New approaches to gene and cell therapy for hemophilia.

Abstract excerpt

Hemophilia is considered suitable for gene therapy because it is caused by a single gene abnormality, and therapeutic coagulation factor levels may vary across a broad range. Recent success of hemophilia B gene therapy with an adeno-associated virus (AAV) vector in a clinical trial showed the real prospect that, through gene therapy, a cure for hemophilia may become a reality. However, AAV-mediated gene therapy...

Topics

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

• Share to Blood Coagulation
• Share to Dependovirus
• Share to Gene Transfer Techniques