Marina Cavazzana-Calvo

Summary

Country: France

Publications

  1. ncbi request reprint Efficacy of gene therapy for SCID is being confirmed
    Marina Cavazzana-Calvo
    Département de Biothérapie, Hopital Necker Enfants Malades, 75015 Paris, France
    Lancet 364:2155-6. 2004
  2. ncbi request reprint Gene therapy for severe combined immunodeficiency
    Marina Cavazzana-Calvo
    Département de Biothérapie, Hopital Necker Enfants Malades, 75015 Paris, France
    Annu Rev Med 56:585-602. 2005
  3. ncbi request reprint Gene therapy of RAG-2-/- mice: sustained correction of the immunodeficiency
    Frank Yates
    Institut National de la Santé et de la Recherche Médicale INSERM U429, Hopital Necker Enfants Malades, Paris, France
    Blood 100:3942-9. 2002
  4. ncbi request reprint Gene transfer for activation of CMV specific T cells
    Isabelle Andre-Schmutz
    INSERM U429 I A S, M C C, Hopital Necker Enfants Malades, Paris, France
    Hum Immunol 65:565-70. 2004
  5. pmc Partial T and B lymphocyte immunodeficiency and predisposition to lymphoma in patients with hypomorphic mutations in Artemis
    Despina Moshous
    Unité Développement Normal et Pathologique du Système Immunitaire, Institut National de la Santé et de la Recherche Médicale INSERM U429, Hopital Necker Enfants Malades, Paris, France
    J Clin Invest 111:381-7. 2003
  6. pmc A human postnatal lymphoid progenitor capable of circulating and seeding the thymus
    Emmanuelle M Six
    Institut National de la Sante et de la Recherche Medicale INSERM, U768, 75015 Paris, France
    J Exp Med 204:3085-93. 2007
  7. ncbi request reprint [Human hematopoiesis: from CD34 cells to T lymphocytes]
    Marina Cavazzana-Calvo
    Département de Biothérapie, Hopital Necker Enfants Malades, Inserm U768, 149, rue de Sevres, 75015 Paris, France
    Med Sci (Paris) 23:151-9. 2007
  8. doi request reprint Immune reconstitution after haematopoietic stem cell transplantation: obstacles and anticipated progress
    Marina Cavazzana-Calvo
    Assistance Publique Hopitaux de Paris AP HP, Department of Biotherapy, Hopital Necker Enfants Malades, Paris, France
    Curr Opin Immunol 21:544-8. 2009
  9. doi request reprint Haematopoietic stem cell transplantation for SCID patients: where do we stand?
    Marina Cavazzana-Calvo
    Département de Biothérapie, AP HP, Hôpital Universitaire Necker Enfants Malades, Paris, France
    Br J Haematol 160:146-52. 2013
  10. ncbi request reprint Medical perspectives of adults and embryonic stem cells
    Marina Cavazzana-Calvo
    INSERM, Unite 429, Hopital Necker, 149, rue de Sevres, 75015 Paris, France
    C R Biol 325:1053-8. 2002

Detail Information

Publications73

  1. ncbi request reprint Efficacy of gene therapy for SCID is being confirmed
    Marina Cavazzana-Calvo
    Département de Biothérapie, Hopital Necker Enfants Malades, 75015 Paris, France
    Lancet 364:2155-6. 2004
  2. ncbi request reprint Gene therapy for severe combined immunodeficiency
    Marina Cavazzana-Calvo
    Département de Biothérapie, Hopital Necker Enfants Malades, 75015 Paris, France
    Annu Rev Med 56:585-602. 2005
    ..This review discusses recent developments in SCID identification and treatment...
  3. ncbi request reprint Gene therapy of RAG-2-/- mice: sustained correction of the immunodeficiency
    Frank Yates
    Institut National de la Santé et de la Recherche Médicale INSERM U429, Hopital Necker Enfants Malades, Paris, France
    Blood 100:3942-9. 2002
    ..Altogether, this study demonstrates the efficiency of ex vivo RAG-2 gene transfer in HSCs to correct the immune deficiency of RAG-2-/- mice, constituting a significant step toward clinical application...
