High and long-term von Willebrand factor expression after Sleeping Beauty transposon-mediated gene therapy in a mouse model of severe von Willebrand disease

Author:

Portier I.1,Vanhoorelbeke K.1,Verhenne S.1,Pareyn I.1,Vandeputte N.1,Deckmyn H.1,Goldenberg D. S.2,Samal H. B.3,Singh M.3,Ivics Z.4,Izsvák Z.3,De Meyer S. F.1

Affiliation:

1. Laboratory for Thrombosis Research; KU Leuven Campus Kulak Kortrijk; Kortrijk Belgium

2. The Goldyne Savad Institute of Gene Therapy; Hadassah-Hebrew University Medical Center; Jerusalem Israel

3. Max Delbrück Center for Molecular Medicine in the Helmholtz Association; Berlin Germany

4. Division of Medical Biotechnology; Paul Ehrlich Institute; Langen Germany

Funder

Bayer Hemophilia Awards Program

Fonds Wetenschappelijk Onderzoek

European Research projects on Rare Disease

TRANSPOSMART

Flanders Agency for Innovation and Entrepreneurship

Kamea Scientific Foundation

E-RARE TRANSPOSMART program

Deutsche Forschungsgemeinschaft

Robert H. Benson Living Trust

Jay Raskin Foundation

Selma Kron Foundation

Publisher

Wiley

Subject

Hematology

Cited by 21 articles. 订阅此论文施引文献 订阅此论文施引文献,注册后可以免费订阅5篇论文的施引文献,订阅后可以查看论文全部施引文献

1. von Willebrand disease;Nature Reviews Disease Primers;2024-07-25

2. Diagnosis and treatment of von Willebrand disease in 2024 and beyond;Haemophilia;2024-03-13

3. Murine models of hemostasis;HEMATOLOGIE;2023

4. Prolonged activity of the transposase helper may raise safety concerns during DNA transposon-based gene therapy;Molecular Therapy - Methods & Clinical Development;2023-06

5. Toward gene therapy for congenital thrombotic thrombocytopenic purpura;Journal of Thrombosis and Haemostasis;2023-05

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