Short CV/Education and training

  • 1983
    MD

  • 1983 – 1987
    Residency in Paediatric, University of Padua, Italy

  • 1984
    Master in Human Biology / Haematology, Paris VII University

  • 1985 – 1987
    Clinical Oncology Certification, Paris VII University

  • 1987
    Master in Human Biology / Experimental Oncology, Paris XI University

  • 1988
    Immunobiotechnologies, Immunogenetics & Blood Transfusion DEA

  • 1989 – 1993
    PhD, Paris VII University

  • Since 1994
    Chief of Research team No.6 (New therapeutic approach to primary immune deficiencies), INSERM Unit 768

  • 1997
    Ability to Lead a Research – Paris Descartes University

  • 2000
    Professor of Haematology – Full Practicioner, Paris Descartes University

  • 2001
    Qualification in Transfusion Technologies, Franche-Comté University

  • Since 2003
    Head of the Department of Biotherapy, Hopital Necker Enfants-Malades (Paris)

  • Since 2006
    Coordinator of the Biotherapy Clinical Investigation Center (CIC), GHU Necker-Cochin-HEGP (APHP / INSERM)

Selected publications

  • Cavazzana-Calvo M*, Payen E*, Negre O, Wang G, Hehir K, Fusil F, Down J, Denaro M, Brady T, Westerman KA, Cavallesco R, Gillet-Legrand B, Caccavelli L, Sgarra R, Maouche-Chrétien L, Bernaudin F, Girot R, Dorazio R, Mulder GJ, Polak A, Bank A, Soulier J, Larghero J, Kabbara N, Dalle B, Gourmel B, Socie G, Chretien S, Cartier N, Aubourg P, Fischer A, Cornetta K, Galacteros F, Beuzard Y, Gluckman E, Bushman F, Hacein-Bey-Abina S*, Leboulch P* (* These authors contributed equally to this work). Transfusion independence and HMGA2 activation after gene therapy of human -thalassaemia. Nature 2010;467:318-22.

  • Hacein-Bey-Abina S, Hauer J, Lim A, Picard C, Wang GP, Berry CC, Martinache C, Rieux-Laucat F, Latour S, Belohradsky BH, Leiva L, Sorensen R, Debré M, Casanova JL, Blanche S, Durandy A, Bushman FD, Fischer A, Cavazzana-Calvo M. Efficacy of Gene Therapy for X-linked Severe Combined Immunodeficiency. N Engl J Med 2010 ; 2010 ; 363 :355-64.

  • Cartier N*, Hacein-Bey-Abina S*, Bartholomae CC, Veres G, Schmidt M, Kutschera I, Vidaud M, Abel U, Dal-Cortivo L, Caccavelli L, Mahlaoui N, Kiermer V, Mittelstaedt D, Bellesme C, Lahlou N, Lefrère F, Blanche S, Audit M, Payen E, Leboulch P, l'Homme B, Bougnères P, Von Kalle C, Fischer A, Cavazzana-Calvo M*, Aubourg P* (*These authors contributed equally to this work). Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy. Science 2009 ; 326:818-23.

  • Lagresle-Peyrou C, Six EM, Picard C, Rieux-Laucat F, Michel V, Ditadi A, Demerens-de Chappedelaine C, Morillon E, Valensi F, Simon-Stoos KL, Mullikin JC, Noroski LM, Besse C, Wulffraat NM, Ferster A, Abecasis MM, Calvo F, Petit C, Candotti F, Abel L, Fischer A, Cavazzana-Calvo M. Human adenylate kinase 2 deficiency causes a profound haematopoietic defect. Nat Genet 2009 ; 41:106-11.

  • Deichmann A, Hacein-Bey-Abina S, Schmidt M, Garrigue A, Brugman M, Hu J, Glimm H, Gyapay G, Prum B, Fraser CC, Fischer N, Schwarzwaelder K, Siegler ML, de Ridder D, Pike-Overzet K, Howe SJ, Thrasher AJ, Wagemaker G, Abel U, Staal F, Delabesse E, Villeval JL, Aronow B, Hue C, Prinz C, Wissler M, Klanke C, Weissenbach J, Alexander I, Fischer A, von Kalle C, Cavazzana-Calvo M. Vector integration is non-random, clustered and influences the in vivo fate of lymphopoiesis in SCID-X1 gene therapy. J Clin Invest 2007 ; 117 : 2225-32.

