.G.); the Institute of Allergology, Charité-Universitätsmedizin Berlin, Freie Universität Berlin and Humboldt-Universität zu Berlin, and Immunology and Allergology, Fraunhofer Institute for Translational Medicine and Pharmacology ITMP, Berlin (M.M.), and the Department of Children and Adolescents, University Hospital Frankfurt, Goethe University Frankfurt, Frankfurt (E.A.-P.) - all in Germany; the Department of Medicine, Campbelltown Hospital and Western Sydney University, Sydney (C.H.K.); University of Lille, U1286-INFINITE-Institute for Translational Research in Inflammation, F-59000, INSERM, and the Department of Internal Medicine and Clinical Immunology, CHU Lille, National Reference Center for Angioedema (CREAK), Lille (D.L.), and CREAK, Angioedema Center of Reference and Excellence (ACARE), Grenoble Alpes University Hospital, and the Translational Research in Autoimmunity and Inflammation Arm (T-RAIG), French National Center for Scientific Research (CNRS), Grenoble (L.B.) - all in France; Intellia Therapeutics, Cambridge, MA (D.M., J.S.B., M.Y.S., A.G., Y.X., A.M.A., D.L.); and the Department of Immunology, Auckland City Hospital, and the Department of Medicine, University of Auckland - both in Auckland, New Zealand (K.L., H.J.L.).<br /><searchLink fieldCode="AU" term="%22Gurugama+P%22">Gurugama P</searchLink>; From Amsterdam Cardiovascular Sciences, Amsterdam University Medical Center, University of Amsterdam, Amsterdam (D.M.C., R.S.P); Cambridge University Hospitals, NHS Foundation Trust, Cambridge, United Kingdom (P.G.); the Institute of Allergology, Charité-Universitätsmedizin Berlin, Freie Universität Berlin and Humboldt-Universität zu Berlin, and Immunology and Allergology, Fraunhofer Institute for Translational Medicine and Pharmacology ITMP, Berlin (M.M.), and the Department of Children and Adolescents, University Hospital Frankfurt, Goethe University Frankfurt, Frankfurt (E.A.-P.) - all in Germany; the Department of Medicine, Campbelltown Hospital and Western Sydney University, Sydney (C.H.K.); University of Lille, U1286-INFINITE-Institute for Translational Research in Inflammation, F-59000, INSERM, and the Department of Internal Medicine and Clinical Immunology, CHU Lille, National Reference Center for Angioedema (CREAK), Lille (D.L.), and CREAK, Angioedema Center of Reference and Excellence (ACARE), Grenoble Alpes University Hospital, and the Translational Research in Autoimmunity and Inflammation Arm (T-RAIG), French National Center for Scientific Research (CNRS), Grenoble (L.B.) - all in France; Intellia Therapeutics, Cambridge, MA (D.M., J.S.B., M.Y.S., A.G., Y.X., A.M.A., D.L.); and the Department of Immunology, Auckland City Hospital, and the Department of Medicine, University of Auckland - both in Auckland, New Zealand (K.L., H.J.L.).<br /><searchLink fieldCode="AU" term="%22Magerl+M%22">Magerl M</searchLink>; From Amsterdam Cardiovascular Sciences, Amsterdam University Medical Center, University of Amsterdam, Amsterdam (D.M.C., R.S.P); Cambridge University Hospitals, NHS Foundation Trust, Cambridge, United Kingdom (P.G.); the Institute of Allergology, Charité-Universitätsmedizin Berlin, Freie Universität Berlin and Humboldt-Universität zu Berlin, and Immunology and Allergology, Fraunhofer Institute for Translational Medicine and Pharmacology ITMP, Berlin (M.M.), and the Department of Children and Adolescents, University Hospital Frankfurt, Goethe University Frankfurt, Frankfurt (E.A.-P.) - all in Germany; the Department of Medicine, Campbelltown Hospital and Western Sydney University, Sydney (C.H.K.); University of Lille, U1286-INFINITE-Institute for Translational Research in Inflammation, F-59000, INSERM, and the Department of Internal Medicine and Clinical Immunology, CHU Lille, National Reference Center for Angioedema (CREAK), Lille (D.L.), and CREAK, Angioedema Center of Reference and Excellence (ACARE), Grenoble Alpes University Hospital, and the Translational Research in Autoimmunity and Inflammation Arm (T-RAIG), French National Center for Scientific Research (CNRS), Grenoble (L.B.) - all in France; Intellia Therapeutics, Cambridge, MA (D.M., J.S.B., M.Y.S., A.G., Y.X., A.M.A., D.L.); and the Department of Immunology, Auckland City Hospital, and the Department of Medicine, University of Auckland - both in Auckland, New Zealand (K.L., H.J.L.).