Publications Biothérapies

Publications Scientifiques Originales 2006-2008

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. Gennery AR*, Slatter MA*, Grandin L, Taupin P, Cant AJ, Veys P, Amrolia PJ, Gaspar HB, Davies EG, Friedrich W, Hoenig M, Notarangelo LD, Mazzolari E, Porta F, Bredius RG, Lankester AC, Wulffraat NM, Seger R, Güngör T, Fasth A, Sedlacek P, Neven B, Blanche S, Fischer A, Cavazzana-Calvo M*, Landais P* (* These authors contributed equally to this work). Inborn Errors Working Party of the European Group for Blood and Marrow Transplantation ; European Society for Immunodeficiency. Transplantation of hematopoietic stem cells and long-term survival for primary immunodeficiencies in Europe : Entering a new century, do we do better ? J Allergy Clin Immunol 2010 ;126:602-10 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 ; 363 :355-64. Zhou S, Mody D, DeRavin SS, Hauer J, Lu T, Ma Z, Hacein-Bey-Abina S, Gray JT, Greene MR, Cavazzana-Calvo M, Malech HL, Sorrentino BP. A self-inactivating lentiviral vector for SCID-X1 gene therapy that does not activate LMO2 expression in human T cells. Blood 2010 ; 116:900-8. Bordon V, Gennery AR, Slatter MA, Vandecruys E, Laureys G, Veys P, Qasim W, Friedrich W, Wulffraat NM, Scherer F, Cant AJ, Fischer A, Cavazzana-Calvo M, Bredius RG, Notarangelo LD, Mazzolari E, Neven B, Güngör T. Clinical and immunological outcome of patients with cartilage hair hypoplasia after hematopoietic stem cell transplantation. Blood 2010 ; 116:27-35. Wang GP, Berry CC, Malani N, Leboulch P, Fischer A, Hacein-Bey-Abina S, Cavazzana-Calvo M, Bushman FD. Dynamics of gene-modified progenitor cells analyzed by tracking retroviral integration sites in a human SCID-X1 gene therapy trial. Blood 2010 ; 115:4356-66. Ginn SL, Liao SH, Dane AP, Hu M, Hyman J, Finnie JW, Zheng M, Cavazzana-Calvo M, Alexander SI, Thrasher AJ, Alexander IE. Lymphomagenesis in SCID-X1 mice following lentivirus-mediated phenotype correction independent of insertional mutagenesis and gamma c overexpression. Mol Ther 2010 ; 18:965-76. Fischer A, Hacein-Bey-Abina S, Cavazzana-Calvo M. 20 years of gene therapy for SCID. Commentary. Nature Immunol 2010 ; 6:1-4. 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. Pachlopnik Schmid J, Moshous D, Boddaert N, Neven B, Dal Cortivo L, Tardieu M, Cavazzana-Calvo M, Blanche S, de Saint-Basile G, Fischer A. Hematopoietic stem cell transplantation in Griscelli syndrome type 2 : a single-center report on 10 patients. Blood 2009 ; 114:211-8. Canaud G, Zuber J, Sberro R, Royale V, Anglicheau D, Snanoudj R, Gaha K, Thervet E, Lefrère F, Cavazzana-Calvo M, Noël LH, Méjean A, Legendre C, Martinez F. Intensive and prolonged treatment of focal and segmental glomerulosclerosis recurrence in adult kidney transplant recipients : a pilot study. Am J Transplant 2009 ; 9:1081-6. Ditadi A, De Coppi P, Picone O, Gautreau L, Smati R, Six E, Bonhomme D, Ezine S, Frydman R, Cavazzana-Calvo M*, André-Schmutz I* (* These authors contribued equally to this work). Human and murine amniotic fluid c-Kit+Lin- cells display hematopoietic activity. Blood 2009 ; 113:3953-60. Neven B, Leroy S, Decaluwe H, Le Deist F, Picard C, Moshous D, Mahlaoui N, Debré M, Casanova JL, Dal Cortivo L, Madec Y, Hacein-Bey-Abina S, de Saint Basile G, de Villartay JP, Blanche S, Cavazzana-Calvo M, Fischer A. Long-term outcome after haematopoietic stem cell transplantation of a single-centre cohort of 90 patients with severe combined immunodeficiency. Human and murine amniotic fluid c-Kit+Lin- cells display hematopoietic activity. Blood 2009 ; 113:4114-24. Boelens JJ, Rocha V, Aldenhoven M, Wynn R, O’Meara A, Michel G, Ionescu I, Parikh S, Prasad VK, Szabolcs P, Escolar M, Gluckman E, Cavazzana-Calvo M, Kurzberg J ; EUROCORD, Inborn error Working Party of EBMT and Duke University. Risk factor analysis of outcomes after unrelated cord blood transplantation in patients with Hurler syndrome. Biol Blood Marrow Transplant 2009 ; 15:618-25. Qasim W, Cavazzana-Calvo M, Davies EG, Davis J, Duval M, Eames G, Farinha N, Filopovich A, Fischer A, Friedrich W, Gennery A, Heilmann C, Landais P, Horwitz M, Porta F, Sedlacek P, Seger R, Slatte M, Teague L, Eapen M, Veys P. Allogeneic hematopoietic stem cell transplantation for Leukocyte Adhesion Deficiency. Pediatrics 2009 ; 123 : 836-40. Cavazzana-Calvo M. Basic research tries to decrease the risks of translational medicine – Gene Therapy Trials : lessons and remaining questions. Commentary. Gene Ther 2009 ; 16 : 309-10. 