Article
Impact of allele-selective silencing of von Willebrand factor in mice based on a single nucleotide allelic difference in von Willebrand factor.
Thrombosis research - 1 Apr 2024
Jongejan Yvonne K, Linthorst Noa A, Schrader Echeverri Elisa, Laan Sebastiaan N J, Dirven Richard J, Dahlman James E, van Vlijmen Bart J M, Denis Cécile V, Eikenboom Jeroen C J
Abstract excerpt
INTRODUCTION: Von Willebrand factor (VWF) plays a pathophysiological role in hemostatic disorders. Partial inhibition of the VWF gene through small interfering RNA (siRNA)-mediated allele-selective silencing could be a promising therapeutic strategy. For von Willebrand disease, allele-selectively inhibiting dominant-negative VWF-alleles might ameliorate the phenotype. For thrombotic disorders, partial VWF...
Read the complete abstract on PubMedTopics
Share this publication in a Topic to start or enrich a Post.