  4. ncbi request reprint Gene transfer for activation of CMV specific T cells
    Isabelle Andre-Schmutz
    INSERM U429 I A S, M C C, Hopital Necker Enfants Malades, Paris, France
    Hum Immunol 65:565-70. 2004
    ..The protocols as well as the preclinical and clinical results obtained in the field of anti-CMV immunotherapy using gene transfer are reported and discussed...
  5. pmc Partial T and B lymphocyte immunodeficiency and predisposition to lymphoma in patients with hypomorphic mutations in Artemis
    Despina Moshous
    Unité Développement Normal et Pathologique du Système Immunitaire, Institut National de la Santé et de la Recherche Médicale INSERM U429, Hopital Necker Enfants Malades, Paris, France
    J Clin Invest 111:381-7. 2003
    ..This syndrome emphasizes the role of Artemis in the NHEJ pathway of DNA repair and suggests that other, yet ill-defined, conditions associating immunodeficiency and lymphoma could be caused by mutations in genes encoding NHEJ factors...
  6. pmc A human postnatal lymphoid progenitor capable of circulating and seeding the thymus
    Emmanuelle M Six
    Institut National de la Sante et de la Recherche Medicale INSERM, U768, 75015 Paris, France
    J Exp Med 204:3085-93. 2007
    ..Moreover, they belong to the most immature thymocyte population. Collectively, these findings unravel the existence of a postnatal lymphoid-polarized population that is capable of migrating from the BM to the thymus...
  7. ncbi request reprint [Human hematopoiesis: from CD34 cells to T lymphocytes]
    Marina Cavazzana-Calvo
    Département de Biothérapie, Hopital Necker Enfants Malades, Inserm U768, 149, rue de Sevres, 75015 Paris, France
    Med Sci (Paris) 23:151-9. 2007
    ....
  8. doi request reprint Immune reconstitution after haematopoietic stem cell transplantation: obstacles and anticipated progress
    Marina Cavazzana-Calvo
    Assistance Publique Hopitaux de Paris AP HP, Department of Biotherapy, Hopital Necker Enfants Malades, Paris, France
    Curr Opin Immunol 21:544-8. 2009
    ..If these objectives could be achieved, the haploidentical procedure would become more readily available to patients affected by acquired or inherited disorders of the haematopoietic system...
  9. doi request reprint Haematopoietic stem cell transplantation for SCID patients: where do we stand?
    Marina Cavazzana-Calvo
    Département de Biothérapie, AP HP, Hôpital Universitaire Necker Enfants Malades, Paris, France
    Br J Haematol 160:146-52. 2013
    ..New approaches for reconstituting T cell compartments more rapidly are under intense preclinical development and are discussed herein...
  10. ncbi request reprint Medical perspectives of adults and embryonic stem cells
    Marina Cavazzana-Calvo
    INSERM, Unite 429, Hopital Necker, 149, rue de Sevres, 75015 Paris, France
    C R Biol 325:1053-8. 2002
    ..We have tried here to summarise the advances in this field and to discuss the limits of these biological data...
  11. ncbi request reprint Severe combined immunodeficiency. A model disease for molecular immunology and therapy
    Alain Fischer
    INSERM U429, Hopital Necker Enfants Malades, Paris, France
    Immunol Rev 203:98-109. 2005
    ..SCID is thus a disease model for experimental therapy in the hematopoietic system...
  12. ncbi request reprint IL-7 effect on immunological reconstitution after HSCT depends on MHC incompatibility
    Isabelle Andre-Schmutz
    INSERM U 429, Hopital Necker Enfants Malades, Paris, France
    Br J Haematol 126:844-51. 2004
    ....
  13. ncbi request reprint Gene therapy of X-linked severe combined immunodeficiency
    Salima Hacein-Bey-Abina
    Gene and Cell Therapy Unit, Institut National de la Sante et de la Recherche Medicale, Hopital Necker Enfants Malades, Paris, France
    Methods Mol Biol 215:247-59. 2003
  14. ncbi request reprint Gene therapy of X-linked severe combined immunodeficiency
    Marina Cavazzana-Calvo
    INSERM U429, Hopital Necker Enfants Malades, Paris, France
    Curr Opin Allergy Clin Immunol 2:507-9. 2002
    ..Definitive conclusions cannot be thrown due to the limited number of gene therapy-treated patients and their relatively short follow-up...
