Roncarolo Maria Grazia

Biography

03/07-present Ordinary Professor in Pediatrics, Faculty of Medicine and Surgery, Vita-Salute University San Raffaele, Milan, Italy; 07/03-present Director of the Strategic Research Program of Pediatric Immunohematology IRCCS San Raffaele, Milan, Italy; 02/98-now Head Unit of Immunological Tolerance Unit, San Raffaele Telethon Institute for Genital Therapy (TIGET), Milan, Italy; 03 / 08-09 / 13 Scientific Director of the San Raffaele Institute, Milan, Italy; 06 / 00-09 / 08 Director of San Raffaele Telethon Institute for Genital Therapy (HSR-TIGET), Milan, Italy; 12 / 98-05 / 00 Co-Director of the San Raffaele Telethon Institute for Genital Therapy (HSR-TIGET), Milan, Italy; 02 / 98-12 / 98 Director of Cell Therapy Laboratory, San Raffaele Telethon Institute for Therapy Genica Milano, Italy; 11 / 01-02 / 07 Associate Professor in Pediatrics, Faculty of Medicine and Surgery, Vita-Salute University San Raffaele Milan, Italy; 11 / 94-10 / 01 Associate Professor in Pediatrics, Department of Pediatrics, Faculty of Medicine and Surgery, University of Turin, Turin, Italy; 3 / 92-12 / 96 Senior Scientist, Human Immunology Department, DNAX Research Institute for Molecular and Cellular Biology, Palo Alto, CA; 3 / 89-3 / 92 Staff Scientist, Human Immunology Department, DNAX Research Institute for Molecular and Cellular Biology, Palo Alto, CA; 1990 Specialization (summa cum laude) in Clinical Immunology, Faculty of Medicine and Surgery, University of Milan, Milan, Italy; 2 / 88-2 / 89 Foreign Assistant (Medecin Resident Evidence of Hospices Civils de Lyon), Center for Transplantation and Clinical Immunology, E. Herriot Hospital, Lyon, France; 8 / 86-1 / 88 Senior Research Scientist (Chargée de Recherches), Schering-Plow Immunological Research Laboratory, Lyon, France; Assistant Professor, Faculty of Medicine and Surgery, University Claude Bernard, Lyon, France; 1986 Specialization (summa cum laude) in Pediatrics, Faculty of Medicine and Surgery, University of Turin, Turin, Italy; 1985-7 / 86 Internal Medicine, Pediatric Clinic, Division of Immunology, Faculty of Medicine and Surgery, University of Turin, Turin, Italy; 1984 Research Associate, Schering-Plow Immunological Research Laboratory, Lyon, France; 1983-84 Research Fellow, Transplantation Center and Clinical Immunology E. Herriot Hospital, Lyon, France; 1980-83 Internal Medicine, Pediatric Clinic, Division of Immunology, Faculty of Medicine and Surgery, University of Turin, Turin, Italy; 1982 Degree in Medicine (summa cum laude), University of Turin, Turin, Italy. Research activity He has been working for many years in Lyon, at the Transplant Center of Edouard Herriot Hospital and at the Schering Plow Immunological Research Laboratory, on the mechanisms of tolerance in patients with severe combined immune deficiency (SCID), healed by stem cell transplantation blood. She worked for more than 8 years at the DNAX Research Institute of Molecular and Cellular Biology, Palo Alto, CA on cytokine biology, gene transfer to blood stem cells, and mechanisms for immunology tolerance. He focused his career on basic research and clinical research, both in the immunological and hematological fields. His main interest has always been the application of basic research to the care of pediatric patients suffering from hematologic and immunological diseases, with the belief that the transfer of results from the laboratory to the clinic and vice versa is of fundamental importance for the advancement of medicine and the development of new drugs. Since the beginning of his career, he has studied the identification of mechanisms that characterize genetic and acquired diseases of the immune system and hematopoietic system and the definition of new approaches to cell and cell therapy for these diseases. In the field of genetic diseases he focused his research on children with severe combined immune deficiency (SCID), complex primitive immunodeficiencies such as Wiskott Aldrich syndrome and Talassemia major. In this context, he has developed research into the mechanisms of in vitro expansion and gene transfer into stem-derived hematopoietic cells derived from fetal liver, umbilical cord and bone marrow. It has developed a new clinical gene therapy protocol for ADA-SCID that ensures long-term correction of genetic disease with proven clinical benefits. These results have made it possible to designate gene therapy for ADA-SCID as Orphan Drug, and today's clinical trial is considered to be the most important achievement in gene therapy for genetic diseases. He has also conducted numerous researches in the field of immunological tolerance and autoimmune pediatric diseases. His research group has discovered a new subclass of regulatory T cells called T-type regulatory cells. Since the publication of the result on "Nature", it has been shown that these cells play a key role in immunological homeostasis and in particular in prevention of self-immunity and suppression of transplant rejection. Clinical cell therapy protocols already concluded or ongoing have used these regulatory cells in transplant patients.

Research Interest

Immunotollerance: Mechanisms that preserve T cell tolerance, induction of T cell anergy and regulatory T cells Immunosuppression: Mechanisms that prescribe the activation and immunological tolerance of T cells Primary Immunodeficiency: Characterization of molecular and immunological defects Gene therapy: Gene transfer of hematopoietic cells for gene therapy of primary immunodeficiency and metabolic diseases? Ematopoiesis: Mechanisms that preserve the growth and differentiation of hematopoietic precursors Transplants: Immune reconstitution and T cell tolerance after allogeneic stem cell transplantation in genetic diseases? Cytokines: Role in regulating immune and inflammatory responses Its main clinical interests are: Primary Immunodeficiency, Autoimmune Diseases, Allogeneic Cell Transplantation, Clinical Trials of Cell Therapy, Clinical Cell Therapy Trials, Clinical Trails for Autoimmune Diseases and Organ Transplantation, Clinical Trails for Hemoglobinopathies. He has published more than 280 articles in international scientific journals, and has written 22 chapters of books. Throughout his work, we have quoted> 19000 times (Scopus index "h": 64). Holder of 14 patents partly already published and partly in the approval phase. Professional activities: Member of the California Institute for Regenerative Medicine (CIRM) Sceintific Advisory Board (April 2013-present). Member of the Scientific Advisory Board of the French Rare Diseases Foundation (June 2012-today). Member of the Scientific Advisory Board of the Global Health Institute (GHI) in Lausanne (April 2011-present). Founder of the Eureka Institute for Translational Medicine (May 2008-present). He is a member of the "Group 2003" (of which he is President since October 2012), which brings together Italian scientists working in Italy, the most cited in the world in scientific literature, and foresees in his statute the development of proposals to support a "rebirth of scientific research in Italy "(2008 to today). Member of the External Scientific Advisory Board (ESAB) of Tumorzentrum L. Heilmeyer, Comprehensive Cancer Center Freiburg (CCCF) (2007-2012). Member of the Scientific Advisory Board of the Institute of Transplantation and Transplantation (ITERT) University of Nantes, France (2001-January 2009). Chairman of the Scientific Advisory Board of the Association Française Contre les Myopathies (AFM) / Genethon (1999-2002). Novartis Pharmaceutical Adviser in Immunology, Transplantation and Genital Therapy (1997-2002). Member of the Board of Directors of the Italian Society of Allergology and Pediatric Immunology (SIAIP) (1997-2001). Member of the Scientific Advisory Board of Kinetix Pharmaceutical (1997-2000). Member of the "Cytokine Team" of Schering-Plow, responsible for the development of cytokines for clinical applications (1993-1996).