Sustained high expression of human FVII following AAV8-mediated gene delivery in mice

Sustained high expression of human FVII following AAV8-mediated gene delivery in mice

Factor VII (FVII) deficiency is a rare bleeding disorder with a prevalence of approximately 1:300,000–500,000 individuals. We explored whether adeno-associated virus (AAV)-mediated gene therapy can achieve durable and functional expression of human FVII (hFVII) in vivo. Wild-type hFVII (hFVIIwt) and...

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Main Authors: Andrea D’Amico, Bas Blits, Joëlle van den Herik, Fred de Winter, Paul H.P. Kaijen, Barbara Hobo, Alexander Henrik van Asbeck, Joost Verhaagen, David G.M. Mosmuller, Jan Voorberg
Format: Article
Language:English
Published: Elsevier 2025-09-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
gene therapy
FVII deficiency
rare bleeding disorders
liver-directed gene therapy
augmentation therapy
AAV8
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050125001184
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http://www.sciencedirect.com/science/article/pii/S2329050125001184

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