Results for AAV Vectors

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

Dr. van der Loo is an Adjunct Associate Professor of Pathology and Laboratory Medicine, Perelman School of Medicine at the University of Pennsylvania and Director of the Clinical Vector Core at CHOP, focusing on manufacturing of GMP and GMP process-comparable AAV and Lentiviral vectors for preclinical and gene therapy early phase clinical studies.

The research in the Sabatino Laboratory is focused on hemophilia, an inherited bleeding disorder. The interests of the laboratory include the study of variants of coagulation factor VIII to understand the biochemical properties of these proteins and to identify novel variants with enhanced function, and the development of gene-based therapeutic approaches for treating hemophilia.