Fulvio Mavilio
Fulvio Mavilio
affiliation: San Raffaele Scientific Institute
research area(s): Molecular Biology, Genetics And Genomics
Course: Cell and Molecular Biology
University/Istitution: Università Vita-Salute San Raffaele
Fulvio Mavilio, Ph.D., is Professor of Molecular Biology at the University of Modena, and Head of the Gene Expression Unit of the San Raffaele Scientific Institute. From 1992 to 1999 Prof. Mavilio was Co-Director of the San Raffaele Gene Therapy Program, which he co-founded together with Claudio Bordignon in 1989. From 1999 to 2002 he was founder and Chief Scientific Officer of Genera S.p.A., and from 2002 to 2005 Director of Discovery of Molmed S.p.A. He had previously served as Visiting Scientist at the Wistar Institute, Philadelphia, (1986 to 1988), as group leader in the Department of Hematology-Oncology of the Istituto Superiore di Sanità, a Government Authority in Rome (1984 to 1988), and as staff scientist of the Institute of Experimental Medicine of the Italian National Research Council, Rome (1982 to 1984). Prof. Mavilio is member of the European Molecular Biology Association (EMBO), and chairman of the Educational Committees of the American and European Societies of Gene and Cell Therapy. He is in member of the Editorial Board of a number of international journals in the areas of gene therapy and molecular medicine. Prof. Mavilio graduated in Biology at the University of Rome in 1976, and obtained a Ph. D. in Medical Genetics at the School of Medicine of the same University in 1979. He was awarded a Special Fellowship by the Italian National Research Council, Rome (1978-1982), and a U.S. Public Health Service International Research Fellowship by the Fogarty International Center, NIH, Bethesda (1986-1988). Prof. Mavilio is an expert and a pioneer in the fields of gene therapy and stem cell research, and author of over 140 articles in major international journals. He was born in Naples in 1953.
The Gene Expression Unit is part of the large San Raffaele effort in stem cell and gene therapy. Research activities are focused on the basic mechanisms of gene transfer, the interaction between viral vectors and the human genome, the design of new viral vectors, and the establishment of pre-clinical and clinical models for gene therapy of genetic and acquired disorders. The Unit is also interested in the basic mechanisms of gene regulation, and particularly in the genetic programs underlying self-renewal and differentiation of adult stem cells
1) Recchia A, Bonini C., Magnani Z., Urbinati F., Sartori D., Muraro S., Tagliafico E., Bondanza A., Lupo Stanghellini M.T., Bernardi M., Pescarollo A., Ciceri F., Bordignon C., Mavilio F. (2006). Retroviral vector integration deregulates gene expression but has no consequence on the biology and function of transplanted T cells. Proc. Natl. Acad Sci. USA, 103:1457-1462Barbaro V., Testa A., Di Iorio E., Mavilio F., Pellegrini G., De Luca M. (2007). C/EBP regulates cell cycle and self-renewal of human limbal stem cells. J. Cell Biol. 177:1037-1049

2) Mavilio F., Pellegrini G., Ferrari S., Di Nunzio F., Di Iorio E., Recchia A., Maruggi G., Ferrari G., Provasi E., Bonini C., Capurro S., Conti A., Magnoni C., Giannetti A., De Luca, M. (2006). Correction of junctional epidermolysis bullosa by transplantation of genetically modified epidermal stem cells. Nature Med. 12:1397-1402

3) Aiuti A., Cassani B., Andolfi G., Mirolo M., Biasco L., Recchia A., Urbinati F., Valacca C., Scaramuzza S., Cazzola M., Sartori D., Ambrosi A., Di Serio C., Roncarolo M.G., Mavilio F., and Bordignon C. (2007). Multilineage hematopoietic reconstitution without clonal selection in ADA-SCID patients treated with stem cell gene therapy. J. Clin. Invest., 117:2233-2240

