Europass Curriculum Vitae

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1 Europass Curriculum Vitae Personal information First name(s) / Surname(s) Barbara Cassani Address(es) Via Aldo Moro 8/831, 20090, CUSAGO (MI), ITALY Telephone(s) Mobile: bcassani1312@gmail.com Nationality Italian Date of birth 12/13/1976 Gender Female Work experience Dates From 09/2012 Junior PI IRGB-National Research Council (CNR) UOS Milan c/o Humanitas Clinical Institute Via Manzoni 113, Rozzano (MI), ITALY Immunology and translational medicine Main activities and responsibilities Dates 06/ /2012 Research Scientist Massachusetts General Hospital, Harvard Medical School Laboratory of Leukocytes trafficking in health and disease. Advisor: Prof. J Rodrigo Mora 55 Fruit St, GRJ 815, Boston, MA 02114, USA Mucosal Immunology Dates 02/ /2010 Post-doctoral Fellow Fondazione Humanitas per la Ricerca-ICH Laboratory of Human Genome TBI-CNR. Advisor: Dr. Anna Villa Via Manzoni 113, Rozzano (MI), ITALY Immunology, molecular biology and translational medicine Dates 08/ /2008 Post-doctoral Fellow Project title: Safety studies of ADA-SCID stem cell gene therapy. In charge of research experimental designing, findings evaluation, collaborative activities, papers writing, and supervision PhD students. San Raffaele Telethon Institute for Gene Therapy (HSR-TIGET) c/o DIBIT Laboratory of Gene therapy for Primary Immunodeficiency. Advisor: Prof. Alessandro Aiuti Via Olgettina 58, Milan, ITALY Immunology, molecular biology and translational medicine Dates 07/ /2007 PhD Student Page 1/6 - Curriculum vitae of

2 Vita-Salute San Raffaele University Laboratory HSR-TIGET Gene therapy for Primary Immunodeficiency. Advisor: Prof. Alessandro Aiuti and Prof. Maria Grazia Roncarolo Via Olgettina 58, Milan, ITALY Immunology, molecular biology and translational medicine Education and training Dates 03/ /2003 Research Fellow San Raffaele Telethon Institute for Gene therapy (HSR-TIGET) Laboratory Gene therapy for Primary Immunodeficiency. Advisor: Prof. Alessandro Aiuti and Prof. Maria Grazia Roncarolo Via Olgettina 58, Milan, ITALY Immunology Dates 09/ /2001 Visiting Scholar Universita degli Studi Laboratory Molecular and Cellular Pharmacology of Purinergic Transmission. Advisor: Prof. Flaminio Cattabeni and Prof. Maria Pia Abbracchio Via Celoria 21, Milan, ITALY Pharmacology Dates PhD Dates 2005 Dates 2002 Dates 2001 Molecular and Cellular Biology Vita-Salute San Raffaele University Laboratory HSR-TIGET Gene therapy for Primary Immunodeficiency. Selected Participant Signal Transduction in the immune system FASEB Summer School, Aspen, Colorado (USA) Professional qualification Pharmacy Universita degli studi, Milan, ITALY BSc in Pharmacy Pharmacology Universita degli studi, Milan, ITALY Dates Dates 1995 Visiting Scholar Molecular and Cell Biology Universita degli studi, Milan, ITALY High School degree Science Page 2/6 - Curriculum vitae of

3 Honours Liceo Scientifico D. Bramante, Magenta (MI), ITALY 2012 BioEconomy Prize on Rare Disease (Rome, Italy) 2011 Travel Award, Annual Meeting Society of Mucosal Immunology (Paris, France) 2009 Marie Curie Travel Award, TRiPR III-Genoa, Italy 2006 Travel Award, American Society of Haematology (Orlando, USA) Grants and Fellowships Grant FIRB- MIUR n. RBFR12I3UB Long-term Fellowship, European Molecular Biology Organization (EMBO) Fellowship, Consiglio Nazionale delle Ricerche (ITB-CNR) 2008: Grant n Fondazione Cariplo per la Ricerca Fellowship, PhD program, HSR-TIGET, Milan Personal skills and competences Mother tongue(s) Italian Other language(s) English Self-assessment European level (*) Understanding Speaking Writing ENGLISH Listening Reading Spoken interaction Spoken