BOEC and iPSC models for von Willebrand disease biology and in vitro VWF allele silencing

Open-label trial of sirna in von Willebrand disease (VWD) (3 arms), sponsored by Leiden University Medical Center. Primary endpoints: Optimized isolation and characterization of BOECs, iPSC-ECs, and iPSC-MKs, Identification of new pathophysiologic mechanisms of von Willebrand disease and 1 more. Started May 2016; status: active, not recruiting.