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. Heuckeroth investigates mechanisms controlling bowel motility in order to find new ways to treat, diagnose, and prevent intestinal motility disorders. He works to define genetic, biochemical, and cellular processes that impact bowel function, with a special interest in the enteric nervous system and intestinal smooth muscle cells.
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. 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. 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.
Dr. Falk is a Clinical Geneticist who serves as executive director of the Mitochondrial Medicine Frontier Program. Her translational research lab investigates the causes and global metabolic consequences of mitochondrial disease, as well as targeted therapies, in C. elegans, zebrafish, mouse, and human tissue models of genetic-based respiratory chain dysfunction.
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. Rubenstein's research focuses on basic and translational studies of novel means to improve outcomes in cystic fibrosis. He initially focused on correcting the dysfunction of mutant cystic fibrosis transmembrane conductance regulator (CFTR) proteins, which led him to study how molecular chaperones regulate the biogenesis and trafficking of CFTR and other proteins that are relevant to cystic fibrosis.
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.