  15. ncbi request reprint Sustained correction of X-linked severe combined immunodeficiency by ex vivo gene therapy
    Salima Hacein-Bey-Abina
    Laboratoire INSERM, Hopital Necker Enfants Malades, Paris, France
    N Engl J Med 346:1185-93. 2002
    ..We investigated whether infusion of autologous hematopoietic stem cells that had been transduced in vitro with the gamma(c) gene can restore the immune system in patients with severe combined immunodeficiency...
  16. doi request reprint Stable and functional lymphoid reconstitution in artemis-deficient mice following lentiviral artemis gene transfer into hematopoietic stem cells
    Fatine Benjelloun
    Institut National de la Santé et de la Recherche INSERM, U768, Universite Paris Descartes, Hopital Necker Enfants Malades, Paris, France
    Mol Ther 16:1490-9. 2008
    ..As a whole, our work provides a basis for supporting the gene therapy approach in Artemis-deficient SCID...
  17. doi request reprint Advances in adoptive immunotherapy to accelerate T-cellular immune reconstitution after HLA-incompatible hematopoietic stem cell transplantation
    Christian Reimann
    Institut National de la Sante et de la Recherche Medicale INSERM, Département de Biothérapie, Hopital Necker Enfants Malades, Assistance Publique Hopitaux de Paris, and Université Paris Descartes 75015 Paris, France
    Immunotherapy 2:481-96. 2010
    ..The present article will provide a brief summary of recent advances in the field of allodepletion and adoptive transfer of pathogen-specific T cells and a detailed discussion of strategies for enhancing thymopoiesis in vivo...
  18. doi request reprint Long-term outcome after hematopoietic stem cell transplantation of a single-center cohort of 90 patients with severe combined immunodeficiency
    Benedicte Neven
    Unité d Immuno Hématologie et Rhumatologie Pédiatrique, Hopital Necker Enfants Malades, Assistance Publique Hopitaux de Paris, Paris, France
    Blood 113:4114-24. 2009
    ..In most cases, HSCT enables long-term survival with infrequent sequelae. However, the occurrence of relatively late-onset complications is a concern that requires specific means of prevention and justifies careful patient follow-up...
  19. ncbi request reprint Immune reconstitution without graft-versus-host disease after haemopoietic stem-cell transplantation: a phase 1/2 study
    Isabelle Andre-Schmutz
    Laboratoire de Thérapie Cellulaire et Génique, INSERM U 429, Hopital Necker Enfants Malades, Paris, France
    Lancet 360:130-7. 2002
    ....
  20. ncbi request reprint Gene therapy of severe combined immunodeficiencies
    Alain Fischer
    INSERM U429, Hopital Necker, 149 rue de Sevres, 75015 Paris, France
    Nat Rev Immunol 2:615-21. 2002
    ..Nevertheless, severe combined immunodeficiencies are a useful model, because gene transfer can confer a selective advantage to transduced cells. In this way, a proof of concept for gene therapy has been provided...
  21. pmc Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1
    Salima Hacein-Bey-Abina
    Department of Biotherapy, Hopital Necker Enfants Malades, Assistance Publique Hopitaux de Paris AP HP, Universite Rene Descartes, Paris, France
    J Clin Invest 118:3132-42. 2008
    ..Successful chemotherapy was associated with restoration of polyclonal transduced T cell populations. As a result, the treated patients continued to benefit from therapeutic gene transfer...
  22. ncbi request reprint Characterization of antigen-specific repertoire diversity following in vitro restimulation by a recombinant adenovirus expressing human cytomegalovirus pp65
    Yamina Hamel
    INSERM U429, Hopital Necker Enfants Malades, 149 rue de Sevres, F 75743 Paris Cedex 15, France
    Eur J Immunol 33:760-8. 2003
    ..Altogether, these results suggest that the use of RAdpp65 to induce CMV- and adenovirus-specific CTL maybe appropriate for immunotherapy...
