Ferrari Giuliana Professore ordinarioMedicineBIO/11




1985: PhD in Biological Science, University of Milan, Italy


1985-1987: Post-doctoral fellow, Wistar Institute of Anatomy and Biology, Philadelphia, PA, USA.


1987-1989: Research Associate, Experimental Oncology D, Istituto Nazionale Tumori, Milan, Italy.


1989-1992: Staff scientist, Laboratory of Hematology, Scientific Institute H. S. Raffaele, Milan, Italy.


1992-1996: Staff scientist, Laboratory of Gene Expression, Department of Biology and Technology Research, Scientific Institute H. S. Raffaele.


1996- 2001: Group leader, HSR-Telethon Institute of Gene Therapy (TIGET), Scientific Institute H. S. Raffaele.


2001-present: Head, Gene transfer into Stem Cells Unit, HSR-TIGET, Scientific Institute H. S. Raffaele.


2002-2005: Adjunct Professor of Molecular Biology, HSR Medical School, University Vita-Salute, Milan, Italy.


2008-2015: Research Coordinator, HSR-TIGET, Scientific Institute H. S. Raffaele.


2009-present: Member of the International PhD Program in Molecular Medicine, Univeristy Vita-Salute San Raffaele, Medical School


2010-present: Member of the Committee for the Appointment and Promotions, Scientific Institute H.S. Raffaele


2012-present: Study Director, Tiget GLP Facility


2014-present: Member of International Post Doctoral Committee, University Vita- Salute san Raffaele, Medical School


2006-2017: Associate Professor of Molecular Biology, HSR Medical School, University Vita-Salute.


2017 – present: Full Professor of Molecular Biology, HSR Medical School, University Vita- Salute.


Scientific Activity


Giuliana Ferrari, Ph.D is Head of Unit at Scientific Institute San Raffaele in SR-TIGET and Associate Professor of Molecular Biology and Gene Expression at UniSR Medical School.

  1. Ferrari started her career working with F. Mavilio and C. Bordignon by developing retroviral vectors for gene transfer in human cells and preclinical models for gene therapy. The first disease targeted by their team was ADA-SCID. In 1991, they were the first to develop a murine model proving the selective survival of human, genetically corrected lymphocytes in a SCID background (Ferrari et al., Science 251:1363, 1991). These data were instrumental in convincing the NIH Recombinant Advisory Committee (RAC) and the FDA to approve the first clinical application of gene therapy on ADA-SCID patients, which started at the NIH at the end of 1990. Soon after, a similar protocol was started in Italy at San Raffaele Hospital in Milan, with the substantial improvement of using bone marrow-derived stem cells rather than peripheral blood lymphocytes (see Nature, 356:465, 1992). The results of these pioneer studies were published back-to-back in 1995 (Bordignon et al., Science 270:470, 1995) and the success of immune reconstitution was published in 2002 (Aiuti et al., Nat. Med. 8:423, 2002). Long term follow-up of treated ADA-SCID patients demonstrates safety and efficacy of gene therapy using the originally developed retroviral vector. At the same time, G. Ferrari worked at the development of vectors for a safe surface marker that could be used to purify and trace T-cells in patients. This marker, a truncated form of the NGF receptor (Mavilio et al., Blood 83:1988, 1994) was successfully used for a second, pioneer clinical application of gene therapy, the prevention of graft-versus-host disease in allogeneic bone marrow transplantation by immunomodulation of T-cell function with a TK transgene (Bonini et al., Science 276:1719, 1997; Bonini et al., Nat. Med. 9:367, 2003). Meanwhile, she also contributed to develop vectors and preclinical data (Mathor et al., PNAS 1996; Dellambra et al., Hum. Gene Ther. 11:2283, 2000), in collaboration with M. De Luca, a leading epidermal stem cell biologist, for the first gene therapy clinical trial for genetic skin adhesion disorder epidermiolysis bullosa. The efficacy of autologous transplantation of genetically corrected epidermal stem cells to treat epidermolysis bullosa was later proved in a seminal clinical study, published in 2006 (Mavilio et al., Nat. Med. 12:1397, 2006).