4) Cattoglio C., Facchini G., Sartori D., Antonelli A., Miccio A., Cassani B., Schmidt M., von Kalle C., Howe S., Thrasher A.J., Aiuti A., Ferrari G., Recchia A., Mavilio F. (2007). Hot spots of retroviral integrations in human CD34+ hematopoietic cells. Blood, 110:1770-1778

5) Felice B., Cattoglio C., Cittaro D., Testa A., Miccio A., Ferrari G., Luzi L., Recchia A., Mavilio F. (2009) Transcription factor binding sites are genetic determinants of retroviral integration in the human genome. PLoS One 4:e4571

6) Cassani B., Montini E., Maruggi G., Ambrosi A., Mirolo M., Selleri S., Biral E., Frugnoli I., Hernandez-Trujillo V., Di Serio C., Roncarolo, M.G. Naldini L., Mavilio F., Aiuti A. (2009). Integration of retroviral vectors induces minor changes in the transcriptional activity of T cells from ADA-SCID patients treated with gene therapy. Blood 114: 3546-3556

7) Gabriel R., Eckenberg R., Paruzynski A., Bartholomae C.C., Nowrouzi A., Arens A., Howe S.J., Recchia A., Cattoglio C., Wang W., Faber K., Schwarzwaelder K., Kirsten R., Deichmann A., Ball C.R., Balaggan K.S., Yáñez-Muñoz R.J., Ali R.A., Gaspar H.B., Biasco L., Aiuti A., Cesana D., Montini E., Naldini L., Cohen-Haguenauer O., Mavilio F., Thrasher A.J., Glimm H., von Kalle C., Saurin W., Schmidt M. (2009). Comprehensive genomic access to vector integration in clinical gene therapy. Nature Med. 15:1431-1436

8) Roselli E.A., Mezzadra R., Frittoli M.C., Maruggi G., Biral E., Mavilio F., Mastropietro F., Amato A., Tonon G., Refaldi C., Cappellini M.D., Andreani M., Lucarelli G., Roncarolo M.G., Marktel S., Ferrari G. (2010). Correction of ß-thalassemia major by gene transfer in hematopoietic progenitors of pediatric patients. EMBO Mol. Med., 8:315-328

9) Cattoglio C., Pellin D., Rizzi E., Maruggi G., Corti G., Miselli F., Sartori D., Guffanti A., Di Serio C., Ambrosi A., De Bellis G. and Mavilio F. (2010). High-definition mapping of retroviral integration sites identifies active regulatory elements in human multipotent hematopoietic progenitors. Blood, 116:5507-5517

10) Cattoglio C., Maruggi G., Bartholomae C., Malani N., Pellin D., Cocchiarella F., Magnani Z., Ciceri F., Ambrosi A., von Kalle C., Bushman F.D., Bonini C., Schmidt M., Mavilio F., Recchia A. (2010). High-definition mapping of retroviral integration sites defines the fate of allogeneic T cells after donor lymphocyte infusion. PLoS One, 5:e15688
Project Title:
Molecular circuitry in human somatic stem cells
The molecular mechanisms underlying fundamental characteristics of human somatic stem cells, such as self-renewal, commitment and differentiation, are still poorly understood. A better knowledge of these mechanisms is crucial to the understanding of stem cell biology and to the development of stem cell-based gene therapy and regenerative medicine. By analyzing genome accessibility to nucleases and viral pre-integration complexes, gene expression profiles, and genome-wide utilization of regulatory elements by selected transcription factors, we aim at the identification of genes and regulatory regions involved in self-renewal, commitment and differentiation of hematopoietic, neural end epithelial stem cells. Cells will be analyzed as undifferentiated or at different stages of commitment and differentiation into their hematopoietic, neural and epithelial progeny. Data coming from the different approaches and cells will be integrated to map cell-specific and common regulatory regions (“regulomes”) and generate models of molecular circuitry with a systems biology-like approach.