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. McCormack investigates the intersection of neuroendocrinology and metabolism. Her translational research program involves two areas. The first involves studying those with genetic disorders, including primary mitochondrial diseases and Friedreich's ataxia, with characterized risk for diabetes mellitus. Second, Dr. McCormack focuses on brain disorders associated with excess weight gain, including brain tumor-related hypothalamic obesity syndrome and idiopathic intracranial hypertension.
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. 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. Pacifici's biomedical research spans three decades and has explored mechanisms of skeletal development and growth in fetal and postnatal life. Specifically, his focus has been on identifying the cellular and molecular mechanisms that regulate the differentiation of progenitor cells and permit assembly of distinct skeletal structures, and on aberrations of these mechanisms in pediatric skeletal disorders.
Dr. Young’s translational research program aims to understand mechanisms underlying interstitial and rare lung diseases and develop new strategies to treat these disorders. Her laboratory focuses on the roles of epithelial cells in alveolar homeostasis, injury, and repair.
Dr. Maris investigates the molecular and genetic mechanisms contributing to the development and progression of neuroblastoma, a common childhood cancer. He also aims to develop new molecular diagnostic tests and less toxic, targeted therapies to treat relapsed or refractory neuroblastoma, including a major effort in immunotherapy discovery and development.
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. 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.