Along with her research in the field of applied science, G. Ferrari continued to be interested in fundamental stem cell research. In collaboration with G. Cossu, a leading muscle stem cell biologist, she published in 1998 the first evidence of plasticity of adult hematopoietic stem cells, able to regenerate damaged muscle tissues under certain conditions (Ferrari et al., Science 279:1528, 1998). This paper originated a whole new concept in stem cell biology and regenerative medicine, and is still one of the most cited papers in the field (2163 citations). Her contribution in defining potential and limitation of stem cell plasticity, as well as molecular mechanisms of the phenomenon, continued throughout years (Ferrari et al., Nature 411:1014, 2001; McKinney et al., PNAS 99:1341, 2002; Xynos et al., Stem Cells 28:965, 2010).


Exploiting her expertise in molecular biology and gene transfer technology, starting from the late ‘90s G. Ferrari developed strategies of transcriptional targeting to regulate transgene expression by retroviral and lentiviral vectors in the differentiated progeny of hematopoietic stem cells (Grande et al., Blood 93:3276, 1999; Lotti et al., J. Virol. 76:3996, 2002; Testa et al., J. Biol. Chem. 279: 10523, 2004). The challenge for vector-mediated transcriptional regulation being beta-thalassemia, her recent research was devoted to bring to clinic the use of regulated beta-globin lentiviral vectors, preclinical efficacy and safety data that led to the recent approval of the clinical trial application (Miccio et al., PNAS 105:10547, 2008; Roselli et al., EMBO Mol. Med. 2:315, 2010; Frittoli et al. Hum. Gene Ther. 22: 507, 2011; Adreani et al. Haematologica 96: 128, 2011; Miccio et al. PLOS ONE 6: e27955, 2012). In 2011 Giuliana Ferrari was coordinator in the creation of the first academic GLP Test Facility for preclinical studies of advanced therapies, which has been certified by Italian Minister of Health in 2014. In this context, she is study director of toxicology studies and tumorigenesis studies for gene therapy. She is the leader of the beta thalassemia project, including the clinical protocol of gene therapy (TIGET-BTHAL, NCT02453477), started in 2015.

Her current basic research projects are focused on hematopoietic stem cell biology (Cattoglio et al. Blood 110: 1770, 2007; Felix et al. PlosOne 4: e4751, 2009; Moiani et al. Journal Clin. Invest., 122: 1653, 2012), vector-genome interactions and molecular control of erythropoiesis (Cantu et al. Blood 117: 3669, 2011; Nai et al. Blood, 119: 5021, 2012)



Editorial Activity

Human Gene Therapy: Associate European Editor (1995-1997).

The Journal of Gene Medicine: Member of Editorial Board (1999-present).



European Society of Gene Therapy (ESGCT)

American Society of Gene Therapy (ASGCT)

American Society of Hematology (ASH)




  • Lidonnici MR and Ferrari G. Gene therapy and gene editing strategies for hemoglobinopathies. Blood Cells Mol Dis. 2018 May;70:87-101. Epub 2018 Jan 3


  • Ferrari G., Cavazzana M. and Mavilio F. Gene therapy approaches to hemoglobinopathies. (2017). Hematol Oncol Clin N Am 31:835–852.


  • Lidonnici MR, Aprile A., Frittoli MC, Mandelli G, Paleari Y, Spinelli A, Gentner B, Zambelli M, Parisi C, Bellio L, Cassinerio E, Zanaboni L, Cappellini MD, Ciceri F, Marktel S and Ferrari G. (2017). Plerixafor and G-CSF combination mobilizes hematopoietic stem and progenitors cells with a distinct transcriptional profile and a reduced in vivo homing capacity compared to Plerixafor alone. Haematologica 102(4):e120-e124.


