Ferrari Giuliana Professore OrdinarioMedicineBIOS-08/A

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.

1990. 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).

 

Memberships

European Society of Gene Therapy (ESGCT)

American Society of Gene Therapy (ASGCT)

American Society of Hematology (ASH)