Alessandra Biffi, Boston Children's Hospital, USA

Alessandra Biffi is the current director of the Gene therapy Program at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center. Her previous position was in Milano, at the San Raffaele Telethon Institute for Gene Therapy where she trained and developed a Research and Clinical Unit dedicated to the treatment of lysosomal storage disorders (LSDs) by means of hematopoietic stem cell (HSC)-based approaches. She is actively involved in gene therapy trials for genetic diseases of childhood. Her specific research is dedicated at enhancing the efficacy of HSC-based therapeutic approaches for LSDs with severe nervous system involvement by i) fostering brain microglia replacement by donor cells after HSC transplantation upon detailed understanding of this phenomenon (Capotondo et al., PNAS 2012), and ii) enhancing the potential of enzyme delivery to the affected nervous system by means of the gene corrected progeny of the transplanted, lentiviral vector (LV)-transduced HSCs (Biffi et al., Science 2013; Sessa et al., Lancet 2016).

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