<br /><searchLink fieldCode="AU" term="%22Katelaris+CH%22">Katelaris CH</searchLink>; From Amsterdam Cardiovascular Sciences, Amsterdam University Medical Center, University of Amsterdam, Amsterdam (D.M.C., R.S.P); Cambridge University Hospitals, NHS Foundation Trust, Cambridge, United Kingdom (P.G.); the Institute of Allergology, Charité-Universitätsmedizin Berlin, Freie Universität Berlin and Humboldt-Universität zu Berlin, and Immunology and Allergology, Fraunhofer Institute for Translational Medicine and Pharmacology ITMP, Berlin (M.M.), and the Department of Children and Adolescents, University Hospital Frankfurt, Goethe University Frankfurt, Frankfurt (E.A.-P.) - all in Germany; the Department of Medicine, Campbelltown Hospital and Western Sydney University, Sydney (C.H.K.); University of Lille, U1286-INFINITE-Institute for Translational Research in Inflammation, F-59000, INSERM, and the Department of Internal Medicine and Clinical Immunology, CHU Lille, National Reference Center for Angioedema (CREAK), Lille (D.L.), and CREAK, Angioedema Center of Reference and Excellence (ACARE), Grenoble Alpes University Hospital, and the Translational Research in Autoimmunity and Inflammation Arm (T-RAIG), French National Center for Scientific Research (CNRS), Grenoble (L.B.) - all in France; Intellia Therapeutics, Cambridge, MA (D.M., J.S.B., M.Y.S., A.G., Y.X., A.M.A., D.L.); and the Department of Immunology, Auckland City Hospital, and the Department of Medicine, University of Auckland - both in Auckland, New Zealand (K.L., H.J.L.).<br /><searchLink fieldCode="AU" term="%22Launay+D%22">Launay D</searchLink>; From Amsterdam Cardiovascular Sciences, Amsterdam University Medical Center, University of Amsterdam, Amsterdam (D.M.C., R.S.P); Cambridge University Hospitals, NHS Foundation Trust, Cambridge, United Kingdom (P.G.); the Institute of Allergology, Charité-Universitätsmedizin Berlin, Freie Universität Berlin and Humboldt-Universität zu Berlin, and Immunology and Allergology, Fraunhofer Institute for Translational Medicine and Pharmacology ITMP, Berlin (M.M.), and the Department of Children and Adolescents, University Hospital Frankfurt, Goethe University Frankfurt, Frankfurt (E.A.-P.) - all in Germany; the Department of Medicine, Campbelltown Hospital and Western Sydney University, Sydney (C.H.K.); University of Lille, U1286-INFINITE-Institute for Translational Research in Inflammation, F-59000, INSERM, and the Department of Internal Medicine and Clinical Immunology, CHU Lille, National Reference Center for Angioedema (CREAK), Lille (D.L.), and CREAK, Angioedema Center of Reference and Excellence (ACARE), Grenoble Alpes University Hospital, and the Translational Research in Autoimmunity and Inflammation Arm (T-RAIG), French National Center for Scientific Research (CNRS), Grenoble (L.B.) - all in France; Intellia Therapeutics, Cambridge, MA (D.M., J.S.B., M.Y.S., A.G., Y.X., A.M.A., D.L.); and the Department of Immunology, Auckland City Hospital, and the Department of Medicine, University of Auckland - both in Auckland, New Zealand (K.L., H.J.L.).<br /><searchLink fieldCode="AU" term="%22Bouillet+L%22">Bouillet L</searchLink>; From Amsterdam Cardiovascular Sciences, Amsterdam University Medical Center, University of Amsterdam, Amsterdam (D.M.C., R.S.P); Cambridge University Hospitals, NHS Foundation Trust, Cambridge, United Kingdom (P.G.); the Institute of Allergology, Charité-Universitätsmedizin Berlin, Freie Universität Berlin and Humboldt-Universität zu Berlin, and Immunology and Allergology, Fraunhofer Institute for Translational Medicine and Pharmacology ITMP, Berlin (M.M.), and the Department of Children and Adolescents, University Hospital Frankfurt, Goethe University Frankfurt, Frankfurt (E.A.