Results for Adeno-associated Viral Vectors

The development of gene-based strategies for the treatment of bleeding and thrombotic diseases is at the heart of research in the Arruda Lab. Working collaboratively, the lab team and their colleagues have carried out early-phase clinical studies on adeno-associated viral vectors for the treatment of severe hemophilia B.

Our lab is interested in the hemostasis system, especially coagulation factor VIII and IX, which are deficient in the bleeding disorders hemophilia A and B. By studying these blood proteins, the lab team aims to improve therapies for children with these diseases including gene therapy.