  23. ncbi request reprint Autoimmunity in Wiskott-Aldrich syndrome: risk factors, clinical features, and outcome in a single-center cohort of 55 patients
    Sophie Dupuis-Girod
    Unité d Immunologie et d Hématologie Pédiatriques, Hopital Necker Enfants Malades, Paris, France
    Pediatrics 111:e622-7. 2003
    ..To evaluate the occurrence of autoimmune and inflammatory complications in Wiskott-Aldrich syndrome (WAS) and to determine risk factors and the prognosis of such complications with the aim of improving the definition of treatment options...
  24. doi request reprint A specific time course for mobilization of peripheral blood CD34+ cells after plerixafor injection in very poor mobilizer patients: impact on the timing of the apheresis procedure
    Francois Lefrere
    Département de Biothérapie, Hopital Necker, Paris, France
    Transfusion 53:564-9. 2013
    ....
  25. ncbi request reprint Gene therapy for immunodeficiency diseases
    Alain Fischer
    INSERM U 429, Hopital Necker Enfants Malades, Paris, France
    Semin Hematol 41:272-8. 2004
    ..New advances in the technology of gene transfer should further promote gene therapy as a safe and effective therapeutic strategy of immunodeficiency diseases...
  26. ncbi request reprint Severe combined immunodeficiency and microcephaly in siblings with hypomorphic mutations in DNA ligase IV
    Dietke Buck
    INSERM, Hopital Necker Enfants Malades, U429, Unité Développement Normal et Pathologique du Système Immunitaire, Paris, France
    Eur J Immunol 36:224-35. 2006
    ..These observations contrast with the severity of the clinical immunodeficiency, suggesting that Lig4 may have additional critical roles in lymphocyte survival beyond V(D)J recombination...
  27. ncbi request reprint Evaluation of an algorithm based on peripheral blood hematopoietic progenitor cell and CD34+ cell concentrations to optimize peripheral blood progenitor cell collection by apheresis
    Francois Lefrere
    Département de Biothérapie, Service d Hématologie Adultes, and Laboratoire d Hématologie, Hopital Necker, 75743 Paris Cedex 15, France
    Transfusion 47:1851-7. 2007
    ..This prospective study has evaluated the potential of such method to predict the PBPC mobilization...
  28. pmc Is normal hematopoiesis maintained solely by long-term multipotent stem cells?
    Marina Cavazzana-Calvo
    Department of Biotherapy, Hopital Necker Enfants Malades, Assistance Publique Hopitaux de Paris AP HP, Paris, France
    Blood 117:4420-4. 2011
    ..The distribution of integration sites observed tends to support the validity of the revised model...
  29. ncbi request reprint Improving immune reconstitution while preventing graft-versus-host disease in allogeneic stem cell transplantation
    Marina Cavazzana-Calvo
    Laboratoire de Thérapie Cellulaire et Génique, INSERM U429, Hopital Necker Enfants Malades, Paris, France
    Semin Hematol 39:32-40. 2002
    ....
  30. ncbi request reprint [Gene therapy of children with X-linked severe combined immune deficiency: efficiency and complications]
    Alain Fischer
    INSERM U 429, Hopital Necker Enfants Malades, 149, rue de Sevres, 75015 Paris, France
    Med Sci (Paris) 20:115-7. 2004
  31. pmc Integration of retroviruses: a fine balance between efficiency and danger
    Alain Fischer
    Department and the Inserm Research Unit 429 at Necker University Hospital, Paris, France
    PLoS Med 2:e10. 2005
  32. doi request reprint Gene therapy of inherited diseases
    Alain Fischer
    INSERM Unit 768, Paris, France alain fi
    Lancet 371:2044-7. 2008
  33. ncbi request reprint Long-term survival and transplantation of haemopoietic stem cells for immunodeficiencies: report of the European experience 1968-99
    Corinne Antoine
    Service de Biostatistique et Service d Immunologie et d Hématologie Pédiatrique, Hopital Necker Enfants Malades, Paris, France
    Lancet 361:553-60. 2003
    ..This European report focuses on the long-term results of such procedures done between 1968 and December, 1999, for primary immunodeficiencies...