-P.) - all in Germany; the Department of Medicine, Campbelltown Hospital and Western Sydney University, Sydney (C.H.K.); University of Lille, U1286-INFINITE-Institute for Translational Research in Inflammation, F-59000, INSERM, and the Department of Internal Medicine and Clinical Immunology, CHU Lille, National Reference Center for Angioedema (CREAK), Lille (D.L.), and CREAK, Angioedema Center of Reference and Excellence (ACARE), Grenoble Alpes University Hospital, and the Translational Research in Autoimmunity and Inflammation Arm (T-RAIG), French National Center for Scientific Research (CNRS), Grenoble (L.B.) - all in France; Intellia Therapeutics, Cambridge, MA (D.M., J.S.B., M.Y.S., A.G., Y.X., A.M.A., D.L.); and the Department of Immunology, Auckland City Hospital, and the Department of Medicine, University of Auckland - both in Auckland, New Zealand (K.L., H.J.L.).<br /><searchLink fieldCode="AU" term="%22Petersen+RS%22">Petersen RS</searchLink>; From Amsterdam Cardiovascular Sciences, Amsterdam University Medical Center, University of Amsterdam, Amsterdam (D.M.C., R.S.P); Cambridge University Hospitals, NHS Foundation Trust, Cambridge, United Kingdom (P.G.); the Institute of Allergology, Charité-Universitätsmedizin Berlin, Freie Universität Berlin and Humboldt-Universität zu Berlin, and Immunology and Allergology, Fraunhofer Institute for Translational Medicine and Pharmacology ITMP, Berlin (M.M.), and the Department of Children and Adolescents, University Hospital Frankfurt, Goethe University Frankfurt, Frankfurt (E.A.-P.) - all in Germany; the Department of Medicine, Campbelltown Hospital and Western Sydney University, Sydney (C.H.K.); University of Lille, U1286-INFINITE-Institute for Translational Research in Inflammation, F-59000, INSERM, and the Department of Internal Medicine and Clinical Immunology, CHU Lille, National Reference Center for Angioedema (CREAK), Lille (D.L.), and CREAK, Angioedema Center of Reference and Excellence (ACARE), Grenoble Alpes University Hospital, and the Translational Research in Autoimmunity and Inflammation Arm (T-RAIG), French National Center for Scientific Research (CNRS), Grenoble (L.B.) - all in France; Intellia Therapeutics, Cambridge, MA (D.M., J.S.B., M.Y.S., A.G., Y.X., A.M.A., D.L.); and the Department of Immunology, Auckland City Hospital, and the Department of Medicine, University of Auckland - both in Auckland, New Zealand (K.L., H.J.L.).<br /><searchLink fieldCode="AU" term="%22Lindsay+K%22">Lindsay K</searchLink>; From Amsterdam Cardiovascular Sciences, Amsterdam University Medical Center, University of Amsterdam, Amsterdam (D.M.C., R.S.P); Cambridge University Hospitals, NHS Foundation Trust, Cambridge, United Kingdom (P.G.); the Institute of Allergology, Charité-Universitätsmedizin Berlin, Freie Universität Berlin and Humboldt-Universität zu Berlin, and Immunology and Allergology, Fraunhofer Institute for Translational Medicine and Pharmacology ITMP, Berlin (M.M.), and the Department of Children and Adolescents, University Hospital Frankfurt, Goethe University Frankfurt, Frankfurt (E.A.-P.) - all in Germany; the Department of Medicine, Campbelltown Hospital and Western Sydney University, Sydney (C.H.K.); University of Lille, U1286-INFINITE-Institute for Translational Research in Inflammation, F-59000, INSERM, and the Department of Internal Medicine and Clinical Immunology, CHU Lille, National Reference Center for Angioedema (CREAK), Lille (D.L.), and CREAK, Angioedema Center of Reference and Excellence (ACARE), Grenoble Alpes University Hospital, and the Translational Research in Autoimmunity and Inflammation Arm (T-RAIG), French National Center for Scientific Research (CNRS), Grenoble (L.B.) - all in France; Intellia Therapeutics, Cambridge, MA (D.M., J.S.B., M.Y.S., A.G., Y.X., A.M.A., D.L.); and the Department of Immunology, Auckland City Hospital, and the Department of Medicine, University of Auckland - both in Auckland, New Zealand (K.L., H.J.L.).