ICPerMed conference


Francesca Ferrua

San Raffaele Telethon Institute for Gene Therapy, San Raffaele Scientific Institute, Milan


Francesca Ferrua is a physician scientist currently working as a staff pediatrician in the Immunohematology and Bone Marrow Transplantation (BMT) Unit of San Raffaele Hospital in Milan, Italy. She graduated at Vita-Salute San Raffaele University in Milan in 2008, where she also completed her éducation with a Residency in Pediatrics (2014) and an international PhD in Molecular Medicine (Basic and Applied Immunology Curriculum) (2019). In 2014, she spent a training period in the Pédiatrie Immunology and BMT Unit at Great North Children's Hospital in Newcastle, UK. Francesca's main interest is the diagnosis, management and treatment of pédiatrie patients affected by inborn errors of immunity and non-malignant hematological diseases. She is expert in allogeneic hematopoietic stem cell transplantation and gene therapy in patients affected by primary immunodeficiencies (ADA-SCID, Wiskott-Aldrich Syndrome - WAS) and lysosomal storage disorders (Metachromatic Leukodystrophy - MLD; Mucopolysaccharidosis type I, MPSI). In particular, she has been primarily involved as key investigator in the development and management of gene therapy clinical trials for WAS at San Raffaele Telethon Institute for Gene Therapy. Moreover, she is currently working on the pre-clinical development of gene-editing for the treatment of a primary immune deficiency, hyper IgM syndrome. She is author of several scientific publications on peer-reviewed journal and member of a number of Scientific Societies.