Gene therapy researchers at The Children’s Hospital of Philadelphia have produced a bioengineered decoy that by fooling the immune system prevents it from undermining the benefits delivered by a corrective gene. The decoy could offer a new treatment for genetic diseases while advancing the broader field of gene therapy if the approach succeeds in humans.
The long-term goal of the Song Lab is to elucidate the cellular and molecular basis governing the formation, maintenance, and function of neural circuits under physiological and pathological conditions, using both Drosophila and mammalian models
The research in the Sabatino Laboratory is focused on the inherited bleeding disorder, hemophilia. 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.
Research in the Margaritis Lab uses basic biochemical, molecular, and complex in vivo methodology within the field of coagulation. The goals of the lab’s research programs are to advance the understanding of molecular mechanisms involved in pro- and anti-coagulant reactions, and conduct translational research for the treatment of coagulation defects.
The Camire Lab is interested in 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. The lab 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.
Research in the Akizu Lab aims to better understand human brain complexity in health and disease, with the ultimate goal of uncovering therapeutic targets for pediatric neurodegenerative disorders. Current research in the lab is focused on cerebellar ataxias and motoneuron disorders, with the specific goals of uncovering the particularities of these neuronal types, better understanding disease mechanisms, and exploring treatment options.