production C2 Proficient user C2 Proficient user C2 Proficient user C2 Proficient user C2 Proficient user Scientific Interests Organisational skills and competences Inherited Primary Immunodeficiency, gene transfer, Immunological Tolerance, Mucosal Immunology Team spirit, Leadership Laboratory and Research Supervisory Training Responsibilities Grant and proposal writing Grant Review activities Editorial activities: Ad-hoc reviewer for Journal of Allergy and Clinical Immunology, Mucosal Immunology, Journal of Immunology, Gut, Gastroenterology, Journal of Clinical Investigation, Journal of Leukocyte Biology, European Journal of Immunology Assistant, Laboratory Practical course in Molecular Paediatrics, Vita-Salute San Raffaele University, Milan, Italy Daily mentorship for graduate students, technician, PhD student, Post-doctoral fellow 2013 Ad-hoc Reviewer National Ministry for Education, University and Research (FIR Grants), Italy Page 3/6 - Curriculum vitae of

4 CONGRESS Oral Communications National Meetings 2006 Invited Lecture (Job talk): Genomic Impact of Retroviral Integration in ADA-SCID. Host: Prof. Fulvio Mavilio, Universita di Modena e Reggio-Emilia, Modena, Italy Invited Lecture (Job talk): Role of B cells in the pathogenesis of Omenn Syndrome. Host: Dr. Elisabetta Traggiai, Istituto Clinico G. Gaslini, Genova, Italy Abstract presentation (short talk): Role of B cells in the pathogenesis of Omenn Syndrome. TRiPR III - Adaptive Immunity and the Pathogenesis of Rheumatic Disease, Genoa, Italy. International Meetings 2006 Abstract presentation (short talk): Vector integration studies in ADA-SCID. Consert meeting on Gene Therapy of Inherited Disease, Leukerbad, Switzerland 2007 Abstract presentation (short talk): Clonal analysis of retroviral vector integration in ADA-SCID patients treated with stem cell gene therapy. Consert meeting on Gene Therapy of Inherited Disease, Leukerbad, Switzerland 2007 Abstract presentation (short talk): Exploring the influence of retroviral integration site selection on gene expression in ADA-SCID patients treated with stem cell gene therapy. Annual meeting of American Society of Gene Therapy (ASGT), Seattle, WA, USA Invited Lecture (Job talk): Molecular mechanisms of immune deficiency in adenosine deaminase-deficient SCID patients: implications for stem cell gene therapy. Host: Dr. Silvia Monticelli Institute for Research in Biomedicine (IRB), Bellinzona, Switzerland 2007 Invited Lecture (Job talk): Molecular mechanisms of immune deficiency in ADA-deficient SCID patients: implications for stem cell gene therapy. Host: Prof. Georg Hollander, University of Basel, Basel, Switzerland 2008 Invited Lecture (Job talk): Molecular mechanisms of immune deficiency in adenosine deaminase-deficient SCID patients: implications for stem cell gene therapy. Host: Dr. Natalie Rufer, Ludwig Institute for Cancer Research, Lausanne, Switzerland 2010 Invited Lecture (Job talk): Role of B cells in the immunopathogenesis of Omenn syndrome. Host: Prof. J. Rodrigo Mora, GastroIntestinal Unit, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA 2010 Invited Lecture (Job talk): Role of B cells in the immunopathogenesis of Omenn syndrome. Host: Prof. Laurie Glimcher, Dr. Antonious Aliprantis, Department of Immunology and Infectious Diseases, Harvard School of Public Health, Boston, Massachusetts, USA 2011 Abstract presentation (short talk): Gut-Specific Lymphocyte Homing as a Key Player in Oral Immunological Tolerance. Keystone Symposium Mucosal Biology: A Fine Balance between Tolerance and Immunity, Vancouver, British Columbia 2011 Abstract presentation (short talk): A critical role of gut-homing T cells in oral immunological tolerance. ICMI th International Congress of Mucosal Immunology, Paris, France Abstract presentation (short talk): Mucosal immune dysregulation in Omenn Syndrome. ICI th International Congress of Immunology, Milan, Italy. Additional information Professional Memberships : European Calcified Tissue Society (ECTS) 2008-PRESENT: European Society for Immunodeficiency (ESID) 2010-PRESENT: Society of Mucosal Immunology Page 4/6 - Curriculum vitae of

