Results for Molecular Therapy

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.

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. Weber is developing approaches to enhance CAR-T cell therapies for pediatric cancer by reprogramming T cells with improved durability and exhaustion resistance. His work will uncover molecular mechanisms that promote CAR-T cell exhaustion and identify new targets for therapeutic intervention.

Dr. Davidson works to understand the molecular basis of childhood onset neurodegenerative diseases and the development of gene and small molecule therapies for treatment. She also focuses on how noncoding RNAs participate in neural development and neurodegenerative disease processes, and how they can be harnessed for therapies.

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. Minturn's primary clinical and research focus is the development of early-phase clinical trials targeting the molecular drivers of brain/spinal cord tumors. She also focuses on late effects of tumor-directed therapies on the developing nervous system and neurocognitive effects of treatment.

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. 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.

Dr. Hunger's focuses his research on molecular and genomic approaches to identify and clinically evaluate targeted cancer treatments for children with relapsed or high-risk acute lymphoblastic leukemia (ALL) such as Philadelphia chromosome-like (Ph-Like) ALL. The long-term goal of Dr. Hunger’s research is to develop better therapies, improve cure rates, and minimize treatment toxicities for children with ALL.

Dr. Morgan is a resuscitation scientist who studies pediatric cardiac arrest and cardiopulmonary resuscitation (CPR) through both translational laboratory work and clinical studies. His focus is to identify disease-specific pathophysiology that can be specifically targeted during CPR.