<br /><searchLink fieldCode="AU" term="%22Aygören-Pürsün+E%22">Aygören-Pürsün E</searchLink>; From Amsterdam Cardiovascular Sciences, Amsterdam University Medical Center, University of Amsterdam, Amsterdam (D.M.C., R.S.P); Cambridge University Hospitals, NHS Foundation Trust, Cambridge, United Kingdom (P.G.); the Institute of Allergology, Charité-Universitätsmedizin Berlin, Freie Universität Berlin and Humboldt-Universität zu Berlin, and Immunology and Allergology, Fraunhofer Institute for Translational Medicine and Pharmacology ITMP, Berlin (M.M.), and the Department of Children and Adolescents, University Hospital Frankfurt, Goethe University Frankfurt, Frankfurt (E.A.-P.) - all in Germany; the Department of Medicine, Campbelltown Hospital and Western Sydney University, Sydney (C.H.K.); University of Lille, U1286-INFINITE-Institute for Translational Research in Inflammation, F-59000, INSERM, and the Department of Internal Medicine and Clinical Immunology, CHU Lille, National Reference Center for Angioedema (CREAK), Lille (D.L.), and CREAK, Angioedema Center of Reference and Excellence (ACARE), Grenoble Alpes University Hospital, and the Translational Research in Autoimmunity and Inflammation Arm (T-RAIG), French National Center for Scientific Research (CNRS), Grenoble (L.B.) - all in France; Intellia Therapeutics, Cambridge, MA (D.M., J.S.B., M.Y.S., A.G., Y.X., A.M.A., D.L.); and the Department of Immunology, Auckland City Hospital, and the Department of Medicine, University of Auckland - both in Auckland, New Zealand (K.L., H.J.L.).<br /><searchLink fieldCode="AU" term="%22Maag+D%22">Maag D</searchLink>; From Amsterdam Cardiovascular Sciences, Amsterdam University Medical Center, University of Amsterdam, Amsterdam (D.M.C., R.S.P); Cambridge University Hospitals, NHS Foundation Trust, Cambridge, United Kingdom (P.G.); the Institute of Allergology, Charité-Universitätsmedizin Berlin, Freie Universität Berlin and Humboldt-Universität zu Berlin, and Immunology and Allergology, Fraunhofer Institute for Translational Medicine and Pharmacology ITMP, Berlin (M.M.), and the Department of Children and Adolescents, University Hospital Frankfurt, Goethe University Frankfurt, Frankfurt (E.A.-P.) - all in Germany; the Department of Medicine, Campbelltown Hospital and Western Sydney University, Sydney (C.H.K.); University of Lille, U1286-INFINITE-Institute for Translational Research in Inflammation, F-59000, INSERM, and the Department of Internal Medicine and Clinical Immunology, CHU Lille, National Reference Center for Angioedema (CREAK), Lille (D.L.), and CREAK, Angioedema Center of Reference and Excellence (ACARE), Grenoble Alpes University Hospital, and the Translational Research in Autoimmunity and Inflammation Arm (T-RAIG), French National Center for Scientific Research (CNRS), Grenoble (L.B.) - all in France; Intellia Therapeutics, Cambridge, MA (D.M., J.S.B., M.Y.S., A.G., Y.X., A.M.A., D.L.); and the Department of Immunology, Auckland City Hospital, and the Department of Medicine, University of Auckland - both in Auckland, New Zealand (K.L., H.J.L.).<br /><searchLink fieldCode="AU" term="%22Butler+JS%22">Butler JS</searchLink>; From Amsterdam Cardiovascular Sciences, Amsterdam University Medical Center, University of Amsterdam, Amsterdam (D.M.C., R.S.P); Cambridge University Hospitals, NHS Foundation Trust, Cambridge, United Kingdom (P.G.); the Institute of Allergology, Charité-Universitätsmedizin Berlin, Freie Universität Berlin and Humboldt-Universität zu Berlin, and Immunology and Allergology, Fraunhofer Institute for Translational Medicine and Pharmacology ITMP, Berlin (M.M.), and the Department of Children and Adolescents, University Hospital Frankfurt, Goethe University Frankfurt, Frankfurt (E.A.