Dr. Samelson-Jones is a pediatric hematologist dedicated to improving the lives of children with bleeding and clotting disorders. His research focuses on gene therapy for hemophilia, the biochemical basis of coagulation, and the immune responses to hemophilia therapies.
Dr. Grupp develops and conducts preclinical testing of engineered cell therapies and signal transduction inhibitors in leukemia, in pediatric immunotherapy trials, and in the manufacture and use of cellular therapeutics in preclinical, good manufacturing practices, and clinical trial settings. Dr. Grupp leads most CTL019 (CD19 CAR) clinical trials, and his colleagues are the global leaders in highly active CAR T cell therapy.
As a physician-scientist, Dr. Bernt's goal is to further the understanding of the role of transcriptional regulation in pediatric hematopoietic stem cell biology and leukemia, and translate findings into novel therapies.
Dr. Moorthy’s research focuses on liquid chromatography-tandem mass spectrometry assays for translational research. He is an expert in developing and validating analytical methods and their implementation for pediatric and adult clinical sample analysis.
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.
Dr. George's basic and clinical research interests are in the development of novel therapeutics for hemophilia. Her basic science laboratory studies the molecular basis of coagulation, and she is the principal investigator of ongoing hemophilia A and B gene therapy trials.