NEJM：体内CRISPR基因编辑疗法可有效治疗血管性水肿

https://www.ebiotrade.com/newsf/2024-2/20240202104435718.htm

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

  Hilary J Longhurst,Karen Lindsay,Remy S Petersen,Lauré M Fijen,Padmalal Gurugama,David Maag,James S Butler,Mrinal Y Shah,Adele Golden,Yuanxin Xu,Carri Boiselle,Joseph D Vogel,Ahmed M Abdelhady,Michael L Maitland,Mark D McKee,Jessica Seitzer,Bo W Han,Samantha Soukamneuth,John Leonard,Laura Sepp-Lorenzino,Eliana D Clark,David Lebwohl,Danny M Cohn

  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.).

  N Engl J Med (P 1533-4406 E 0028-4793) 2024 年 390 卷 5 期 432-441 页

  PMID：38294975 相似文献

  文摘 DOI链接 Atypon

• 2. Clinical Progress in Hepatic Targeting for Novel Prophylactic Therapies in Hereditary Angioedema.

  Marc A Riedl,Laura Bordone,Alexey Revenko,Kenneth B Newman,Danny M Cohn

  Division of Allergy and Immunology, University of California, San Diego, La Jolla, Calif. Electronic address: mriedl@health.ucsd.edu.

  mriedl@health.ucsd.edu

  J Allergy Clin Immunol Pract (P 2213-2201 E ) H指数：0 2023 年

  PMID：38142864 相似文献

  文摘 DOI链接 ClinicalKeyElsevier Science

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