hart_vol

RNA-targeted therapies for von Willebrand disease

Current therapies for patients with von Willebrand Disease (VWD) include the use of desmopressin (DDAVP) or the administration of von Willebrand Factor (VWF) concentrates. In this project we aim to investigate whether we can correct the phenotype of VWF mutations by the use of small interfering RNAs (siRNAs), antisense oligonucleotides (AONs) and CRISPR/cas. These corrections will be studied in blood outgrowth endothelial cells (BOECs), which are endothelial progenitor cells circulating in low numbers in peripheral blood.