Pathogenesis and therapy of primary immunodeficiencies

Alessandro Aiuti

Alessandro Aiuti

Location: Settore D, Floor 3, Room 6

Deputy director clinical research, San Raffaele Telethon Institute for gene therapy (SR-Tiget)
Group leader, Pathogenesis and therapy of primary immunodeficiencies Unit
Group leader, Pediatric Clinical Research Unit, SR-Tiget
Associate professor, Università Vita Salute San Raffaele
Head physician, Pediatric Immunohematology Unit

He initially focused his research studying diseases of the coagulation and HIV infection. His research on hematopoietic stem cells led to the discovery of the first chemokine produced by bone marrow cells inducing migration of human hematopoietic stem cells. His current research is focused on pediatric hematology and immunology and specifically on primary immunodeficiencies, genetic diseases of the immune system. He pioneered the successful gene therapy clinical trial for SCID patients that lack adenosine deaminase (ADA), a fatal disorder of purine metabolism and immunodeficiency. This clinical trial, now completed, introduced a conditioning regimen to favor the engraftment of gene corrected cells and is considered to be among the most important clinical result of gene therapy for genetic diseases. Gene therapy for ADA SCID has been designated as Orphan Drug in Europe and USA. His most recent research with hematopoietic stem cells transduced with lentiviral vectors led to the successful application of therapy for another primary immunodeficiency, Wiskott-Aldrich syndrome.

He is author of more than 100 publications in international peer-review journals and 5 book chapters. He gave more than 200 lectures as invited speaker and abstract presentations to national and international congresses. He received several national and international awards for his scientific activity and is a member of several national and international scientific societies His research is funded by international and national agencies including the European Union, Telethon Foundation, the Italian Ministry of Education University and Research, Italian Ministry of Health, Fondazione Roma.