Doctor Alessandro Aiuti
About the doctor
Prof. Alessandro Aiuti is a hematologist and pediatrician, Deputy Director of Clinical Research at San Raffaele and Head of the Pathogenesis and Therapy of Primary Immunodeficiencies Unit. At IRCCS Ospedale San Raffaele, he heads the Pediatric Immunohematology Unit. He is also Professor of Pediatrics and Director of the Postgraduate School in Pediatrics at Università Vita-Salute San Raffaele.
His main clinical and research focus is on primary immunodeficiencies and inherited disorders of the immune system in children, as well as the development of new treatment approaches. A particular focus of his work is gene therapy using genetically modified hematopoietic stem cells. His research group works on diseases including ADA-SCID, Wiskott-Aldrich syndrome, and several lysosomal storage disorders.
Professor Aiuti combines clinical practice with translational research, ranging from the study of disease mechanisms to clinical trials of innovative treatments. In 2025, his group also received funding for a project focused on developing new approaches to treating skeletal manifestations of lysosomal storage disorders.
In 2020, he received the Else Kröner Fresenius Prize for Medical Research for his contribution to the development of gene therapies for rare diseases.
Key areas:
- Pediatric immunohematology and primary immunodeficiencies
- Gene therapy for inherited diseases
Dr. Alessandro Aiuti’s main professional interests:
- pediatric hematology, immunology, immunohematology
- primary immunodeficiencies
- inherited disorders of the immune system
- ADA-SCID
- Wiskott-Aldrich syndrome
- inborn errors of immunity
- metachromatic leukodystrophy
- mucopolysaccharidosis type I
- disorders associated with impaired immune system function
- gene and cell therapy
- hematopoietic stem cells
- genetically modified stem cells
- stem cell transplantation
- rare genetic diseases
- development of new treatment approaches
- clinical trials of gene therapy
- safety and efficacy of gene therapy
- regenerative medicine
