Correcting dominant-negative von Willebrand disease

Abstract

In this issue of the Journal of Thrombosis and Haemostasis, Campioni et al report correction of dominant-negative von Willebrand disease (VWD) in vivo for the first time. In particular, they demonstrate that targeted inhibition of a mutant VWF allele can significantly improve bleeding phenotype in a Type 2A VWD murine model. Collectively, these data move us another step closer to developing personalized approaches for the treatment of VWD patients that extend beyond traditional von Willebrand factor (VWF) infusion therapy