Blood advances
Allele-selective Disruption of Pathogenic VWF Variants in Type 2 Von Willebrand Disease using CRISPR/Cas9.
Isabel Bär, Stijn A Groten, Alastair Barraclough, Petra E Bürgisser, Calvin van Kwawegen, Peter J Lenting, Iris van Moort, Jeroen C J Eikenboom, Frank W G Leebeek, Jan Voorberg, Maartje van den Biggelaar, Ruben Bierings
Published: 202510.1182/bloodadvances.2025018760
Abstract
In contrast to major innovations in treating severe hemophilia, 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 treatm…
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