CRISPR-Cas9 In Vivo Gene Editing of KLKB1 for Hereditary Angioedema.

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Title: CRISPR-Cas9 In Vivo Gene Editing of KLKB1 for Hereditary Angioedema.
Authors: Longhurst HJ; From Te Toka Tumai, Department of Immunology, Auckland City Hospital (H.J.L., K.L.), and the Department of Medicine, University of Auckland (H.J.L.) - both in Auckland, New Zealand; the Department of Vascular Medicine, Amsterdam Cardiovascular Sciences, Amsterdam University Medical Centers, University of Amsterdam, Amsterdam (R.S.P., L.M.F., D.M.C.); Cambridge University Hospitals, NHS Foundation Trust, Cambridge, United Kingdom (P.G.); and Intellia Therapeutics, Cambridge, MA (D.M., J.S.B., M.Y.S., A.G., Y.X., C.B., J.D.V., A.M.A., M.L.M., M.D.M., J.S., B.W.H., S.S., J.L., L.S.-L., E.D.C., D.L.)., Lindsay K; From Te Toka Tumai, Department of Immunology, Auckland City Hospital (H.J.L., K.L.), and the Department of Medicine, University of Auckland (H.J.L.) - both in Auckland, New Zealand; the Department of Vascular Medicine, Amsterdam Cardiovascular Sciences, Amsterdam University Medical Centers, University of Amsterdam, Amsterdam (R.S.P., L.M.F., D.M.C.); Cambridge University Hospitals, NHS Foundation Trust, Cambridge, United Kingdom (P.G.); and Intellia Therapeutics, Cambridge, MA (D.M., J.S.B., M.Y.S., A.G., Y.X., C.B., J.D.V., A.M.A., M.L.M., M.D.M., J.S., B.W.H., S.S., J.L., L.S.-L., E.D.C., D.L.)., Petersen RS; From Te Toka Tumai, Department of Immunology, Auckland City Hospital (H.J.L., K.L.), and the Department of Medicine, University of Auckland (H.J.L.) - both in Auckland, New Zealand; the Department of Vascular Medicine, Amsterdam Cardiovascular Sciences, Amsterdam University Medical Centers, University of Amsterdam, Amsterdam (R.S.P., L.M.F., D.M.C.); Cambridge University Hospitals, NHS Foundation Trust, Cambridge, United Kingdom (P.G.); and Intellia Therapeutics, Cambridge, MA (D.M., J.S.B., M.Y.S., A.G., Y.X., C.B., J.D.V., A.M.A., M.L.M., M.D.M., J.S., B.W.H., S.S., J.L., L.S.-L., E.D.C., D.L.)., Fijen LM; From Te Toka Tumai, Department of Immunology, Auckland City Hospital (H.J.L., K.L.), and the Department of Medicine, University of Auckland (H.J.L.) - both in Auckland, New Zealand; the Department of Vascular Medicine, Amsterdam Cardiovascular Sciences, Amsterdam University Medical Centers, University of Amsterdam, Amsterdam (R.S.P., L.M.F., D.M.C.); Cambridge University Hospitals, NHS Foundation Trust, Cambridge, United Kingdom (P.G.); and Intellia Therapeutics, Cambridge, MA (D.M., J.S.B., M.Y.S., A.G., Y.X., C.B., J.D.V., A.M.A., M.L.M., M.D.M., J.S., B.W.H., S.S., J.L., L.S.-L., E.D.C., D.L.)., Gurugama P; From Te Toka Tumai, Department of Immunology, Auckland City Hospital (H.J.L., K.L.), and the Department of Medicine, University of Auckland (H.J.L.) - both in Auckland, New Zealand; the Department of Vascular Medicine, Amsterdam Cardiovascular Sciences, Amsterdam University Medical Centers, University of Amsterdam, Amsterdam (R.S.P., L.M.F., D.M.C.); Cambridge University Hospitals, NHS Foundation Trust, Cambridge, United Kingdom (P.G.); and Intellia Therapeutics, Cambridge, MA (D.M., J.S.B., M.Y.S., A.G., Y.X., C.B., J.D.V., A.M.A., M.L.M., M.D.M., J.S., B.W.H., S.S., J.L., L.S.-L., E.D.C., D.L.)., Maag D; From Te Toka Tumai, Department of Immunology, Auckland City Hospital (H.J.L., K.L.), and the Department of Medicine, University of Auckland (H.J.L.) - both in Auckland, New Zealand; the Department of Vascular Medicine, Amsterdam Cardiovascular Sciences, Amsterdam University Medical Centers, University of Amsterdam, Amsterdam (R.S.P., L.M.F., D.M.C.); Cambridge University Hospitals, NHS Foundation Trust, Cambridge, United Kingdom (P.G.); and Intellia Therapeutics, Cambridge, MA (D.M., J.S.B., M.Y.S., A.G., Y.X., C.B., J.D.V., A.M.A., M.L.M., M.D.M., J.S., B.W.H., S.S., J.L., L.S.