  • Zonari E., Desantis G., Petrillo C., Boccalatte FE., Lidonnici MR., Kajaste-Rudnitski A., Aiuti A., Ferrari G., Naldini L., and Gentner B. (2017). Efficient ex vivo engineering and expansion of highly purified human hematopoietic stem/progenitor cell populations for gene therapy. Stem Cell Reports 8:977-990


  • Aprile A., Passerini G., Cappellini M.D.,  Marktel S., Ciceri F., Ferrari G. and  Ceriotti F. (2016). When diagnostics meets translational research: detection of hemoglobin fractions in cellular lysates from in vitro erythroid cultures by Capillarys2 Flex Piercing® analyzer (Sebia). Res. 169: 31-39.e4.


  • Romano O., Peano C., Malagoli Tagliazucchi G., Petiti L., Poletti V., Cocchiarelli F., Rizzi E., Severgnini M., Cavazza A., Rossi C., Pagliaro P., Ambrosi A., Ferrari G., Bicciato S., De Bellis G., Mavilio F. and Miccio A. (2016). Transcriptional, epigenetic and retroviral integration signatures identify regulatory regions involved in lineage commitment of human hematopoietic stem/progenitor cells. Scientific Report 6:24724. doi: 10.1038/srep24724.


  • Nai A.*, Lidonnici MR.*, Rausa M., Mandelli G., Pagani A., Silvestri L., Ferrari G., Camaschella C. (2015). The second transferrin receptor regulates red blood cell production in mice. Blood 125:1170-9.* equal contribution.


  • Nai A., Pagani A., Mandelli G., Lidonnici M.R., Silvestri L., Ferrari G., Camaschella C. (2012) Deletion of Tmprss6 attenuates the phenotype in a mouse model of ß-thalassemia. Blood 119:5021-5029.


  • Moiani A., Paleari Y., Sartori D., Mezzadra R., Miccio A., Cattoglio C., Cocchiarella F., Lidonnici M.R., *Ferrari G., *Mavilio F. (2012) Lentiviral vector integration in the human genome induces alternative splicing and generates aberrant transcripts. J Clin Invest. 122:1653-66. *co-corresponding authors


  • Roth J.C., Ismail M., Reese J.S., Lingas K.T., Ferrari G., Gerson S.L. (2012) Co-transduction with MGMT and Ubiquitous or Erythroid-Specific GFP Lentiviruses Allows Enrichment of Dual-Positive Hematopoietic Progenitor Cells In Vivo. ISRN 2012; 2012:212586. Epub 2012 Jul 9


  • Andreani M., Testi M., Gaziev J., Condello R., Bontadini A., Tazzari PL., Ricci F., De Felice L., Agostini F., Fraboni D., Ferrari G., Battarra M., Troiano M., Sodani P., Lucarelli G. (2011) Quantitatively different red cell/nucleated cell chimerism in patients with long-term, persistent hematopoietic mixed chimerism after bone marrow transplantation for thalassemia major or sickle cell disease. Haematologica 96:128-33.


  • Frittoli MC., Biral E., Cappelli B., Zambelli M., Roncarolo MG., Ferrari G., Ciceri F., Marktel S. (2011) Bone marrow as source of hematopoietic stem cells for human gene therapy of beta thalassemia. Hum Gene Ther. 22:507-13.


  • Cantù C., Ierardi R., Alborelli I., Fugazza C., Cassinelli L., Piconese S., Bosè F., Ottolenghi S., Ferrari G., Ronchi A. (2011) Sox6 enhances erythroid differentiation in human erythroid progenitors. Blood 117:3669-3679


  • Miccio A., Poletti V., Tiboni F., Rossi C., Antonelli A., Fulvio Mavilio F. and Ferrari G. (2011). The GATA1-HS2 enhancer allows persistent and position-independent expression of a ß-globin transgene. PLoS One Epub 6: e27955


  • Sordi V., Melzi R., Mercalli A., Formicola R., Doglioni C., Tiboni F., Ferrari G., Nano R., Chwalek K., Borg D., Lammert E., Bonifacio E., Piemonti L. (2010) Mesenchymal Cells Appearing in Pancreatic Tissue Culture are Bone Marrow-Derived Stem Cells With the Capacity to Improve Transplanted Islet Function. Stem Cells. 28:140-151.


