Article
Small interfering RNA-mediated allele-selective silencing of von Willebrand factor in vitro and in vivo.
Blood advances - 24 Oct 2023
Jongejan Yvonne K, Schrader Echeverri Elisa, Dirven Richard J, Paunovska Kalina, Linthorst Noa A, de Jong Annika, Wellershoff Johannes C, van der Gouw Kim D, van Vlijmen Bart J M, Dahlman James E, Eikenboom Jeroen C J
Abstract excerpt
An imbalance in von Willebrand factor (VWF) may either lead to bleeding (von Willebrand disease, VWD) or thrombosis. Both disorders have shortcomings in the currently available treatments. VWF itself could be a potential therapeutic target because of its role in both bleeding and thrombosis. Inhibiting VWF gene expression through allele-selective silencing of VWF with small interfering RNAs (siRNAs) could be a...
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