5 List of publication H-Index: Isgrò A, Marziali M, Mezzaroma I, Luzi G, Mazzone AM, Guazzi V, Andolfi G, Cassani B, Aiuti A, Aiuti F. Bone marrow clonogenic capability, cytokine production, and thymic output in patients with common variable immunodeficiency. J Immunol Apr 15;174(8): Cattoglio C, Facchini G, Sartori D, Antonelli A, Miccio A, Cassani B, Schmidt M, von Kalle C, Howe S, Thrasher AJ, Aiuti A, Ferrari G, Recchia A, Mavilio F. Hot spots of retroviral integration in human CD34+ hematopoietic cells. Blood Sep15;110(6): Aiuti A, Cassani B, Andolfi G, Mirolo M, Biasco L, Recchia A, Urbinati F, Valacca C, Scaramuzza S, Aker M, Slavin S, Cazzola M, Sartori D, Ambrosi A, Di Serio C, Roncarolo MG, Mavilio F, Bordignon C. Multilineage hematopoietic reconstitution without clonal selection in ADASCID patients treated with stem cell gene therapy. J Clin Invest Aug;117(8): Cassani B, Mirolo M, Cattaneo F, Benninghoff U, Hershfield M, Carlucci F, Tabucchi A, Bordignon C, Roncarolo MG, Aiuti A. Altered intracellular and extracellular signaling leads to impaired T-cell functions in ADA-SCID patients. Blood Apr 15;111(8): Guerrini MM, Sobacchi C, Cassani B, Abinun M, Kilic SS, Pangrazio A, Moratto D, Mazzolari E, Clayton-Smith J, Orchard P, Coxon FP, Helfrich MH, Crockett JC, Mellis D, Vellodi A, Tezcan I, Notarangelo LD, Rogers MJ, Vezzoni P, Villa A, Frattini A. Human osteoclast-poor osteopetrosis with hypogammaglobulinemia due to TNFRSF11A (RANK) mutations. Am J Hum Genet Jul;83(1): Aiuti A, Cattaneo F, Galimberti S, Benninghoff U, Cassani B, Callegaro L, Scaramuzza S, Andolfi G, Mirolo M, Brigida I, Tabucchi A, Carlucci F, Eibl M, Aker M, Slavin S, Al-Mousa H, Al Ghonaium A, Ferster A, Duppenthaler A, Notarangelo L, Wintergerst U, Buckley RH, Bregni M, Marktel S, Valsecchi MG, Rossi P, Ciceri F, Miniero R, Bordignon C, Roncarolo MG. Gene therapy for immunodeficiency due to adenosine deaminase deficiency. N Engl J Med Jan 29;360(5): Villa A, Guerrini MM, Cassani B, Pangrazio A, Sobacchi C. Infantile malignant, autosomal recessive osteopetrosis: the rich and the poor. Calcif Tissue Int Jan;84(1): Tondelli B, Blair HC, Guerrini M, Patrene KD, Cassani B, Vezzoni P, Lucchini F. Fetal liver cells transplanted in utero rescue the osteopetrotic phenotype in the oc/oc mouse. Am J Pathol Mar;174(3): Cassani B, Montini E, Maruggi G, Ambrosi A, Mirolo M, Selleri S, Biral E, Frugnoli I, Hernandez- Trujillo V, Di Serio C, Roncarolo MG, Naldini L, Mavilio F, Aiuti A. Integration of retroviral vectors induces minor changes in the transcriptional activity of T cells from ADA-SCID patients treated with gene therapy. Blood Oct 22;114(17): Cassani B, Poliani PL, Moratto D, Sobacchi C, Marrella V, Imperatori L, Vairo D, Plebani A, Giliani S, Vezzoni P, Facchetti F, Porta F, Notarangelo LD, Villa A, Badolato R. Defect of regulatory T cells in patients with Omenn syndrome. J Allergy Clin Immunol Jan;125(1): A Sauer, B Cassani and A Aiuti. Gene therapy for primary immunodeficiency. Book Charpter in A guide to human gene therapy Ed. RW Herzog and S Zolotukhin, World Scientific Publishing Company; 1st edition (May 30, 2010). 12. Cassani B, Poliani PL, Marrella V, Schena F, Sauer AV, Ravanini M, Strina D, Busse CE, Regenass S, Wardemann H, Martini A, Facchetti F, van der Burg M, Rolink AG, Vezzoni P, Grassi F, Traggiai E, Villa A. Homeostatic expansion of autoreactive immunoglobulin-secreting cells in the Rag2 mouse model of Omenn syndrome. J Exp Med Jul 5;207(7): Page 5/6 - Curriculum vitae of

