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.
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.
High is one of the world’s leading experts in gene therapy, which has long been a “next big thing” in medicine: Take a person with a devastating genetic disease and replace their nonfunctional gene with a normal one — a cure built right into your DNA.