Development of a dual hybrid AAV vector for endothelial-targeted expression of von Willebrand factor

Abstract excerpt

Von Willebrand disease (VWD), the most common inherited bleeding disorder in humans, is caused by quantitative or qualitative defects in von Willebrand factor (VWF). VWD represents a potential target for gene therapy applications, as a single treatment could potentially result in a long-term correction of the disease. In recent years, several liver-directed gene therapy approaches have been exploited for VWD, but...

Topics

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

• Share to Complement system in diseases
• Share to Platelet Disorders and Treatments
• Share to Virus-based gene therapy research