CRISPR-Based Therapy for Hereditary Angioedema

Danny M. Cohn; Padmalal Gurugama; Markus Magerl; Constance H. Katelaris; David Launay; Laurence Bouillet; Remy S. Petersen; Karen Lindsay; Emel Aygören-Pürsün; David Maag; James S. Butler; Mrinal Y. Shah; Adele Golden; Yuanxin Xu; Ahmed M. Abdelhady; David Lebwohl; Hilary J. Longhurst

doi:10.1056/nejmoa2405734

CRISPR-Based Therapy for Hereditary Angioedema

D Danny M. Cohn (Department of Vascular Medicine, Amsterdam Cardiovascular Sciences, Amsterdam University Medical Centers, University of Amsterdam, Amsterdam) P Padmalal Gurugama (Cambridge University Hospitals, NHS Foundation Trust, Cambridge, United Kingdom) M Markus Magerl (Institute of Allergology, Charité-Universitätsmedizin Berlin, Corporate Member of Freie Universität Berlin and Humboldt-Universität zu Berlin, Berlin) C Constance H. Katelaris (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), Gr...) D David Launay (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), Gr...) L Laurence Bouillet (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), Gr...) R Remy S. Petersen (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), Gr...) K Karen Lindsay (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), Gr...) E Emel Aygören-Pürsün (Department of Children and Adolescents, University Hospital Frankfurt, Goethe University Frankfurt, Frankfurt, Germany) D David Maag (Intellia Therapeutics, Cambridge, MA) J James S. Butler (Intellia Therapeutics, Cambridge, MA) M Mrinal Y. Shah (Intellia Therapeutics, Cambridge, MA) A Adele Golden (Intellia Therapeutics, Cambridge, MA) Y Yuanxin Xu (Intellia Therapeutics, Cambridge, MA) A Ahmed M. Abdelhady (Intellia Therapeutics, Cambridge, MA) D David Lebwohl (Intellia Therapeutics, Cambridge, MA) H Hilary J. Longhurst (Te Toka Tumai, Department of Immunology, Auckland City Hospital, Auckland, New Zealand)

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Article Details

Journal New England Journal of Medicine

Volume / Issue Vol. 392, Issue 5

Published January 30, 2025

Pages 458-467

DOI 10.1056/nejmoa2405734

ISSN 0028-4793

Publisher Massachusetts Medical Society

Journal Info

New England Journal of Medicine

Massachusetts Medical Society

ISSN: 0028-4793 Social Sciences

Authors (17)

D

Danny M. Cohn

Department of Vascular Medicine, Amsterdam Cardiovascular Sciences, Amsterdam University Medical Centers, University of Amsterdam, Amsterdam

P

Padmalal Gurugama

Cambridge University Hospitals, NHS Foundation Trust, Cambridge, United Kingdom

M

Markus Magerl

Institute of Allergology, Charité-Universitätsmedizin Berlin, Corporate Member of Freie Universität Berlin and Humboldt-Universität zu Berlin, Berlin

C

Constance H. Katelaris

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), Gr...

D

David Launay

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), Gr...

L

Laurence Bouillet

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), Gr...

R

Remy S. Petersen

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), Gr...

K

Karen Lindsay

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), Gr...

E

Emel Aygören-Pürsün

Department of Children and Adolescents, University Hospital Frankfurt, Goethe University Frankfurt, Frankfurt, Germany

D

David Maag