-P.) - all in Germany; the Department of Medicine, Campbelltown Hospital and Western Sydney University, Sydney (C.H.K.); University of Lille, U1286-INFINITE-Institute for Translational Research in Inflammation, F-59000, INSERM, and the Department of Internal Medicine and Clinical Immunology, CHU Lille, National Reference Center for Angioedema (CREAK), Lille (D.L.), and CREAK, Angioedema Center of Reference and Excellence (ACARE), Grenoble Alpes University Hospital, and the Translational Research in Autoimmunity and Inflammation Arm (T-RAIG), French National Center for Scientific Research (CNRS), Grenoble (L.B.) - all in France; Intellia Therapeutics, Cambridge, MA (D.M., J.S.B., M.Y.S., A.G., Y.X., A.M.A., D.L.); and the Department of Immunology, Auckland City Hospital, and the Department of Medicine, University of Auckland - both in Auckland, New Zealand (K.L., H.J.L.).<br /><searchLink fieldCode="AU" term="%22Shah+MY%22">Shah MY</searchLink>; From Amsterdam Cardiovascular Sciences, Amsterdam University Medical Center, University of Amsterdam, Amsterdam (D.M.C., R.S.P); Cambridge University Hospitals, NHS Foundation Trust, Cambridge, United Kingdom (P.G.); the Institute of Allergology, Charité-Universitätsmedizin Berlin, Freie Universität Berlin and Humboldt-Universität zu Berlin, and Immunology and Allergology, Fraunhofer Institute for Translational Medicine and Pharmacology ITMP, Berlin (M.M.), and the Department of Children and Adolescents, University Hospital Frankfurt, Goethe University Frankfurt, Frankfurt (E.A.-P.) - all in Germany; the Department of Medicine, Campbelltown Hospital and Western Sydney University, Sydney (C.H.K.); University of Lille, U1286-INFINITE-Institute for Translational Research in Inflammation, F-59000, INSERM, and the Department of Internal Medicine and Clinical Immunology, CHU Lille, National Reference Center for Angioedema (CREAK), Lille (D.L.), and CREAK, Angioedema Center of Reference and Excellence (ACARE), Grenoble Alpes University Hospital, and the Translational Research in Autoimmunity and Inflammation Arm (T-RAIG), French National Center for Scientific Research (CNRS), Grenoble (L.B.) - all in France; Intellia Therapeutics, Cambridge, MA (D.M., J.S.B., M.Y.S., A.G., Y.X., A.M.A., D.L.); and the Department of Immunology, Auckland City Hospital, and the Department of Medicine, University of Auckland - both in Auckland, New Zealand (K.L., H.J.L.).<br /><searchLink fieldCode="AU" term="%22Golden+A%22">Golden A</searchLink>; From Amsterdam Cardiovascular Sciences, Amsterdam University Medical Center, University of Amsterdam, Amsterdam (D.M.C., R.S.P); Cambridge University Hospitals, NHS Foundation Trust, Cambridge, United Kingdom (P.G.); the Institute of Allergology, Charité-Universitätsmedizin Berlin, Freie Universität Berlin and Humboldt-Universität zu Berlin, and Immunology and Allergology, Fraunhofer Institute for Translational Medicine and Pharmacology ITMP, Berlin (M.M.), and the Department of Children and Adolescents, University Hospital Frankfurt, Goethe University Frankfurt, Frankfurt (E.A.-P.) - all in Germany; the Department of Medicine, Campbelltown Hospital and Western Sydney University, Sydney (C.H.K.); University of Lille, U1286-INFINITE-Institute for Translational Research in Inflammation, F-59000, INSERM, and the Department of Internal Medicine and Clinical Immunology, CHU Lille, National Reference Center for Angioedema (CREAK), Lille (D.L.), and CREAK, Angioedema Center of Reference and Excellence (ACARE), Grenoble Alpes University Hospital, and the Translational Research in Autoimmunity and Inflammation Arm (T-RAIG), French National Center for Scientific Research (CNRS), Grenoble (L.B.) - all in France; Intellia Therapeutics, Cambridge, MA (D.M., J.S.B., M.Y.S., A.G., Y.X., A.M.A., D.L.); and the Department of Immunology, Auckland City Hospital, and the Department of Medicine, University of Auckland - both in Auckland, New Zealand (K.L., H.J.L.).