Maria Pia Cicalese is a physician-scientist currently working as a staff pediatrician in the Immunohematology and Bone Marrow Transplantation Unit of San Raffaele Hospital in Milan, Italy, and as a Researcher at Vita Salute San Raffaele University, Milan. Maria Pia has an extensive experience in autologous and allogeneic bone marrow transplantation in adults and children, and in gene therapy for the treatment of primary immunodeficiencies and neurodegenerative disorders. She is currently responsible for the Pediatric Immunohaematology outpatient clinic; clinical research coordinator at Tiget; autologous bone marrow transplant coordinator at Pediatric Immunohematology Unit, San Raffaele Hospital, Milan. She has been and currently is a scientific collaborator of several phase I-II clinical trials of genetic and cellular therapy for primary immunodeficiencies (including ADA-SCID and Wiskott-Aldrich Syndrome) and lysosomal storage disorders (MLD and MPSI). She has been a key person in the path of marketing authorization of Strimvelis, the first stem cells ex-vivo gene therapy for the treatment of ADA SCID, and is currently the responsible physician for this treatment at San Raffaele Institute for patients from EU and extra-EU. She is also an advisory board member for DADA2 Foundation. Maria Pia is author of several scientific publications on peer-reviewed journal and she is member of a number of Scientific Societies, as ESGCT, ESID, AIP, IPINET, AIEOP, EBMT, SIP.