-L., E.D.C., D.L.)., Butler JS; From Te Toka Tumai, Department of Immunology, Auckland City Hospital (H.J.L., K.L.), and the Department of Medicine, University of Auckland (H.J.L.) - both in Auckland, New Zealand; the Department of Vascular Medicine, Amsterdam Cardiovascular Sciences, Amsterdam University Medical Centers, University of Amsterdam, Amsterdam (R.S.P., L.M.F., D.M.C.); Cambridge University Hospitals, NHS Foundation Trust, Cambridge, United Kingdom (P.G.); and Intellia Therapeutics, Cambridge, MA (D.M., J.S.B., M.Y.S., A.G., Y.X., C.B., J.D.V., A.M.A., M.L.M., M.D.M., J.S., B.W.H., S.S., J.L., L.S.-L., E.D.C., D.L.)., Shah MY; From Te Toka Tumai, Department of Immunology, Auckland City Hospital (H.J.L., K.L.), and the Department of Medicine, University of Auckland (H.J.L.) - both in Auckland, New Zealand; the Department of Vascular Medicine, Amsterdam Cardiovascular Sciences, Amsterdam University Medical Centers, University of Amsterdam, Amsterdam (R.S.P., L.M.F., D.M.C.); Cambridge University Hospitals, NHS Foundation Trust, Cambridge, United Kingdom (P.G.); and Intellia Therapeutics, Cambridge, MA (D.M., J.S.B., M.Y.S., A.G., Y.X., C.B., J.D.V., A.M.A., M.L.M., M.D.M., J.S., B.W.H., S.S., J.L., L.S.-L., E.D.C., D.L.)., Golden A; From Te Toka Tumai, Department of Immunology, Auckland City Hospital (H.J.L., K.L.), and the Department of Medicine, University of Auckland (H.J.L.) - both in Auckland, New Zealand; the Department of Vascular Medicine, Amsterdam Cardiovascular Sciences, Amsterdam University Medical Centers, University of Amsterdam, Amsterdam (R.S.P., L.M.F., D.M.C.); Cambridge University Hospitals, NHS Foundation Trust, Cambridge, United Kingdom (P.G.); and Intellia Therapeutics, Cambridge, MA (D.M., J.S.B., M.Y.S., A.G., Y.X., C.B., J.D.V., A.M.A., M.L.M., M.D.M., J.S., B.W.H., S.S., J.L., L.S.-L., E.D.C., D.L.)., Xu Y; From Te Toka Tumai, Department of Immunology, Auckland City Hospital (H.J.L., K.L.), and the Department of Medicine, University of Auckland (H.J.L.) - both in Auckland, New Zealand; the Department of Vascular Medicine, Amsterdam Cardiovascular Sciences, Amsterdam University Medical Centers, University of Amsterdam, Amsterdam (R.S.P., L.M.F., D.M.C.); Cambridge University Hospitals, NHS Foundation Trust, Cambridge, United Kingdom (P.G.); and Intellia Therapeutics, Cambridge, MA (D.M., J.S.B., M.Y.S., A.G., Y.X., C.B., J.D.V., A.M.A., M.L.M., M.D.M., J.S., B.W.H., S.S., J.L., L.S.-L., E.D.C., D.L.)., Boiselle C; From Te Toka Tumai, Department of Immunology, Auckland City Hospital (H.J.L., K.L.), and the Department of Medicine, University of Auckland (H.J.L.) - both in Auckland, New Zealand; the Department of Vascular Medicine, Amsterdam Cardiovascular Sciences, Amsterdam University Medical Centers, University of Amsterdam, Amsterdam (R.S.P., L.M.F., D.M.C.); Cambridge University Hospitals, NHS Foundation Trust, Cambridge, United Kingdom (P.G.); and Intellia Therapeutics, Cambridge, MA (D.M., J.S.B., M.Y.S., A.G., Y.X., C.B., J.D.V., A.M.A., M.L.M., M.D.M., J.S., B.W.H., S.S., J.L., L.S.-L., E.D.C., D.L.)., Vogel JD; From Te Toka Tumai, Department of Immunology, Auckland City Hospital (H.J.L., K.L.), and the Department of Medicine, University of Auckland (H.J.L.) - both in Auckland, New Zealand; the Department of Vascular Medicine, Amsterdam Cardiovascular Sciences, Amsterdam University Medical Centers, University of Amsterdam, Amsterdam (R.S.P., L.M.F., D.M.C.); Cambridge University Hospitals, NHS Foundation Trust, Cambridge, United Kingdom (P.G.); and Intellia Therapeutics, Cambridge, MA (D.M., J.S.B., M.Y.S., A.G., Y.X., C.B., J.D.V., A.M.A., M.L.M., M.D.M., J.S., B.W.H., S.S., J.L., L.S.