6 13. S. Selleri, I., Brigida, M., Casiraghi, S., Scaramuzza, B. Cappelli, B. Cassani, A. Scarselli, C. Cancrini, S. Marktel, M.G. Roncarolo anda. Aiuti. In vivo hematopoietic cell dynamics during immune reconstitution after hematopoietic stem cell gene therapy in ADA-SCID. J Allergy Clin Immunol Jun;127(6): e EJ. Villablanca*, B. Cassani*, UH. von Andrian and JR. Mora. Blocking Lymphocyte Localization to the Gastrointestinal Mucosa as a Therapeutic Strategy for Inflammatory Bowel Diseases. * equal contribution. Gastroenterology.2011 May 140(6): Deichmann A, Brugman MH, Bartholomae CC, Schwarzwaelder K, Verstegen MM, Howe SJ, Arens A, Ott MG, Hoelzer D, Seger R, Grez M, Hacein-Bey-Abina S, Cavazzana-Calvo M, Fischer A, Paruzynski A, Gabriel R, Glimm H, Abel U, Cattoglio C, Mavilio F, Cassani B, Aiuti A, Dunbar CE, Baum C, Gaspar HB, Thrasher AJ, von Kalle C, Schmidt M, Wagemaker G. Insertion sites in engrafted cells cluster within a limited repertoire of genomic areas after gammaretroviral vector gene therapy. Mol Ther Nov;19(11): Cassani B, Villablanca EJ, Quintana FJ, Love PE, Lacy-Hulbert A, Blaner WS, Sparwasser T, Snapper SB, Weiner HL, Mora JR. Gut-Tropic T Cells That Express Integrin α4β7 and CCR9 Are Required for Induction of Oral Immune Tolerance in Mice. Gastroenterology Dec;141(6): Pangrazio A*, Cassani B*, Guerrini MM, Crockett JC, Marrella V, Zammataro L, Strina D, Schulz A, Schlack C, Kornak U, Mellis DJ, Duthie A, Helfrich MH, Durandy A, Moshous D, Vellodi A, Chiesa R, Veys P, Iacono NL, Vezzoni P, Fischer A, Villa A, Sobacchi C. RANK-dependent autosomal recessive osteopetrosis: characterisation of 5 new cases with novel mutations. * equal contribution. J Bone Miner Res Feb;27(2): Cassani B, Villablanca EJ, De Calisto J, Wang S, Mora JR. Vitamin A and immune regulation: Role of retinoic acid in gut-associated dendritic cell education, immune protection and tolerance. Mol Aspects Med Feb;33(1): Marrella V, Poliani PL, Fontana E, Casati A, Maina V, Cassani B, Ficara F, Cominelli M, Schena F, Paulis M, Traggiai E, Vezzoni P, Grassi F, Villa A. Anti-CD3{varepsilon} mab improves thymic architecture and prevents autoimmune manifestations in a mouse model of Omenn syndrome: therapeutic implications. Blood Aug 2;120(5): Lo Iacono N, Blair HC, Poliani PL, Marrella V, Ficara F, Cassani B, Facchetti F, Fontana E, Guerrini MM, Traggiai E, Schena F, Paulis M, Mantero S, Inforzato A, Valaperta S, Pangrazio A, Crisafulli L, Maina V, Kostenuik P, Vezzoni P, Villa A, Sobacchi C. Osteopetrosis rescue upon RANKL administration to Rankl(-/-) mice: A new therapy for human RANKL-dependent ARO. J Bone Miner Res Jul van Til NP, de Boer H, Mashamba N, Wabik A, Huston M, Visser TP, Fontana E, Poliani PL, Cassani B, Zhang F, Thrasher AJ, Villa A, Wagemaker G. Correction of Murine Rag2 Severe Combined Immunodeficiency by Lentiviral Gene Therapy Using a Codon-optimized RAG2 Therapeutic Transgene. Mol Ther Jun Villablanca EJ, De Calisto J, Torregrosa Paredes P, Cassani B, Nguyen DD, Gabrielsson S, Mora JR. β7 integrins are required to give rise to intestinal mononuclear phagocytes with tolerogenic potential. Gut Sep 12. Epub ahead of print 23. Maina V, Marrella V, Mantero S, Cassani B, Fontana E, Anselmo A, Del Prete A, Sozzani S, Vezzoni P, Poliani PL, Villa A. Hypomorphic mutation in the RAG2 gene affects dendritic cell distribution and migration. J Leukoc Biol Sep 19. Epub ahead of print 17 th October, 2013 Page 6/6 - Curriculum vitae of