<br /><searchLink fieldCode="AU" term="%22Xu+Y%22">Xu Y</searchLink>; From Amsterdam Cardiovascular Sciences, Amsterdam University Medical Center, University of Amsterdam, Amsterdam (D.M.C., R.S.P); Cambridge University Hospitals, NHS Foundation Trust, Cambridge, United Kingdom (P.G.); the Institute of Allergology, Charité-Universitätsmedizin Berlin, Freie Universität Berlin and Humboldt-Universität zu Berlin, and Immunology and Allergology, Fraunhofer Institute for Translational Medicine and Pharmacology ITMP, Berlin (M.M.), and the Department of Children and Adolescents, University Hospital Frankfurt, Goethe University Frankfurt, Frankfurt (E.A.-P.) - all in Germany; the Department of Medicine, Campbelltown Hospital and Western Sydney University, Sydney (C.H.K.); University of Lille, U1286-INFINITE-Institute for Translational Research in Inflammation, F-59000, INSERM, and the Department of Internal Medicine and Clinical Immunology, CHU Lille, National Reference Center for Angioedema (CREAK), Lille (D.L.), and CREAK, Angioedema Center of Reference and Excellence (ACARE), Grenoble Alpes University Hospital, and the Translational Research in Autoimmunity and Inflammation Arm (T-RAIG), French National Center for Scientific Research (CNRS), Grenoble (L.B.) - all in France; Intellia Therapeutics, Cambridge, MA (D.M., J.S.B., M.Y.S., A.G., Y.X., A.M.A., D.L.); and the Department of Immunology, Auckland City Hospital, and the Department of Medicine, University of Auckland - both in Auckland, New Zealand (K.L., H.J.L.).<br /><searchLink fieldCode="AU" term="%22Abdelhady+AM%22">Abdelhady AM</searchLink>; From Amsterdam Cardiovascular Sciences, Amsterdam University Medical Center, University of Amsterdam, Amsterdam (D.M.C., R.S.P); Cambridge University Hospitals, NHS Foundation Trust, Cambridge, United Kingdom (P.G.); the Institute of Allergology, Charité-Universitätsmedizin Berlin, Freie Universität Berlin and Humboldt-Universität zu Berlin, and Immunology and Allergology, Fraunhofer Institute for Translational Medicine and Pharmacology ITMP, Berlin (M.M.), and the Department of Children and Adolescents, University Hospital Frankfurt, Goethe University Frankfurt, Frankfurt (E.A.-P.) - all in Germany; the Department of Medicine, Campbelltown Hospital and Western Sydney University, Sydney (C.H.K.); University of Lille, U1286-INFINITE-Institute for Translational Research in Inflammation, F-59000, INSERM, and the Department of Internal Medicine and Clinical Immunology, CHU Lille, National Reference Center for Angioedema (CREAK), Lille (D.L.), and CREAK, Angioedema Center of Reference and Excellence (ACARE), Grenoble Alpes University Hospital, and the Translational Research in Autoimmunity and Inflammation Arm (T-RAIG), French National Center for Scientific Research (CNRS), Grenoble (L.B.) - all in France; Intellia Therapeutics, Cambridge, MA (D.M., J.S.B., M.Y.S., A.G., Y.X., A.M.A., D.L.); and the Department of Immunology, Auckland City Hospital, and the Department of Medicine, University of Auckland - both in Auckland, New Zealand (K.L., H.J.L.).<br /><searchLink fieldCode="AU" term="%22Lebwohl+D%22">Lebwohl D</searchLink>; From Amsterdam Cardiovascular Sciences, Amsterdam University Medical Center, University of Amsterdam, Amsterdam (D.M.C., R.S.P); Cambridge University Hospitals, NHS Foundation Trust, Cambridge, United Kingdom (P.G.); the Institute of Allergology, Charité-Universitätsmedizin Berlin, Freie Universität Berlin and Humboldt-Universität zu Berlin, and Immunology and Allergology, Fraunhofer Institute for Translational Medicine and Pharmacology ITMP, Berlin (M.M.), and the Department of Children and Adolescents, University Hospital Frankfurt, Goethe University Frankfurt, Frankfurt (E.A.