-L., E.D.C., D.L.)., Abdelhady AM; From Te Toka Tumai, Department of Immunology, Auckland City Hospital (H.J.L., K.L.), and the Department of Medicine, University of Auckland (H.J.L.) - both in Auckland, New Zealand; the Department of Vascular Medicine, Amsterdam Cardiovascular Sciences, Amsterdam University Medical Centers, University of Amsterdam, Amsterdam (R.S.P., L.M.F., D.M.C.); Cambridge University Hospitals, NHS Foundation Trust, Cambridge, United Kingdom (P.G.); and Intellia Therapeutics, Cambridge, MA (D.M., J.S.B., M.Y.S., A.G., Y.X., C.B., J.D.V., A.M.A., M.L.M., M.D.M., J.S., B.W.H., S.S., J.L., L.S.-L., E.D.C., D.L.)., Maitland ML; From Te Toka Tumai, Department of Immunology, Auckland City Hospital (H.J.L., K.L.), and the Department of Medicine, University of Auckland (H.J.L.) - both in Auckland, New Zealand; the Department of Vascular Medicine, Amsterdam Cardiovascular Sciences, Amsterdam University Medical Centers, University of Amsterdam, Amsterdam (R.S.P., L.M.F., D.M.C.); Cambridge University Hospitals, NHS Foundation Trust, Cambridge, United Kingdom (P.G.); and Intellia Therapeutics, Cambridge, MA (D.M., J.S.B., M.Y.S., A.G., Y.X., C.B., J.D.V., A.M.A., M.L.M., M.D.M., J.S., B.W.H., S.S., J.L., L.S.-L., E.D.C., D.L.)., McKee MD; From Te Toka Tumai, Department of Immunology, Auckland City Hospital (H.J.L., K.L.), and the Department of Medicine, University of Auckland (H.J.L.) - both in Auckland, New Zealand; the Department of Vascular Medicine, Amsterdam Cardiovascular Sciences, Amsterdam University Medical Centers, University of Amsterdam, Amsterdam (R.S.P., L.M.F., D.M.C.); Cambridge University Hospitals, NHS Foundation Trust, Cambridge, United Kingdom (P.G.); and Intellia Therapeutics, Cambridge, MA (D.M., J.S.B., M.Y.S., A.G., Y.X., C.B., J.D.V., A.M.A., M.L.M., M.D.M., J.S., B.W.H., S.S., J.L., L.S.-L., E.D.C., D.L.)., Seitzer J; From Te Toka Tumai, Department of Immunology, Auckland City Hospital (H.J.L., K.L.), and the Department of Medicine, University of Auckland (H.J.L.) - both in Auckland, New Zealand; the Department of Vascular Medicine, Amsterdam Cardiovascular Sciences, Amsterdam University Medical Centers, University of Amsterdam, Amsterdam (R.S.P., L.M.F., D.M.C.); Cambridge University Hospitals, NHS Foundation Trust, Cambridge, United Kingdom (P.G.); and Intellia Therapeutics, Cambridge, MA (D.M., J.S.B., M.Y.S., A.G., Y.X., C.B., J.D.V., A.M.A., M.L.M., M.D.M., J.S., B.W.H., S.S., J.L., L.S.-L., E.D.C., D.L.)., Han BW; From Te Toka Tumai, Department of Immunology, Auckland City Hospital (H.J.L., K.L.), and the Department of Medicine, University of Auckland (H.J.L.) - both in Auckland, New Zealand; the Department of Vascular Medicine, Amsterdam Cardiovascular Sciences, Amsterdam University Medical Centers, University of Amsterdam, Amsterdam (R.S.P., L.M.F., D.M.C.); Cambridge University Hospitals, NHS Foundation Trust, Cambridge, United Kingdom (P.G.); and Intellia Therapeutics, Cambridge, MA (D.M., J.S.B., M.Y.S., A.G., Y.X., C.B., J.D.V., A.M.A., M.L.M., M.D.M., J.S., B.W.H., S.S., J.L., L.S.-L., E.D.C., D.L.)., Soukamneuth S; From Te Toka Tumai, Department of Immunology, Auckland City Hospital (H.J.L., K.L.), and the Department of Medicine, University of Auckland (H.J.L.) - both in Auckland, New Zealand; the Department of Vascular Medicine, Amsterdam Cardiovascular Sciences, Amsterdam University Medical Centers, University of Amsterdam, Amsterdam (R.S.P., L.M.F., D.M.C.); Cambridge University Hospitals, NHS Foundation Trust, Cambridge, United Kingdom (P.G.); and Intellia Therapeutics, Cambridge, MA (D.M., J.S.B., M.Y.S., A.G., Y.X., C.B., J.D.V., A.M.A., M.L.M., M.D.M., J.S., B.W.H., S.S., J.L., L.S.