  34. ncbi request reprint [Cell therapy for inherited diseases of the hematopoietic system]
    Marina Cavazzana-Calvo
    INSERM, U768, Paris and université René Descartes Paris 5, Hopital Necker Enfants Malades, 149, rue de Sevres, 75743 Paris Cedex 15, France
    C R Biol 330:538-42. 2007
    ..In this review, we have summarized the difficulties and the scientific advances leading us to improve the clinical results; the therapeutic research's track for primary immunodeficiencies is also discussed...
  35. pmc Human adenylate kinase 2 deficiency causes a profound hematopoietic defect associated with sensorineural deafness
    Chantal Lagresle-Peyrou
    Research Laboratory on Normal and Pathologic Development of the Immune System, U768, Institut National de la Sante et de la Recherche Medicale, 75015 Paris, France
    Nat Genet 41:106-11. 2009
    ..These results identify a previously unknown mechanism involved in regulation of hematopoietic cell differentiation and in one of the most severe human immunodeficiency syndromes...
  36. ncbi request reprint Adenovirally transduced dendritic cells induce bispecific cytotoxic T lymphocyte responses against adenovirus and cytomegalovirus pp65 or against adenovirus and Epstein-Barr virus EBNA3C protein: a novel approach for immunotherapy
    Yamina Hamel
    INSERM U429, Hopital Necker Enfants Malades, 75743 Paris Cedex 15, France
    Hum Gene Ther 13:855-66. 2002
    ..Therefore, this approach could be considered in order to generate efficient virus cytolytic T cells to be used as adoptive immunotherapy in transplanted patients...
  37. ncbi request reprint Gene therapy for human severe combined immunodeficiencies
    Alain Fischer
    INSERM Unit 429, Gene Therapy Laboratory, Unit of Pediatric Immunology and Hematology, Necker Hospital, Paris, France
    Isr Med Assoc J 4:51-4. 2002
  38. pmc Vector integration is nonrandom and clustered and influences the fate of lymphopoiesis in SCID-X1 gene therapy
    Annette Deichmann
    Institute for Molecular Medicine and Cell Research and Department of Internal Medicine I, German Cancer Research Center, University of Freiburg, Freiburg, Germany
    J Clin Invest 117:2225-32. 2007
    ..Beyond the observed cases of insertional mutagenesis in 3 patients, these data help to elucidate the relationship between vector insertion and long-term in vivo selection of transduced cells in human patients with SCID-X1...
  39. ncbi request reprint LMO2 and gene therapy for severe combined immunodeficiency
    Alain Fischer
    N Engl J Med 350:2526-7; author reply 2526-7. 2004
  40. pmc Gene therapy for severe combined immunodeficiency: are we there yet?
    Marina Cavazzana-Calvo
    INSERM U768 and Université Paris Descartes, Paris, France
    J Clin Invest 117:1456-65. 2007
    ..In this review, we summarize the advantages and limitations associated with the use of gene therapy to cure SCID. Insertional mutagenesis and technological improvements aimed at increasing the safety of this strategy are also discussed...
  41. ncbi request reprint The future of gene therapy
    Marina Cavazzana-Calvo
    Immunology and Pediatric Haematology Unit, Hospital Necker, 75743 Paris, Cedex 15, France
    Nature 427:779-81. 2004
  42. ncbi request reprint [Curing a congenital immunologic deficiency]
    Nizar Mahlaoui
    Unite d Immuno Hematologie et Rhumatologie Pediatriques, Hopital Necker Enfants Malades, 75743 Paris 15
    Rev Prat 57:1699-700. 2007
  43. doi request reprint Restoration of human B-cell differentiation into NOD-SCID mice engrafted with gene-corrected CD34+ cells isolated from Artemis or RAG1-deficient patients
    Chantal Lagresle-Peyrou
    Institut National de la Sante et de Recherche Medicale, Unit 768, Necker site, Paris, France
    Mol Ther 16:396-403. 2008
    ..This overall approach represents a useful tool for evaluating gene transfer efficiency in human SCID forms affecting B-cell development (such as Artemis deficiency) and for testing new vectors for improving in vivo RAG1 complementation...
  44. pmc DNA bar coding and pyrosequencing to analyze adverse events in therapeutic gene transfer
    Gary P Wang
    University of Pennsylvania School of Medicine, Department of Microbiology, 3610 Hamilton Walk, Philadelphia, PA 19104 6076, USA
    Nucleic Acids Res 36:e49. 2008
    ..The methods described here should allow integration site populations from human gene therapy to be deeply characterized with spatial and temporal resolution...