  • Cavazzana-Calvo M, Carlier F, le Deist F, Morillon E, Taupin P, Gautier D, Radford-Weiss I, Caillat-Zucman S, Neven B, Blanche S, Cheynier R, Fischer A, Hacein-Bey-Abina S. Long-term T cell reconstitution after haematopoietic stem cell transplantation in primary T cell immunodeficient patients is associated with myeloid chimerism and possibly the primary disease phenotype. Blood 2007 ; 109 : 4575-81.

  • Thrasher AJ, Gaspar HB, Baum C, Modlich U, Schambach A, Candotti F, Otsu M, Sorrentino B, Scobie L, Cameron E, Blyth K, Neil J, Hacein-Bey-Abina S, Cavazzana-Calvo M, Fischer A. Gene therapy: X-SCID transgene leukaemogenicity. Nature. 2006 ; 443 : E5-6.

  • Hacein-Bey-Abina S, von Kalle C, Schmidt M, McCormack MP, Wulffraat N, Leboulch P, Lim A, Osborne CS, Pawliuk R, Morillon E, Sorensen R, Forster A, Fraser P, Cohen JI, de Saint-Basile G, Alexander I, Wintergerst U, Frebourg T, Aurias A, Stoppa-Lyonnet D, Romana S, Radford-Weiss I, Gross F, Valensi F, Delabesse E, Macintyre E, Sigaux F, Soulier J, Leiva LE, Wissler M, Prinz C, Rabbitts TH, le Deist F, Fischer A, Cavazzana-Calvo M. LMO2-associated T-cell proliferation in two patients after gene therapy for SCID-X1 . Science 2003 ; 302 : 415-9.

  • André-Schmutz I, le Deist F, Hacein-Bey S, Vitetta E, Schindler J, Ghetie V, Pellier I, Quartier P, Chedeville G, Luby JM, Martinache C, Hamel Y, Vilmer E, Casanova JL, Audat F, Caillat-Zucman S, Radford I, Buffet R, Fischer A, Cavazzana-Calvo M. Preventing GVHD while improving immune reconstitution in allogeneic stem cell transplantation by infusion of donor T lymphocytes after ex vivo depletion of CD25+ alloreactive cells with an immunotoxin. Lancet 2002 ; 360: 130-7.

  • Cavazzana-Calvo M, Hacein-Bey S, de Saint-Basile G, Gross F, Yvon E, Nussbaum P, Selz F, Hué C, Certain S, Casanova JL, Bousso P, le Deist F, Fischer A. Gene Therapy of human severe combined immunodeficiency (SCID)-X1 disease, Science 2000 ; 288 : 669-72.


Selected projects

  • National Agency of AIDS Research (ANRS) – Title : Development of a gene therapy protocol for HIV patients affected with hematopoietic malignancies – Acronym “HIV against HIV” (Principal investigator : M. Cavazzana-Calvo), 2012 – 2014

  • FP7-Health – ERC-2010 Call for Projects (Project #269037 « RegenerativeTherapy») – Title : Cell and gene therapy approaches for inherited diseases with unsatisfying or no therapeutic option (Principal Investigator : M. Cavazzana-Calvo), 2011 – 2015

  • FP7-HEALTH-2010-single-stage European Call for Projects (Project #261392 «GENEGRAFT ») – Title : Phase I/II ex vivo gene therapy trial for recessive dystrophic epidermolysis bullosa using skin equivalent grafts genetically corrected with a COL7A1-encoding SIN retroviral vector (Coordinator : A. Hovnanian ; Co-investigators : M. Cavazzana-Calvo, J. McGrath, K. Kühlcke, D. Caizergues, J. Dart), 2011 – 2015

  • FP7-HEALTH-2010-single-stage European Call for Projects (Project #261387 « CELL-PID ») – Title : Advanced cell-based therapies for the treatment of primary immunodeficiency (Coordinator : A. Aiuti ; Scientific Steering Committee : M. Cavazzana-Calvo, A. Thrasher, G. Wagemaker, C. Baum), 2010 – 2014

  • National 2010 PHRC1 (Project #AOM10013) – Title : Injection of anti-cytomegalovirus and anti-adenovirus CD4+CD8+ T lymphocytes for the treatment of viral infections occurring after allogeneic haematopoietic stem cell transplantation (Principal Investigator : M. Cavazzana-Calvo), 2010 – 2013