-P.) - all in Germany; the Department of Medicine, Campbelltown Hospital and Western Sydney University, Sydney (C.H.K.); University of Lille, U1286-INFINITE-Institute for Translational Research in Inflammation, F-59000, INSERM, and the Department of Internal Medicine and Clinical Immunology, CHU Lille, National Reference Center for Angioedema (CREAK), Lille (D.L.), and CREAK, Angioedema Center of Reference and Excellence (ACARE), Grenoble Alpes University Hospital, and the Translational Research in Autoimmunity and Inflammation Arm (T-RAIG), French National Center for Scientific Research (CNRS), Grenoble (L.B.) - all in France; Intellia Therapeutics, Cambridge, MA (D.M., J.S.B., M.Y.S., A.G., Y.X., A.M.A., D.L.); and the Department of Immunology, Auckland City Hospital, and the Department of Medicine, University of Auckland - both in Auckland, New Zealand (K.L., H.J.L.).<br /><searchLink fieldCode="AU" term="%22Longhurst+HJ%22">Longhurst HJ</searchLink>; From Amsterdam Cardiovascular Sciences, Amsterdam University Medical Center, University of Amsterdam, Amsterdam (D.M.C., R.S.P); Cambridge University Hospitals, NHS Foundation Trust, Cambridge, United Kingdom (P.G.); the Institute of Allergology, Charité-Universitätsmedizin Berlin, Freie Universität Berlin and Humboldt-Universität zu Berlin, and Immunology and Allergology, Fraunhofer Institute for Translational Medicine and Pharmacology ITMP, Berlin (M.M.), and the Department of Children and Adolescents, University Hospital Frankfurt, Goethe University Frankfurt, Frankfurt (E.A.-P.) - all in Germany; the Department of Medicine, Campbelltown Hospital and Western Sydney University, Sydney (C.H.K.); University of Lille, U1286-INFINITE-Institute for Translational Research in Inflammation, F-59000, INSERM, and the Department of Internal Medicine and Clinical Immunology, CHU Lille, National Reference Center for Angioedema (CREAK), Lille (D.L.), and CREAK, Angioedema Center of Reference and Excellence (ACARE), Grenoble Alpes University Hospital, and the Translational Research in Autoimmunity and Inflammation Arm (T-RAIG), French National Center for Scientific Research (CNRS), Grenoble (L.B.) - all in France; Intellia Therapeutics, Cambridge, MA (D.M., J.S.B., M.Y.S., A.G., Y.X., A.M.A., D.L.); and the Department of Immunology, Auckland City Hospital, and the Department of Medicine, University of Auckland - both in Auckland, New Zealand (K.L., H.J.L.). – Name: TitleSource Label: Source Group: Src Data: <searchLink fieldCode="JN" term="%220255562%22">The New England journal of medicine</searchLink> [N Engl J Med] 2025 Jan 30; Vol. 392 (5), pp. 458-467. <i>Date of Electronic Publication: </i>2024 Oct 24. – Name: TypePub Label: Publication Type Group: TypPub Data: Clinical Trial, Phase II; Journal Article; Multicenter Study; Randomized Controlled Trial – 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/NEJMoa2405734 Languages: – Code: eng Text: English PhysicalDescription: Pagination: StartPage: 458 Titles: – TitleFull: CRISPR-Based Therapy for Hereditary Angioedema. Type: main BibRelationships: HasContributorRelationships: – PersonEntity: Name: NameFull: Cohn DM – PersonEntity: Name: NameFull: Gurugama P – PersonEntity: Name: NameFull: Magerl M – PersonEntity: Name: NameFull: Katelaris CH – PersonEntity: Name: NameFull: Launay D – PersonEntity: Name: NameFull: Bouillet L – PersonEntity: Name: NameFull: Petersen RS – PersonEntity: Name: NameFull: Lindsay K – PersonEntity: Name: NameFull: Aygören-Pürsün E – PersonEntity: Name: NameFull: Maag D – PersonEntity: Name: NameFull: Butler JS – PersonEntity: Name: NameFull: Shah MY – PersonEntity: Name: NameFull: Golden A – PersonEntity: Name: NameFull: Xu Y – PersonEntity: Name: NameFull: Abdelhady AM – PersonEntity: Name: NameFull: Lebwohl D – PersonEntity: Name: NameFull: Longhurst HJ IsPartOfRelationships: – BibEntity: Dates: – D: 30 M: 01 Text: 2025 Jan 30 Type: published Y: 2025 Identifiers: – Type: issn-electronic Value: 1533-4406 Numbering: – Type: volume Value: 392 – Type: issue Value: 5 Titles: – TitleFull: The New England journal of medicine Type: main |
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