Alessandra Biffi is in charge of the pediatric hemathology, oncology and stem cell transplant clinics and laboratory at University Hospital of Padua since October 2011 and she coordinates the research area on oncohemathology, stem cell transplant and gene therapy at the Pediatric Research Institute in Padua. Previously, she was the director of the Gene therapy Program at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center (2015-2018), and head of research and clinical unit for HSC gene therapy of lysosomal storage disorders (LSD) at the San Raffaele Telethon Institute for Gene Therapy, in Milano (up to 2015). She has trained over 20 fellows and post-doctoral fellows and numerous residents and medical students in her laboratory, the majority of which are still in academic medicine. She has published over 45 peer-reviewed manuscripts and textbook chapters. She is actively involved in gene therapy trials for neurological genetic diseases, hemoglobinopathies and immunodeficiencies. 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). Seminal preclinical work on Metachromatic Leukodystrophy (Biffi et al., JCI 2004 and 2006) generated solid grounds for an on-going Phase I/II clinical trial that she has conducted; she has treated over 20 patients and provided first obvious evidence of therapeutic efficacy of gene therapy for this disease (Biffi et al., Science 2013; Sessa et al., Lancet 2016).
Similar promising preclinical data were generated in her laboratory in the Globoid Leukodystrophy and Mucopolysaccharidosis type I (Visigalli et al., Blood 2010; Gentner et al., Sci Transl Med 2010; Visigalli et al., Blood 2010) animal models constitute the basis for new clinical trails that are in active phase of development also for these disorders. Novel indications are now being developed in the area of LSDs. Additional research activities comprise the characterization of new arylsulfatase A gene mutations (Cesani et al., Hum Mutations 2009), the identification and exploitation of novel biomarkers and therapeutic targets for lysosomal storage diseases (Cesani et al., Annals Neurology 2014), and novel exploratory activities on the therapeutic role of engineered microglia in neurodegenerative diseases. She is also involved in other gene therapy clinical trails active in the Gene Therapy Program and in advanced allogeneic transplant protocols for LSDs.
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