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. Romberg investigates the regulatory mechanisms enabling our immune systems to fight infections without injuring ourselves. He is particularly interested in the immune system of patients with primary immunodeficiency who are susceptible to both life-threatening infections and autoimmune diseases. Greater insights into these rare diseases may enable rationale development of targeted therapies for more common diseases with an immunologic basis.
Dr. Ackermann studies diabetes (types 1 and 2) and congenital hyperinsulinism using mouse models, cell lines, and primary human tissue. She aims to identify novel pathways regulating beta cell insulin secretion, leading to innovative therapeutic strategies for these disorders. Current studies include in vivo mouse physiology, ex vivo human islet physiology, CRISPR-Cas9 gene editing, epigenetic modification, and single-cell functional genomics.
Dr. Goldberg's research program focuses on investigating cerebral cortical circuit function and dysfunction in neurodevelopmental disorders. Using a variety of research techniques, Dr. Goldberg has a specific research interest in the workings of neuron subtype called GABAergic inhibitory interneuron and the role of interneuron dysfunction in disease.
Dr. Bunin's current research focuses on the development of hematopoietic stem cell graft engineering for allogeneic transplantation to minimize graft-versus-host disease (GVHD), promote engraftment and immune reconstitution. She has also developed protocols for rapid manufacture of viral cytotoxic T lymphocytes to treat or prevent life threatening viral infections post hematopoietic stem cell transplant.
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. Marsh's research program focuses on understanding how changes in brain development lead to epilepsy, intellectual disability, and autism. He combines molecular and physiological tools in mouse models to ask questions about the interaction of normal development with single gene mutations to determine how the brain responds to perturbations in development.
Dr. Ortiz-Gonzalez is a physician-scientist specializing in pediatric neurogenetics. Her clinical work focuses on finding a unifying genetic diagnosis for children with rare neurodevelopmental disorders. Her research is informed by her patients and focuses on understanding how genetic changes, in particular those affecting mitochondrial function, cause disease so we can develop better treatments for these children in the future.