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.
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.
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. Prosser's research focuses are the development and rehabilitation of movement in children, particularly those with neurological impairments. This includes the investigation of the development of impaired movement, the study of novel motor rehabilitation interventions in children, and the interaction between the processes of neuroplasticity and neuromaturation in sensorimotor systems.
Dr. Bhoj's genetics research aims to discover new human disease genes, their mechanisms, and potential targeted therapies. In addition to ongoing gene discovery efforts, Dr. Bhoj focuses on three novel genes that lead to pediatric neurologic dysfunction: TBC1 domain-containing kinase, Histone 3.3 (H3F3A and H3F3B), and MAP4K4.
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.
The cure rate for children with neuroblastoma is unacceptable, making it imperative that new therapies are developed. Dr. Bosse's laboratory is focused on discovering and developing new neuroblastoma cell surface immunotherapeutic targets. Along with his colleagues, Dr. Bosse's aim is to capitalize on the robust differential expression of these molecules with immune-based therapies and also define their mechanisms of overexpression and roles in tumorigenesis.
Dr. Gonzalez-Alegre's long-range research goal is to advance the application of precision medicine in the neurology clinic. His research focus revolves around genetic disorders that affect the brain, spanning from the diagnosis of novel genetic disease in the clinic to the identification of novel molecular targets using disease models and the design of early-phase human clinical trials.
Dr. De Leon-Crutchlow’s translational research program focuses on examining the pathophysiology of disorders of insulin regulation, identifying novel therapeutic targets, and developing new therapies for these conditions. The program approach includes patient-oriented research and bench research employing mouse models and primary islet cultures.
Dr. De Raedt researches pediatric high grade glioma development and aims to understand the involvement of crucial pathways. He investigates pathway interaction, and explores ways to develop therapies through analyzing human tumors, performing cellular studies, and developing accurate mouse models. This allows Dr. De Raedt and his team to perform novel pre-clinical studies that can lead to clinical trials.