  • Frenquelli M., Muzio M., Scielzo C., Fazi C., Scarfo’ L., Rossi C., Ferrari G., Ghia P., Caligaris-Cappio F. (2010) MicroRNA and proliferation control in chronic lymphocytic leukemia: functional relationship between miR-221/222 cluster and p27. Blood. 115:3949-3959.


  • Xynos A., Corbella P., Belmonte N., Zini R., Manfredini R, Ferrari G. (2010). Bone marrow-derived hematopoietic cells undergo myogenic differentiation following a Pax-7 independent pathway. Stem Cells. 28:965-973.


  • Roselli EA., Mezzadra R., Frittoli MC., Maruggi G., Biral E., Mavilio F., Mastropietro F., Amato A., Tonon G., Refaldi C., Cappellini MD., Andreani M., Lucarelli G., Roncarolo MG., Marktel S., Ferrari G. (2010) Correction of ß-thalassemia major by gene transfer in hematopoietic progenitors of pediatric patients. EMBO Mol Med. 2:315-328.


  • Cantù C., Grande V., Alborelli I., Cassinelli L., Cantù I., Colzani MT., Ierardi R., Ronzoni L., Cappellini MD., Ferrari G., Ottolenghi S., Ronchi A. (2010) A Highly conserved SOX6 double binding site mediates SOX6 gene downregulation in erythroid cells. Nucl Acid Res. 39:486-501


  • 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.


  • Cantarelli E., Melzi R., Mercalli A., Sordi V., Ferrari G., Lederer CW., Mrak E., Rubinacci A., Ponzoni M., Sitia G., Guidotti LG., Bonifacio E., Piemonti L. (2009) Bone marrow as an alternative site for islet transplantation. 114:4566-4574.


  • Mavilio F. and Ferrari G. (2008). Genetic modification of somatic stem cells. The progress, problems and prospects of a new therapeutic technology. EMBO Rep. 9:64-69.


  • Miccio A., Cesari R., Lotti F., Rossi C., Sanvito F., Ponzoni M., Routledge SJ., Chow CM., Antoniou MN., Ferrari G. (2008). In vivo selection of genetically modified erythroblastic progenitors leads to long-term correction of beta-thalassemia. Natl. Acad. Sci. U S A. 105:10547-10552.


  • Cattoglio C., Facchini G., Sartori D., Antonelli A., Cassani B., Schmidt M., von Kalle C., Thrasher A., Aiuti A., Ferrari G., Recchia A. and Mavilio F. (2007). Hot spots of retroviral integration in CD34+ hematopoietic stem/progenitor cells. 110:1770-1778.


  • Di Rosa P., Villaescusa J.C., Longobardi E, Lotti G., Ferretti E., Diaz V.M., Miccio A., Ferrari G. and Blasi F. (2007). The homeodomain transcription factor Prep1 (pKnox1) is required for hematopoietic stem and progenitor cell activity. Biol. 311:324-334.


  • Ferretti E., Villaescusa J.C., Di Rosa P., Fernandez-Diaz L.C., Longobardi E., Mazzieri R., Miccio A., Micali N., Selleri L., Ferrari G. and Blasi F. (2006). Hypomorhic mutation of the TALE gene Prep1 (pKnox1) causes a major reduction of Pbx and Meis proteins and a pleiotropic embryonic phenotype. Mol Cell. Biol. 26:5650-5662.


