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Biography

Curriculum vitae Instruction * 1998 Specialization in Hematology, University of Milan * 1996 PhD in Molecular and Cellular Biology, University of Rome "La Sapienza" * 1990 Degree in Medicine and Surgery, University of Rome "La Sapienza" Work experiences * 2014-today Director of the Immunematologic Pediatrics Clinic, San Raffaele Hospital * 2014-today Associate Professor of Pediatrics, San Raffaele Vita-Salute University * 2011-today Head of Unit, Pediatric Clinical Research Unit, HSR-TIGET * 2009-today Area Coordinator Clinic, HSR-TIGET * 2007-present Head of Unit "Pathogenesis and Gene Therapy of ADA-SCID", HSR-TIGET * 2007-2014 Associate Professor of Pediatrics, University of Rome "Tor Vergata" * 2003-2007 Head Unit, Doctor Researcher, San Raffaele Hospital Scientific Institute * 2000-2014 Hematologist, Pediatric Clinical Research Unit, HSR-TIGET * 2000-2007 Medical Researcher, San Raffaele Hospital Research Institute * 1997-2000 Researcher, Telethon Foundation, Italy * 1996-1997 Post-Doctoral Fellow, TIGET, San Raffaele Hospital Institute, Milan * 1994-1996 Post-doctoral fellow, Center for Blood Research, Harvard Medical School, Boston, MA, USA * 1991-1995 PhD, University of Rome "La Sapienza" Initially he conducted his studies on coagulation and HIV infection. His research on hematopoietic stem cells conducted in the US led to the discovery of the first chemokine produced by bone marrow cells that is capable of attracting human hematopoietic stem cells. His current research focuses on pediatric hematology and immunology, particularly on primary immunodeficiencies, genetic disorders of the immune system. He conducted the first successful clinical trial for gene therapy for patients with ADD-SCID deficiency (ADA-SCID), who lose adenosine deaminase (ADA). In this clinical trial (now completed), for the first time, a low-intensity conditioning was introduced to encourage correct cellular implantation. The clinical trial is considered one of the most important clinical findings of genetic therapy for genetic diseases. Gene therapy for ADA SCID has been named as "Orphan Drug" in Europe and the USA. Recent research has led to the successful application of gene therapy with lentiviral vectors transplanted with hematopoietic stem cells for another primitive immune deficiency, Wiskott-Aldrich Syndrome. He is author of more than 100 publications on international journals and 5 chapters of books. He has presented more than 200 communications as invited speakers or abstracts at national and international conferences. He has received numerous national and international awards for his scientific work and is a member of several national and international scientific societies. His research is funded by international and national agencies such as the European Community, the Telethon Foundation, the Ministry of Education and Research, the Ministry of Health, the Rome Foundation. Awards and scholarships * 2014 National Prize for Solidarity Culture for "the important activity that many benefits bring to humanity" * 2010 AACS Prize in Rome - Special Mention "Heart of Rome" * 2010 XVIIIth Congress ESGCT (European Society of Therapy and Cellular Therapy) : Award for outstanding career and pioneering contribution to the field * 2009 HSR Scientific Retreat: Award for Best Translational Translation Paper * 2004 JOUAN BIOTHERAPY AWARD Research Award for Best Clinical Research Project * 2003 Young Investigator Award, American Society of Gene Therapy * 2002 Best Abstract Award presented at the plenary session of the American Society of Gene Therapy conference. * 1996 American Society of Hematology travel bag for presentation of oral communication at the annual ASH Congress in San Diego * 1993 Scholarship from the Ministry of Health for Aids research abroad 1991 1991 Pasteur Institute Award Cenci Bolognetti Foundation , University of Rome "La Sapienza", for the best experimental thesis of the year * 1989 Study prize of the University of Rome and the Sigma Tau Foundation for the best basic research work Membership in American Society of Hematology (ASH), American Society of Therapy and Cell Physiology (ASGCT), European Society of Thyroid and Cellular Therapy (ESGCT), Italian Society of Pediatrics (SIP), Italian Association of Pediatric Oncology Hematology (AIEOP) European Society of ImmunoDefficiencies (ESID), Strategic Committee and Immunodeficiency Study (AIEOP-IPINET), European Working Party on Inborn Errors (EBMT). Professional activities * 2013-today Chair of the ASGCT * Hematologic and Immunologic Gene and Cell Therapy Committee 2012-today ESCGT Board Member 2011-today Review Editor, Frontiers in Primary Immunodeficiencies * 2010-present Member of Italian Primary Immunodeficiency Network Working Group (IPINET) * 2011-2012 Member of the AIEOP Executive Committee * 2008-2012 Member of the ASGCT * 2005-2009 Member of the Editorial Board of the Human Gene Therapy Journal * 1998-2003 Assistant European Publisher, Human Gene Therapy Journal Institutional appointments * 2012-present Ad hoc expert of the drafting group for the Committee for Advanced Therapies (CAT) EMA * 2010-2012 Italian member of the European Medicines Agency (EMA) Gene Therapy Working Party

Research Interest

Medicine and Surgery