-L., E.D.C., D.L.)., Leonard J; From Te Toka Tumai, Department of Immunology, Auckland City Hospital (H.J.L., K.L.), and the Department of Medicine, University of Auckland (H.J.L.) - both in Auckland, New Zealand; the Department of Vascular Medicine, Amsterdam Cardiovascular Sciences, Amsterdam University Medical Centers, University of Amsterdam, Amsterdam (R.S.P., L.M.F., D.M.C.); Cambridge University Hospitals, NHS Foundation Trust, Cambridge, United Kingdom (P.G.); and Intellia Therapeutics, Cambridge, MA (D.M., J.S.B., M.Y.S., A.G., Y.X., C.B., J.D.V., A.M.A., M.L.M., M.D.M., J.S., B.W.H., S.S., J.L., L.S.-L., E.D.C., D.L.)., Sepp-Lorenzino L; From Te Toka Tumai, Department of Immunology, Auckland City Hospital (H.J.L., K.L.), and the Department of Medicine, University of Auckland (H.J.L.) - both in Auckland, New Zealand; the Department of Vascular Medicine, Amsterdam Cardiovascular Sciences, Amsterdam University Medical Centers, University of Amsterdam, Amsterdam (R.S.P., L.M.F., D.M.C.); Cambridge University Hospitals, NHS Foundation Trust, Cambridge, United Kingdom (P.G.); and Intellia Therapeutics, Cambridge, MA (D.M., J.S.B., M.Y.S., A.G., Y.X., C.B., J.D.V., A.M.A., M.L.M., M.D.M., J.S., B.W.H., S.S., J.L., L.S.-L., E.D.C., D.L.)., Clark ED; From Te Toka Tumai, Department of Immunology, Auckland City Hospital (H.J.L., K.L.), and the Department of Medicine, University of Auckland (H.J.L.) - both in Auckland, New Zealand; the Department of Vascular Medicine, Amsterdam Cardiovascular Sciences, Amsterdam University Medical Centers, University of Amsterdam, Amsterdam (R.S.P., L.M.F., D.M.C.); Cambridge University Hospitals, NHS Foundation Trust, Cambridge, United Kingdom (P.G.); and Intellia Therapeutics, Cambridge, MA (D.M., J.S.B., M.Y.S., A.G., Y.X., C.B., J.D.V., A.M.A., M.L.M., M.D.M., J.S., B.W.H., S.S., J.L., L.S.-L., E.D.C., D.L.)., Lebwohl D; From Te Toka Tumai, Department of Immunology, Auckland City Hospital (H.J.L., K.L.), and the Department of Medicine, University of Auckland (H.J.L.) - both in Auckland, New Zealand; the Department of Vascular Medicine, Amsterdam Cardiovascular Sciences, Amsterdam University Medical Centers, University of Amsterdam, Amsterdam (R.S.P., L.M.F., D.M.C.); Cambridge University Hospitals, NHS Foundation Trust, Cambridge, United Kingdom (P.G.); and Intellia Therapeutics, Cambridge, MA (D.M., J.S.B., M.Y.S., A.G., Y.X., C.B., J.D.V., A.M.A., M.L.M., M.D.M., J.S., B.W.H., S.S., J.L., L.S.-L., E.D.C., D.L.)., Cohn DM; From Te Toka Tumai, Department of Immunology, Auckland City Hospital (H.J.L., K.L.), and the Department of Medicine, University of Auckland (H.J.L.) - both in Auckland, New Zealand; the Department of Vascular Medicine, Amsterdam Cardiovascular Sciences, Amsterdam University Medical Centers, University of Amsterdam, Amsterdam (R.S.P., L.M.F., D.M.C.); Cambridge University Hospitals, NHS Foundation Trust, Cambridge, United Kingdom (P.G.); and Intellia Therapeutics, Cambridge, MA (D.M., J.S.B., M.Y.S., A.G., Y.X., C.B., J.D.V., A.M.A., M.L.M., M.D.M., J.S., B.W.H., S.S., J.L., L.S.-L., E.D.C., D.L.).
Source: The New England journal of medicine [N Engl J Med] 2024 Feb 01; Vol. 390 (5), pp. 432-441.
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 Cited Medium: Internet ISSN: 1533-4406 (Electronic) Linking ISSN: 00284793 NLM ISO Abbreviation: N Engl J Med Subsets: MEDLINE
Database: MEDLINE Ultimate
Description
ISSN:1533-4406
DOI:10.1056/NEJMoa2309149