Results for In Vivo Gene Therapy

Dr. Margaritis uses biochemical, molecular, and complex in vivo methodology within the field of coagulation to advance the understanding of molecular mechanisms involved in pro- and anti-coagulant reactions, and translate research for the treatment of coagulation defects.

Dr. Camire's research focuses on understanding the components of the blood coagulation system, how they interface with activated cells, and how disturbances in their function lead to bleeding and thrombosis. He is also interested in developing novel therapeutic approaches (protein, gene-based, small molecule) to mitigate these events, which are major causes of morbidity and mortality worldwide.

Perry Demsko's current research projects include in-vivo gene therapies for diseases of the hematopoietic lineage and novel viral pseudotypes.

Dr. Castracani’s research work is focused on the most common form of X-linked sideroblastic anemia which results from mutations in the ALAS2 gene and inhibits normal heme biosynthesis.

Omar Banda investigates therapeutic applications of novel nanoparticle delivery systems for the treatment of Fanconi Anemia.