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Rosa Bacchetta’s professional goal as a pediatrician specializing in immunology, has been to challenge the limits of "inexplicable" and "untreatable" diseases. Her translational research focuses on Autoimmune Genetic Diseases and Primary Immunodeficiencies, from identifying causative genes, understanding the pathogenesis, defining novel diagnostic and prognostic markers and investigating new therapeutic approaches. Following the completion of her pediatrics residency at the University of Turin, she received training in molecular and cellular immunology in the United States (DNAX Research Institute of Molecular and Cellular Biology, Palo Alto). There, she was first exposed to the importance of integrating in depth laboratory research with clinical observations to develop a translational research approach to science. She then worked for fifteen years at the San Raffaele Scientific Institute (HSR-TIGET), where she focused on dissecting the genetic and immunological basis of primary immune-regulatory diseases that might be treated by gene therapy. She is Faculty at Stanford since 2015.
FOXP3-Engineered T Cells: A Novel Treg Immunotherapy
This talk will cover the characterization of CD4+ T cells converted into Treg-like cells upon lenti-viral mediated expression of FOXP3. The rationale for a First-In-Human trial in patients with IPEX Syndrome, a rare genetic autoimmune disease, will be presented and their potential broader use in different immune-mediated diseases will be discussed.