  • Menegon A., Bonanomi D., Albertinazzi C., Lotti F., Ferrari G., Kao H.T., Benfenati F. and Valtorta F. (2006). Protein kinase A-mediated synapsin I phosphorylation is a central modulator of Ca2+-dependent synaptic activity. Neurosci. 26:11670-11681.


  • 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. and De Luca M. (2006). Correction of junctional epidermolysis bullosa by transplantation of genetically-modified epidermal stem cells. Med. 12:1397-1402.


  • Lemoli R.M., Bertolini F., Cancedda R., De Luca M., Del Santo A., Ferrari G., Ferrari S., Martino G, Mavilio F. and Tura S. (2005). Stem cell plasticity: time for a reappraisal? 90:360-381.


  • Sordi V., Malosio M.L., Marchesi F., Mercalli A., Melzi R., Giordano T., Belmonte N., Ferrari G., Leone B.E., Bertuzzi F., Zerbini G., Allavena P., Bonifacio E., and Piemonti L. (2005). Bone marrow mesenchymal stem cells express a restricted set of functionally active chemokine receptors capable of promoting migration to pancreatic islets. Blood. 106:419-427.


  • Urbinati F., Lotti F., Facchini G., Montanari M., Ferrari G., Mavilio F. and Grande A. (2005). Competitive engraftment of hematopoietic stem cells genetically modified with a truncated erythropoietin receptor. Gene Ther. 16:594-608.


  • Bonanomi D., Menegon A., Miccio A., Ferrari G., Benfenati F., Kao H.T., Greengard P., Corradi A. and Valtorta F. (2005). Phosphorilation of synapsin I by cyclic AMP-dependent protein kinase controls synaptic vesicle dynamics in developing neurons. Neurosci. 25:7299-7308.


  • Testa A, Lotti F, Cairns L, Grande A, Ottolenghi S, Ferrari G, Ronchi A. (2004). Deletion of a negatively acting sequence in a chimeric GATA-1 enchancer-LTR greatly increases retroviral-mediated erythroid expression. J Biol Chem. 279:10523-31.


  • Bonini C., Grez M., Traversari C., Ciceri F., Marktel S., Ferrari G., Dinauer M., Sadat M., Aiuti A., Deola S., Radrizzani M., Hagenbeek A., Apperley J., Ebeling S., Martens A., Kolb HJ., Weber M., Lotti F., Grande A., Weissinger E., Bueren JA., Lamana M., Falkenburg JH., Heemskerk MH., Austin T., Kornblau S., Marini F., Benati C., Magnani Z., Cazzaniga S., Toma S., Gallo-Stampino C., Introna M., Slavin S., Greenberg PD., Bregni M., Mavilio F., Bordignon C. (2003). Safety of retroviral gene marking with a truncated NGF receptor. Med. 9:367-69.


  • Lotti F., Menguzzato E., Rossi C., Naldini L., Ailles L., Mavilio F. and Ferrari G. (2002). Transcriptional targeting of lentiviral vectors by LTR enhancer replacement. Virol. 76: 3996-4007.
  • McKinney-Freeman S.L., Jackson K.A., Camargo F.D., Ferrari G., Mavilio F. and Goodell M.A. (2002). Muscle-derived hematopoietic stem cells are hematopoietic in origin. Natl. Acad. Sci. USA. 99:1341-1346.


  • Aiuti A., Vai S., Mortellaro A., Casorati G., Ficara F., Andolfi G., Ferrari G., Tabucchi A., Carlucci F., Marinello E., Marziliano N., Bernardi M., Notarangelo L., Ochs H.D., Notarangelo L.D., Roncarolo M.G. and Bordignon C. (2002). Immune reconstitution in ADA-SCID after PBL gene therapy and discontinuation of enzyme replacement. Med. 8:423-425.


  • Ferrari G. and Mavilio F. (2002). Myogenic stem cells from the bone marrow: a therapeutic alternative for muscular dystrophy? Neuromuscular Disord. 12:S7-S10.


