Dr. Bacchetta has a long-standing experience investigating the mechanisms of immunological tolerance, specifically in pediatric patients after hematopoietic stem cell transplantation, and those with genetic diseases of the immune system. 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. Her work has significantly contributed, in the past twenty years, to dissecting the role of FOXP3 and Treg cells in immune responses in humans, as well as to raising the interest of scientists and clinicians in investigating diseases with severe immune dysregulation, such as the prototype Tregopathy, IPEX Syndrome. 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). She then worked for fifteen years at the San Raffaele Telethon 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 in Pediatrics. Dissecting the role of FOXP3 and Treg cells in human immune responses, her goal is to establish cell and gene transfer-based therapies for IPEX Syndrome, using lentivirus-based transduction of CD4+ T cells and CRISPR/Cas9 gene editing into HSCs. She is the Sponsor of the Phase 1 gene therapy trial using autologous engineered CD4LVFOXP3 Treg-like cells to treat IPEX patients.