Alessandra Biffi is the Chief of the Pediatric Hematology, Oncology and Stem Cell Transplant Division at Padua University and at the Padua University Hospital since October 2018, and coordinates the Research Area on Onco-Hematology, Stem Cell Transplant and Gene Therapy at the Pediatric Research Institute in Padua. Previously, she was the Director of the Gene therapy Program and clinical attending in Stem Cell Transplant at the at Dana-Farber/Boston Children’s Cancer in Boston (2015-2018), and Head of unit at the San Raffaele Telethon Institute for Gene Therapy in Milano. Her preclinical and clinical research, and clinical activity are dedicated at developing innovative treatment modalities for monogenic disorders based on hematopoietic stem cell (HSC) transplantation and gene therapy. Her research is devoted at enhancing the efficacy of HSC-based therapeutic approaches for neurometabolic disorders with severe nervous system involvement by fostering brain microglia replacement by donor cells after HSC transplantation upon detailed understanding of this phenomenon, and enhancing the potential of protein delivery to the affected nervous system by means of the gene corrected progeny of the transplanted and engineered HSCs. She received a Consolidator ERC grant award on this research and she is now conducting exploratory novel work on the therapeutic role of engineered microglia in adult onset neurodegenerative conditions.