Article
Allele-selective disruption of pathogenic VWF variants in type 2 von Willebrand disease using CRISPR/Cas9.
Blood advances - 10 Mar 2026
Bär Isabel, Groten Stijn A, Barraclough Alastair, Bürgisser Petra E, van Kwawegen Calvin, Lenting Peter J, van Moort Iris, Eikenboom Jeroen C J, Leebeek Frank W G, Voorberg Jan, van den Biggelaar Maartje, Bierings Ruben
Abstract excerpt
ABSTRACT: In contrast to major innovations in treating severe hemophilia, the treatment of severe von Willebrand disease (VWD) remains limited to intravenous infusion of von Willebrand factor (VWF) concentrates. To date, no gene therapy-based approaches for the treatment of VWD have been developed, largely owing to the disease's heterogeneous mutational landscape and the challenge of specifically targeting VWF...
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