Phenotypic correction of von Willebrand disease type 3 blood-derived endothelial cells with lentiviral vectors expressing von Willebrand factor

Abstract excerpt

Von Willebrand disease (VWD) is an inherited bleeding disorder, caused by quantitative (type 1 and 3) or qualitative (type 2) defects in von Willebrand factor (VWF). Gene therapy is an appealing strategy for treatment of VWD because it is caused by a single gene defect and because VWF is secreted into the circulation, obviating the need for targeting specific organs or tissues. However, development of gene...

Topics

Share this publication in a Topic to start or enrich a Post.

• Share to Blood Coagulation and Thrombosis Mechanisms
• Share to Complement system in diseases
• Share to Platelet Disorders and Treatments