  • National 2009 PHRC1 – Title : Hepatocytes allogeneic transplantation after reversible partial portal embolization to treat congenital defects of the urea cyle (Principal investigator : I. Dagher, Hôpital Antoine Beclère, Clamart, France), 2009 – 2011

  • Genethon – Clinical Research Trial Collaboration – Title : Phase I/II clinical trial of haematopoietic stem cell gene therapy for the Wiskott-Aldrich Syndrome (Principal Investigators: A. Fischer, M. Cavazzana-Calvo, Scientific Coordinator: S. Hacein-Bey-Abina), 2009 – 2012

  • National 2008 PHRC – Title : Protocol n.2 of gene therapy for X-linked severe combined immunodeficiency (SCID-X1) using a secured retroviral vector (project AOM 08 064) (Principal investigators : M. Cavazzana-Calvo, A. Fischer), 2008 – 2011

  • Ministry of Health Hospital Direction (DHOS) / National Institute for Health and Medical Research (INSERM) / National Institute for Cancer (INCA) : Call for Projects Funding 2008 – Translational Clinical Research – Title : Development of an ex vivo expansion method for T lymphoid progenitors by short exposition to the Noth ligand to try to accelerate the immunological reconstitution after allogenic hematopoietic stem cell transplantation using a partially HLA compatible donor (Principal Investigators : M. Cavazzana-Calvo, I. André-Schmutz), 2008 – 2010

Membership in scientific bodies/juries

Memberships in Scientific Societies

  • Pediatric Hematology and Immunology Society, since 1993

  • Hematopoietic and Oncogenesis Club, since 1994

  • The International Society for Hemotherapy and Graft Engineering, since 1995

  • French Society of Haematology, since 1996

  • French Society of Gene and Cell Therapy (member of the scientific committee), since 1996

  • American Society of Hematology, since 1996

  • American Society of Gene Therapy, since 1996

  • European Society for Gene & Cell Therapy, since 1999

National appointments and collective responsibilities

  • Member of the Academic judging committee to the nomination of the Biotherapy Chair, University Paris Decartes, 2010

  • Vice-President of the Scientific Board of the Foundation for Medical Research (FRM), 2010

  • Member of the Clinical Research Commission, University Paris Descartes, 2010

  • Member of EUROCORD Association, since 2010

  • Member of the Scientific Advisory Board of Genethon, since 2008

  • Chairwoman of the Scientific Committee for Strategies and Evaluation of Clinical Research Protocols (INSERM), 2006 – 2011

  • Member of AFM Commission for clinical trials & of AFM’s Scientific Committee, since 2002

  • Responsible for the Stem Cell Sub-committee, since 2001

  • Member of the French Cancer Research Association National Commission No.1, 2000 – 2005

  • Vice chairwoman of INSERM intercommission No.5, 2000 – 2002

  • Member of the consortium of researchers group No. 21 ("Constituted lymphocyte hemostasis") for National Agency of AIDS Research (ANRS), 1998 – 2000

  • Expert with the French Agency for the Safety of Health Products, since 1997

  • Member of the Scientific Board of the French Society of Bone Marrow Graft, 1996 – 2000

  • Biotherapy Consortium of Researchers Member, INSERM

International appointments and collective responsibilities

  • Member of the Board of Directors of the American Society for Gene and Cell Therapy (ASGCT), 2011 – 2014

  • Member of the Editorial Board of Stem Cells Translational Medicine, since 2011

  • Invited Member of the Andalusian Initiative for Advanced Therapies (AIATA) Education Advisory Board, since Oct. 2011

  • Blood Editorial Board Invited Member, 2008 – 2012

  • Member of the International Society for Stem Cell Research Ethics Committee. since 2008

  • Member of the Scientific Board of the European CONSERT Advanced Course Symposium and Practical Training on “Lentiviral vectors: concepts, practice, hope and reality” ; Chairwoman of the 2008 Course, since 2008

  • Member of the American Society of Gene Therapy Hematopoietic Committee & of the Ethics Committee, 2006 – 2008

  • Member of the Transatlantic Gene Therapy Consortium, since 2005

  • Inborn Errors Working Party Chairwoman & European Bone Marrow Transplantation (EBMT) Group Scientific Council Member, 2001 – 2008

Media coverage

Additional qualifications

  • Extensive academic teaching


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