Scopus ID: 25655343700
Dr. Poletti’s scientific career and interest in Gene Therapy began in 2005 as a student at the University of Modena and Reggio Emilia in the laboratory of Prof. F. Mavilio where she worked on a gene therapy strategy for Junctional Epidermolysis Bullosa (JEB) (Di Nunzio et al. Molecular Therapy 2008). She then continued her research working on the development of a gene therapy strategy for a dominant monogenic disease of the cornea, Meesmann’s Corneal Dystrophy (MCD), which later became the subject of her Master’s thesis. She was accepted at the graduate school of Molecular Medicine at the University Vita-Salute San Raffaele (Milan, Italy) where she investigated the early commitment of human stem cells by applying genomics, epigenetics and gene expression technology (Poletti V. et al., PLOS ONE 2015). At the same time, taking advantage of her knowledge in vectorology, she collaborated with Prof. G. Ferrari at TIGET (Milan, Italy) for the optimization of a lentiviral vector designed for the gene therapy of the β-thalassemia (GLOBE) (Miccio A. et al., PLOS ONE 2011). Additionally, she contributed to a study on the human hematopoietic lineage commitment conducted by Dr. A. Miccio (Romano O. et al., Scientific Reports 2016; Romano O. et al; JoVE 2016).After her PhD, she moved to the Department of Molecular Medicine of the University of Padua where she worked on the design of lentiviral vectors expressing silencing RNAs against several HIV proteins for the gene therapy of AIDS with Prof. Cristina Parolin and on the characterization of glioblastoma cancer stem cells with Prof. Giorgio Palu’.In 2014 she joined Genethon, a French institute dedicated to translational research in gene therapy for rare diseases, as a staff scientist where she developed pre-clinical IND-enabling studies of gene therapy for X-linked severe combined immunodeficiency (SCID-X1) and sickle-cell disease (SCD)(V. Poletti. et al. June 2018, V. Poletti et al. December 2018). Both studies were instrumental for the approval of two clinical trials by the FDA and the ANSM. Additionally, she contributed to a preclinical study by Dr. Galy for the ex vivo gene therapy for ARTEMIS-SCID (Charrier et al. 2019). She set up and supervised a research team for Vector Integration Site Analysis focused on the recovery and analysis of γ-retroviral and lentiviral integration sites in the human genome and she collaborated on several projects includinga preclinical study of the gene therapy for β-thalassemia (Liddonici M.R. et al 2018) and the characterization of the integration preferences of the Sleeping Beauty transposon-vector in human HSPCs (Holstein et al. 2018). She contributed to the design and characterization of innovative anti-sickling lentiviral vectors for the gene therapy for SCD (Weber L. et al, 2018, and Urbinati F. et al. 2018).In 2018 she moved to the Gene Therapy Program at Dana-Farber/ Boston Children’s Hospital (Boston, MA, USA), where she worked with Prof. Biffi as Instructor in pediatrics (Harvard University, Faculty of Medicine) at the preclinical development of an ex-vivo gene therapy for Infantile Neuronal Lipofuscinosis (manuscript in preparation), and investigated the long-term effects of prostaglandin exposure on human Hematopoietic Stem and Progenitor Cells (manuscript in preparation). She is currently an affiliated Faculty member at Harvard University. In 2020 she moved back to Italy awarded with a Marie Skłodowska-Curie Fellowship dedicated to HSC gene therapy application for GM1-gangliosidosis in the Department of Women and Child Health, University of Padua (Padua, Italy).
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Early development of a viral-free ex vivo gene therapy for β-hemoglobinopathiesβ-Thalassemia and Sickle Cell Disease are the most common monogenic diseases worldwide, with >317,000 newborn/year, caused by defects of β-globin expression or structure.
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