  45. ncbi request reprint Transduced CD34+ cells from adrenoleukodystrophy patients with HIV-derived vector mediate long-term engraftment of NOD/SCID mice
    Sonia Benhamida
    INSERM U561, Hopital Saint Vincent de Paul, 75014 Paris, France
    Mol Ther 7:317-24. 2003
    ..Transplantation of transduced ALD CD34(+) cells into NOD/SCID mice resulted in long-term expression of ALD protein in monocytes/macrophages derived from engrafted stem cells...
  46. ncbi request reprint A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency
    Salima Hacein-Bey-Abina
    N Engl J Med 348:255-6. 2003
  47. ncbi request reprint Gene therapy of X-linked severe combined immunodeficiency
    Salima Hacein-Bey-Abina
    INSERM U 429, Gene and Cell Therapy Unit, Hĵpital Necker Enfants Malades, Paris, France
    Int J Hematol 76:295-8. 2002
    ..These 8 patients are doing well and living in a normal environment. This yet successful gene therapy demonstrates that in a setting where transgene expression provides a selective advantage, a clinical benefit can be expected...
  48. pmc A hypomorphic R229Q Rag2 mouse mutant recapitulates human Omenn syndrome
    Veronica Marrella
    Human Genome Department, Istituto di Tecnologie Biomediche, CNR, Via Fratelli Cervi 93, Segrate, Milan 20090, Italy
    J Clin Invest 117:1260-9. 2007
    ..In conclusion, Rag2(R229Q/R229Q) mice mimicked most symptoms of human OS; our findings support the notion that impaired immune tolerance and defective immune regulation are involved in the pathophysiology of OS...
  49. ncbi request reprint Long-term T-cell reconstitution after hematopoietic stem-cell transplantation in primary T-cell-immunodeficient patients is associated with myeloid chimerism and possibly the primary disease phenotype
    Marina Cavazzana-Calvo
    Institut National de la Sante et de la Recherche Medicale, Unité 768, Paris, France
    Blood 109:4575-81. 2007
    ....
  50. ncbi request reprint Severe cutaneous papillomavirus disease after haemopoietic stem-cell transplantation in patients with severe combined immune deficiency caused by common gammac cytokine receptor subunit or JAK-3 deficiency
    Caroline Laffort
    Unite d immunologie, d Hématologie, et de Rhumatologie Pédiatrique, Paris, France
    Lancet 363:2051-4. 2004
    ..That genetic causes are the only predisposing factor to be identified for severe combined immune deficiency, suggests that natural-killer cells or gammac/JAK-3-dependent signalling in keratinocytes could have a role in anti-HPV immunity...
  51. ncbi request reprint Competition within the early B-cell compartment conditions B-cell reconstitution after hematopoietic stem cell transplantation in nonirradiated recipients
    Allen Liu
    Developpement Normal et Pathologique du Systeme Immunitaire, Institut National de la Santé et de la Recherche Médicale U768 Site Necker Enfants Malades, Paris Cedex 15, France
    Blood 108:1123-8. 2006
    ....
  52. ncbi request reprint Gene therapy of metabolic diseases
    Alain Fischer
    INSERM, U768, Paris, France
    J Inherit Metab Dis 29:409-12. 2006
    ..A significant safety issue has also been observed. Several strategies are being experimentally tested for a number of metabolic diseases (more than 20) and may provide a rationale for the future development of clinical trials...
  53. ncbi request reprint Long-term immune reconstitution in RAG-1-deficient mice treated by retroviral gene therapy: a balance between efficiency and toxicity
    Chantal Lagresle-Peyrou
    Universite Paris Descartes, Faculte de Medecine, INSERM Unit 429, Site Necker Enfants Malades, 149 rue de Sevres, 75743 Paris Cedex15, France
    Blood 107:63-72. 2006
    ....