  • Ferrari G., Stornaiuolo A. and Mavilio F. (2001). Failure to correct murine muscular dystrophy. 411:1014-1015.


  • Rogge L., Bianchi E., Biffi M., Bono E., Chang S.P., Alexander H., Santini C., Ferrari G., Sinigaglia L., Seiler M., Neeb M., Mous J., Sinigaglia F. and Certa U. (2000). Transcript imaging of human T helper cell development using oligonucleotide arrays. Genet. 25:96-101.


  • Dellambra E., Pellegrini G., Guerra L., Ferrari G., Zambruno G., Mavilio F. and De Luca M. (2000). Toward epidermal stem cell-mediated ex vivo gene therapy of junctional epidermiolysis bullosa. Gene Ther. 11.2283-2287.


  • Grande A., Piovani B., Aiuti A., Ottolenghi S., Mavilio F. and Ferrari G. (1999). Transcriptional targeting of retroviral vectors to the erythroblastic progeny of transduced hematopoietic stem cells. Blood. 93: 3276-3285.


  • Berghella L., De Angelis L., Coletta M., Berarducci B., Sonnino C., Salvatori G., Anthonissen C., Couper R., Butler-Brown G.S., Mouly V., Ferrari G., Mavilio F. and Cossu G. (1999). Reversible immortalization of human primary myoblasts by site-specific excision of a retrovirally-transferred oncogene. Gene Ther. 10: 1607-1617.


  • Lattanzi L., Salvatori G., Coletta M., Sonnino C., Cusella De Angelis M.G., Gioglio L., Murry C.E., Kelly R., Ferrari G., Molinaro M., Crescenzi M., Mavilio F. and Cossu G. (1998). High efficiency myogenic conversion of human fibroblasts by adenoviral vector-mediated MyoD gene transfer. An alternative strategy for ex vivo gene therapy of primary myopathies. Clin. Invest. 101: 2119-2128.


  • Ferrari G., Cusella De Angelis M.G., Coletta M., Paolucci E., Stornaiuolo A., Cossu G. and Mavilio F. (1998). Muscle regeneration by bone marrow-derived myogenic progenitors. 279: 1528-1530.


  • Ferrari G. and Mavilio F. (1998). Retroviral vectors for human gene therapy. Gene Ther. NATO ASI Series vol.105, (K.G. Xanthopoulos Ed.) Springer Verlag, 119-132.


  • D’Aloja P., Olivetta E., Bona R., Nappi F., Pedacchia D., Pugliese K., Ferrari G., Verani P., Federico M. (1998). gag, vif, and nef genes contribute to the homologous viral interference induced by a nonproducer human immunodeficiency virus type 1 (HIV-1) variant: identification of novel HIV-1-inhibiting viral protein mutants. Virol. 72: 4308-4319.


  • Bonini C., Ferrari G., Verzeletti S., Servida P., Zappone E., Ruggieri L., Ponzoni M., Rossini S., Mavilio F., Traversari C., Bordignon C. (1997). HSV-tk gene transfer into donor lymphocytes for controlled allogeneic graft versus leukemia. 276: 1719-1724.


  • Ruggieri L., Aiuti A., Salomoni M., Zappone E., Ferrari G., Bordignon C. (1997). Cell surface marking of CD34+ DR+/DR- restricted phenotypes of human hematopoietic progenitor cells by retroviral-mediated gene transfer. Gene Ther. 8:1611-1623.


  • Bona R., D’Aloja P., Olivetta E., Modesti A., Modica A., Geraci A., Ferrari G., Verani P., Federico M. (1997). Aberrant, noninfectious HIV-1 particles are released by chronically infected human T cells transduced with a retroviral vector expressing an interfering HIV-1 variant. Gene Ther. 4:1085-1092.


  • Mathor M.B., Ferrari G., Dellambra E., Cilli M., Mavilio F., Cancedda R., De Luca M. (1996). Clonal analysis of stably transduced human epidermal stem cells in culture. Natl. Acad. Sci. USA. 93: 10371-10376.


