Towards long-term correction of severe von Willebrand disease via non-viral Sleeping Beauty transposon gene therapy. KU Leuven
Von Willebrand disease (VWD) is the most common inherited bleeding disorder, caused by defects in the von Willebrand factor (VWF). Under normal conditions, this factor is present in blood where it functions as a ‘glue’ that sticks blood cells (platelets) to damaged blood vessels, thereby preventing excessive blood loss. For the most severely affected patients (complete absence of VWF), there is currently only one single treatment option: ...