  54. ncbi request reprint Treatment of an infant with X-linked severe combined immunodeficiency (SCID-X1) by gene therapy in Australia
    Samantha L Ginn
    Gene Therapy Research Unit, The Children s Hospital at Westmead and Children s Medical Research Unit, Sydney, NSW
    Med J Aust 182:458-63. 2005
    ..To report the outcome of gene therapy in an infant with X-linked severe combined immunodeficiency (SCID-X1), which typically causes a lack of T and natural killer (NK) cells...
  55. ncbi request reprint Failure of SCID-X1 gene therapy in older patients
    Adrian J Thrasher
    Molecular Immunology Unit, Institute of Child Health, London, United Kingdom
    Blood 105:4255-7. 2005
    ..In particular, there is likely to be a limitation to initiation of normal thymopoiesis, and we therefore suggest that intervention for these patients should be considered as early as possible...
  56. ncbi request reprint Bone marrow transplantation attenuates the myopathic phenotype of a muscular mouse model of spinal muscular atrophy
    Nouzha Salah-Mohellibi
    Molecular Neurogenetics Laboratory, Institut National de la Sante et de la Recherche Medicale, INSERM, U798, Evry, F 91057 France
    Stem Cells 24:2723-32. 2006
    ..Taken together, our data suggest that a biological activity is likely involved in muscle regeneration improvement mediated by BM transplantation. HGF may represent an attractive paracrine mechanism to support this activity...
  57. ncbi request reprint Gene therapy: X-SCID transgene leukaemogenicity
    Adrian J Thrasher
    Molecular Immunology Unit, Institute of Child Health, University College London, London WC1N 1EH, UK
    Nature 443:E5-6; discussion E6-7. 2006
    ..Here we show that transgenic IL2RG does not necessarily have potent intrinsic oncogenic properties, and argue that the interpretation of this observation with respect to human trials is overstated...
  58. ncbi request reprint CD34 stem cell top-ups without conditioning after initial haematopoietic stem cell transplantation for correction of incomplete haematopoietic and immunological recovery in severe congenital immunodeficiencies
    Claire Booth
    Department of Clinical Immunology, Great Ormond Street Hospital NHS Trust, London, UK
    Br J Haematol 135:533-7. 2006
    ..Unconditioned stem cell boosts have limited toxicity but should be given early after the original graft to be effective...
  59. ncbi request reprint Clonal evidence for the transduction of CD34+ cells with lymphomyeloid differentiation potential and self-renewal capacity in the SCID-X1 gene therapy trial
    Manfred Schmidt
    Department of Internal Medicine, University of Freiburg, Germany
    Blood 105:2699-706. 2005
    ..These results provide a first evidence in the setting of a clinical trial that CD34+ cells maintain both lymphomyeloid potential as well as self-renewal capacity after ex vivo manipulation...
  60. ncbi request reprint Management options for adenosine deaminase deficiency; proceedings of the EBMT satellite workshop (Hamburg, March 2006)
    Claire Booth
    Molecular Immunology Unit, Institute of Child Health, University College London, and Department of Clinical Immunology, Great Ormond Street Hospital NHS Trust, London WC1N 3JH, UK
    Clin Immunol 123:139-47. 2007
    ..Long-term follow-up of treated patients highlights a significant incidence of non-immunological problems with cognitive, neurological and audiological abnormalities most prominent...
  61. pmc Severe combined immunodeficiency caused by deficiency in either the delta or the epsilon subunit of CD3
    Genevieve De Saint Basile
    Unité Développement Normal et Pathologique du Système Immunitaire, INSERM U 429, Paris, France
    J Clin Invest 114:1512-7. 2004
    ..e., CD3epsilon deficiency, and emphasize the essential roles played by the CD3epsilon and CD3delta subunits in human thymocyte development, since these subunits associate with both the pre-TCR and the TCR...
  62. ncbi request reprint Treatment of CD40 ligand deficiency by hematopoietic stem cell transplantation: a survey of the European experience, 1993-2002
    Andrew R Gennery
    Newcastle General Hospital, Westgate Rd, Newcastle upon Tyne, NE4 6BE United Kingdom
    Blood 103:1152-7. 2004
    ..Preexisting lung damage was the most important adverse risk factor. Further studies will determine optimal timing and type of HSCT...