  • Ferrari G., Salvatori G., Rossi C., Cossu G., Mavilio F. (1995). A retroviral vector containing a muscle-specific enhancer drives gene expression only in differentiated muscle fibers. Gene Ther. 6: 733-742.


  • Silvani A., Ferrari G., Paonessa G., Toniatti C., Parmiani G., Colombo M.P. (1995). Down regulation of IL-6Ra chain in IL-6-transduced melanoma cells causes selective resistance to IL-6- but not to oncoststin M. Cancer Res. 55: 2200-2205.


  • Salvatori G., Lattanzi L., Coletta M., Aguanno S., Vivarelli E., Kelly R., Ferrari G., Harris A.J., Mavilio F., Molinaro M., Cossu G. (1995). Myogenic conversion of mammalian fibroblasts induced by differentiating muscle cells. Cell Sci. 108:2733-2739.


  • Federico M., Nappi F., Ferrari G., Chelucci C., Mavilio F. and Verani P. (1995). A non-producer interfering HIV-1 provirus can be transduced through a MLV-based retroviral vector: recovery of an anti-HIV mouse/human pseudotype retrovirus. Virol. 69: 6618-6626.


  • Bordignon C., Notarangelo L.D., Nobili N., Ferrari G., Casorati G., Panina P., Mazzolari E., Maggioni D., Rossi C., Servida P., Ugazio A.G., Mavilio F. (1995). Gene Therapy in Peripheral Blood Lymphocytes and Bone Marrow for ADA-Immunodeficient Patients. 270: 470-475.


  • Mavilio, F., Ferrari G., Rossini S., Nobili N., Bonini C., Casorati G., Traversari C., Bordignon C. (1994). Peripheral blood lymphocytes as target cells of retroviral vector-mediated gene transfer. Blood. 83: 1988-1997.


  • Pericle F., Giovarelli M., Colombo M.P., Ferrari G., Musiani P., Modesti A. Cavallo F. Di Pierro F., Novelli F., Forni G. (1994). An efficient Th2-type memory follows CD8+lymphocyte driven and eosinophil mediated rejection of a spontaneous mouse mammary adenocarcinoma engineered to release IL-4. Immunol.153: 5659-5673.


  • Bordignon C., Mavilio F., Ferrari G., Servida P., Ugazio A.G., Notarangelo L.D., Gilboa E. (1993) Clinical protocol: transfer of the ADA gene into bone marrow cells and peripheral blood lymphocytes for the treatment of patients affected by ADA-deficient SCID. Gene Ther. 4: 513-520.


  • Salvatori G., Ferrari G., Mezzogiorno A., Servidei S., Coletta M., Tonali P., Giavazzi R., Cossu G., Mavilio F. (1993). Retroviral vector-mediated gene transfer into human primary myogenic cells leads to expression into muscle fibers in vivo. Gene Ther. 4: 713-723.


  • Ferrari G., Rossini S., Nobili N., Maggioni D., Garofalo A., Giavazzi R., Mavilio F., Bordignon C. (1992). Transfer of the ADA gene into human ADA-deficient T lymphocytes reconstitutes specific immune function. Blood. 80: 1120-1124.


  • Ferrari G., Rossini S., Giavazzi R., Maggioni D., Nobili N., Soldati M., Ungers G., Mavilio F., Gilboa E. and Bordignon C. (1991). An in vivo model of Somatic cell gene therapy for human severe combined immunodeficiency. Science. 251: 1363-1366.


  • Colombo M.P., Ferrari G., Stoppacciaro A., Parenza M., Rodolfo M., Mavilio F. and Parmiani G. (1991). Granulocyte colony-stimulating factor (G-CSF) gene transfer suppresses tumorigenicity of a murine adenocarcinoma in vivo. Exp. Med. 173: 889-897.

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