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Alessandra Biffi is the director of the Gene Therapy Program at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center. Biffi's research is focused on gene therapy trials for genetic diseases in childhood, particularly on enhancing the efficacy of hematopoietic stem cell (HSC)-based therapeutic approaches for lysosomal storage disorders (LSDs) with severe nervous system involvement. Her work explores HSC transplantation as a vehicle for the delivery of therapeutic molecules to the central nervous system (CNS), aiming to improve outcomes in conditions such as neurodegenerative diseases (NDDs). Biffi has demonstrated the therapeutic potential of HSC transplantation in treating NDDs by employing innovative gene transfer methods that increase the production of therapeutic molecules from transplanted HSC progeny. Her lab is also investigating strategies for optimizing the timing and methods of HSC delivery to enhance microglial cell reconstitution in the CNS, with a specific focus on using lentiviral vectors to express therapeutic genes. Biffi's expertise includes a significant clinical background in treating lysosomal storage disorders through HSC-based approaches, having previously worked at the San Raffaele Telethon Institute in Milano. Her research is committed to advancing gene therapy methodologies to address the challenges posed by neurodegenerative diseases, including Amyotrophic Lateral Sclerosis (ALS) and Alzheimer’s Disease.
Gene Therapy Program, Dana-Farber/Boston Children’s Cancer and Blood Disorders Center • Cambridge, MA
Oversees the gene therapy research and clinical trials aimed at treating genetic diseases in children.