Maria Grazia Roncarolo
Stem Cell Transplantation
United States of America
Dr. Roncarolo leads efforts to translate scientific discoveries in genetic diseases and regenerative medicine into novel patient therapies, including treatments based on stem cells and gene therapy. A pediatric immunologist by training, she earned her medical degree at the University of Turin, Italy. She spent her early career in Lyon, France, where she focused on severe inherited metabolic and immune diseases, including severe combined immunodeficiency (SCID), better known as the "bubble boy disease." Dr. Roncarolo was a key member of the team that carried out the first stem cell transplants given before birth to treat these genetic diseases.
Immunetolerance: Mechanisms underlying T-cell tolerance, induction of T-cell anergy and regulatory T cells; Immunomodulation: mAbs, proteins and low molecular weight compounds which can modulate T-cell activation; Primary immunodeficiencies: Characterization of molecular and immunological defects; Gene therapy: Gene transduction of hematopoietic cells for gene therapy in primary immunodeficiencies and metabolic diseases; Hematopoiesis: Mechanisms underlying growth and differentiation of hematopoietic stem cells; Transplantation: Immune reconstitution and T-cell tolerance after allogenic stem cell transplantation; Cytokines/Cytokine receptors: Role in regulation of immune and inflammatory responses