Dr. Rivella is an expert in the pathophysiology of erythroid and iron disorders and in the generation of lentiviral vectors for the cure of hemoglobinopathies. He also investigates additional disorders such as anemia of inflammation and hemochromatosis.
The Raymond G. Perelman Center for Cellular and Molecular Therapeutics has established state-of-the-art Current Good Manufacturing Practices (cGMP) clinical vector manufacturing suites for adeno-associated virus (AAV) and lentiviral (LV) vectors.
Bookended by references to the 1980s and 1990s — in the form of an iconic U.S. Navy commercial and a discussion of the 1991 measles outbreak in Philadelphia — the 2015 CHOP Research Institute Scientific Symposium offered a snapshot of the inspirational work going on at CHOP every day.
Whether they study helmets on the football field or hemophilia in a lab, our scientists at Children’s Hospital of Philadelphia Research Institute are always on the cutting-edge of their respective fields, as the latest roundup of research news shows.
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.