  63. doi request reprint Lymphoid-affiliated genes are associated with active histone modifications in human hematopoietic stem cells
    Jérôme Maës
    Institut Universitaire d Hematologie, Universite Paris 7 Denis Diderot, Paris, France
    Blood 112:2722-9. 2008
    ..This permissive chromatin structure is progressively lost as the stem cell differentiates...
  64. ncbi request reprint Dynamics of thymus-colonizing cells during human development
    Rima Haddad
    Laboratoire d Immunologie Cellulaire et Immunopathologie de l Ecole Pratique des Hautes Etudes and UMR 7151, Centre National de la Recherche Scientifique, Universite Paris 7, Paris, France
    Immunity 24:217-30. 2006
    ....
  65. ncbi request reprint Hematopoietic stem cell transplantation in hemophagocytic lymphohistiocytosis: a single-center report of 48 patients
    Marie Ouachée-Chardin
    Department of Pediatric Immuno Hematology, Necker Enfants Malades Hospital, Paris, France
    Pediatrics 117:e743-50. 2006
    ..Chemotherapy- or immunotherapy-based treatments can achieve remission. Hematopoietic stem cell transplantation (HSCT), however, is the only curative option, but optimal modalities and long-term outcome are not yet well known...
  66. doi request reprint HIV-1 Nef protein expression in human CD34+ progenitors impairs the differentiation of an early T/NK cell precursor
    Céline Dorival
    INSERM U 841, Faculte de Medecine, Creteil, France
    Virology 377:207-15. 2008
    ..Altogether, these data demonstrate that Nef interferes with the differentiation of a primitive lymphoid human precursor with a T/NK potential...
  67. doi request reprint Bone marrow-derived mononuclear cell therapy induces distal angiogenesis after local injection in critical leg ischemia
    Jean Paul Duong Van Huyen
    University Paris Descartes, Paris, France
    Mod Pathol 21:837-46. 2008
    ....
  68. ncbi request reprint A double-blind low dose-finding phase II study of granulocyte colony-stimulating factor combined with chemotherapy for stem cell mobilization in patients with non-Hodgkin's lymphoma
    Francois Lefrere
    Service d Hématologie Adultes, Centre d Investigation Clinique Hôpital Necker, Direction de la Recherche Clinique, AP HP, Paris, France
    Haematologica 91:550-3. 2006
    ..7%, 91%, 93.9 and 96.4%, respectively. Low G-CSF doses may be used with a similar probability of success as conventional doses and could allow significant savings...
  69. ncbi request reprint Allogeneic bone marrow transplantation in mevalonic aciduria
    Benedicte Neven
    Unité d Immuno Hématologie et Rhumatologie Pédiatrique, Assistance Publique Hopitaux de Paris, Paris, France
    N Engl J Med 356:2700-3. 2007
    ..We observed sustained remission of febrile attacks and inflammation during a 15-month follow-up period...
  70. ncbi request reprint 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
    Hulya Ozsahin
    Department of Pediatrics, Geneva University Hospital, Geneva, Switzerland
    Blood 111:439-45. 2008
    ....
  71. ncbi request reprint Human leucocyte antigen-identical haematopoietic stem cell transplantation in major histocompatiblity complex class II immunodeficiency: reduced survival correlates with an increased incidence of acute graft-versus-host disease and pre-existing viral infe
    Raffaele Renella
    Unité d Immunologie et Hématologie Pédiatrique, Faculté de Médecine Université Réné Descartes, Paris, France
    Br J Haematol 134:510-6. 2006
    ..We suggest that the reduced survival after HLA-identical HSCT may be caused by the high incidence of pre-existing viral infections and associated with the onset of severe acute GVHD...
  72. ncbi request reprint Successful peripheral blood stem cell harvesting with granulocyte colony-stimulating factor alone after previous mobilization failure
    Francois Lefrere
    Haematologica 89:1532-4. 2004
    ..Stem cell mobilization was successful in 90 patients, who yielded a median of 3.5x10(6) CD34(+) cells/kg, partially successful in 17 patients (1-2.4x10(6) CD34+ cells/kg) and failed in the remaining 31 patients...
  73. ncbi request reprint Failure of bone marrow transplantation to eradicate HIV reservoir despite efficient HAART
    Véronique Avettand-Fenoel
    AIDS 21:776-7. 2007