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. Pozzobon M, Piccoli M, Ditadi A, Bollini S, Destro R, André-Schmutz I, Masiero L, Lenzini E, Zanesco L, Petrelli L, Cavazzana-Calvo M, Gazzola MV, De Coppi P. Mesenchymal stromal cells can be derived from bone marrow CD133+ cells : implications for therapy. Stem Cells Dev 2009 ; 113:4114-24. André-Schmutz I, Six E, Bonhomme D, Rouiller J, Dal Cortivo L, Fischer A, Cavazzana-Calvo M. Shortening the immunodeficient period after hematopoietic stem cell transplantation. Immunol Res 2009 ; 44:54-60. Neven B, Cavazzana-Calvo M, Fischer A. Late immunologic and clinical outcomes for children with SCID. Biol Blood Marrow Transplant 2008 ; 14:17-8. Hyun I, Lindvall O, Hrlund-Richter LA, Cattaneo E, Cavazzana-Calvo M, Cossu G, de Luca M, Fox IJ, Gerstle C, Goldstein RA, Hermere G, High KA, Kim HO, Peng Lee H, Levy-Lahad E, Lo B, Marshak DR, McNab A, Munsie M, Nakauchi H, Rao M, Rooke HM, Valles CS, Srivastava A, Sugarman J, Taylor PL, Veiga A, Wong AL, Zoloth L, Daley GQ. New ISSCR guidelines underscore major principles for responsible translational stem cell research. Cell Stem Cell 2008 ; 3:607-9. Maes J, Maleszewska M, Guillemin C, Pflumio F, Six E, Andre-Schmutz I, Cavazzana-Calvo M, Charron D, Francastel C, Goodhoardt M. Lymphoid-affiliated genes are associated with active histone modifications in human hematopoietic stem cells. Blood 2008 ; 112 ; 2722-9. Hacein-Bey-Abina S, Garrigue A, Wang GP, Soulier J, Lim A, Morillon E, Clappier E, Caccavelli L, Delabesse E, Beldjord K, Asnafi V, MacIntyre E, dal Cortivo L, Radford I, Brousse N, Sigaux F, Moshous D, Hauer J, Borkhardt A, Belohradsky BH, Wintergerst U, Velez MC, Leiva L, Sorensen R, Wulffraat N, Blanche S, Bushman FD, Fischer A, Cavazzana-Calvo M. Insertional Oncogenesis in Four Patients after Retrovirus-Mediated Gene Therapy of SCID-X1. J Clin Invest 2008 ; 118:3132-42. Benjelloun F, Garrigue A, Demerens-de Chappedelaine C, Soulas-Sprauel P, Malassis-Séris M, Stockholm D, Hauer J, Blondeau J, Rivière J, Lim A, Le Lorc’h M, Romana S, Brousse N, Pâques F, Galy A, Charneau P, Fischer A, de Villartay JP, Cavazzana-Calvo M. Stable and functional lymphoid reconstitution in Artemis-deficient mice following lentiviral Artemis gene transfer into hematopoietic stem cells. Mol Ther 2008 ; 16:1490-9. Van Huyen JP, Smadja DM, Bruneval P, Gaussem P, Dal Cortivo L, Julia P, Fiessigner JN, Cavazzana-Calvo M, Aiach M, Emmerich J. Bone marrow-derived mononuclear cell therapy induces distal angiogenesis after local injection in critical leg ischemia. Mod Pathol 2008 ; 21:837-46. Dorival C, Brizzi F, Lelièvre J.D., Sol-Foulon N, Six E, Henry A, André-Schmutz I, Cavazzana-Calvo M, Coulombel L, Estaquier J, Schwartz O, Lévy Y. HIV-1 Nef protein expression in human CD34+ progenitors impairs the differentiation of an early T/NK cell precursor. Virology 2008 ; 377 : 207-15. Wang GP, Garrigue A, Ciuffi A, Ronen K, Leipzig J, Berry C, Lagresle-Peyrou C, Benjelloun F, Hacein-Bey-Abina S, Fischer A, Cavazzana-Calvo M, Bushman FD. DNA bar coding and pyrosequencing to analyze adverse events in therapeutic gene transfer. Nucleic Acids Res 2008 ; 36 : e49. Gannage M, Buzyn A, Bogiatzi SI, Lambert M, Soumelis V, Dal Cortivo L, Cavazzana-Calvo M, Brousse N, Caillat-Zucman S. Induction of NKG2D ligands by gamma radiation and tumor necrosis factor-a may participate in the tissue damage during acute graft-versus-host disease. Transplantation 2008 ; 85 : 911–5. Lagresle-Peyrou C, Benjelloun F, Hue C, Andre-Schmutz I, Bonhomme D, Forveille M, Beldjord K, Hacein-Bey-Abina S, de Villartay JP, Charneau P, Durandy A, Fischer A, Cavazzana-Calvo M. Restoration of Human B-cell Differentiation Into NOD-SCID Mice Engrafted With Gene-corrected CD34(+) Cells Isolated From Artemis or RAG1-deficient Patients. Mol Ther 2008 ; 16 : 396-403. Ozsahin H, Cavazzana-Calvo M, Notarangelo ND, Schulz A, Thrasher AJ, Mazzolari E, Slatter MA, Le Deist F, Blanche S, Veys P, Fasth A, Bredius R, Sedlacek P, Wulffraat N, Ortega J, Heilmann C, O’Meara A, Wachowiak J, Kalwak K, Matthes-Martin S, Gungor T, Ikinciogullari A, Landais P, Cant AJ, Friedrich W, Fischer A. Long-term outcome following hematopoietic stem cell transplantation in Wiskott-Aldrich syndrome : collaborative study of the European Society for Immunodeficiencies and European Group for Blood and Marrow Transplantation. Blood 2008 ; 111 : 439-45.