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Blood, 1 June 2005, Vol. 105, No. 11, pp. 4255-4257. Prepublished online as a Blood First Edition Paper on February 1, 2005; DOI 10.1182/blood-2004-12-4837. This Article Full Text Full Text (PDF) All Versions of this Article: 2004-12-4837v1 105/11/4255    most recent Alert me when this article is cited Alert me if a correction is posted Citation Map Services Email this article to a friend Similar articles in this journal Similar articles in PubMed Alert me to new issues of the journal Download to citation manager Rights and Permissions Citing Articles Citing Articles via HighWire Citing Articles via CrossRef Citing Articles via Google Scholar Google Scholar Articles by Thrasher, A. J. Articles by Cavazzana-Calvo, M. Search for Related Content PubMed PubMed Citation Articles by Thrasher, A. J. Articles by Cavazzana-Calvo, M. Related Collections Immunobiology Clinical Trials and Observations Gene Therapy Related Article in Blood Online Social Bookmarking                   What's this?  Previous Article  |  Table of Contents  |  Next Article  GENE THERAPY Brief report Failure of SCID-X1 gene therapy in older patients Adrian J. Thrasher, Salima Hacein-Bey-Abina, H. Bobby Gaspar, Stephane Blanche, E. Graham Davies, Kathryn Parsley, Kimberly Gilmour, Douglas King, Steven Howe, Joanna Sinclair, Christophe Hue, Frédérique Carlier, Christof von Kalle, Geneviève de Saint Basile, Françoise le Deist, Alain Fischer, and Marina Cavazzana-Calvo From the Molecular Immunology Unit, Institute of Child Health, London, United Kingdom; Department of Biotherapy, Unité d'Immunologie et d'Hématologie Pédiatriques, Institut National de la Santé et de la Recherche Médicale (INSERM) Unit 429, and Laboratoire d'Immunologie Pédiatrique, Hôpital Necker, Paris, France; Immunology Unit, Great Ormond Street Hospital for Children, London, United Kingdom; and Cincinnati Children's Research Foundation, Cincinnati, OH. Gene therapy has been shown to be a highly effective treatment for infants with typical X-linked severe combined immunodeficiency (SCID-X1, c-deficiency). For patients in whom previous allogeneic transplantation has failed, and others with attenuated disease who may present later in life, the optimal treatment strategy in the absence of human leukocyte antigen (HLA)–matched donors is unclear. Here we report the failure of gene therapy in 2 such patients, despite effective gene transfer to bone marrow CD34+ cells, suggesting that there are intrinsic host-dependent restrictions to efficacy. 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. CiteULike    Connotea    Del.icio.us    Digg    Reddit    Technorati    What's this? Related Article in Blood Online: Old before its time: age-related thymic dysfunction may preclude efficacy of gene therapy in older SCID-X1 patients Ruth Seggewiss and Cynthia E. Dunbar Blood 2005 105: 4160-4161. [Full Text] [PDF] This article has been cited by other articles: A. W. Nienhuis Development of gene therapy for blood disorders Blood, May 1, 2008; 111(9): 4431 - 4444. [Abstract] [Full Text] [PDF] W. Qasim, H Bobby Gaspar, and A. J Thrasher Update on clinical gene therapy in childhood Arch. Dis. Child., November 1, 2007; 92(11): 1028 - 1031. [Abstract] [Full Text] [PDF] D. B. Kohn Gene therapy as salvage Blood, July 1, 2007; 110(1): 4 - 4. [Full Text] [PDF] J. Chinen, J. Davis, S. S. De Ravin, B. N. Hay, A. P. Hsu, G. F. Linton, N. Naumann, E. Y. H. Nomicos, C. Silvin, J. Ulrick, et al. Gene therapy improves immune function in preadolescents with X-linked severe combined immunodeficiency Blood, July 1, 2007; 110(1): 67 - 73. [Abstract] [Full Text] [PDF] J. Flygare and S. Karlsson Diamond-Blackfan anemia: erythropoiesis lost in translation Blood, April 15, 2007; 109(8): 3152 - 3154. [Abstract] [Full Text] [PDF] P. J. Amrolia, G. Muccioli-Casadei, H. Huls, S. Adams, A. Durett, A. Gee, E. Yvon, H. Weiss, M. Cobbold, H. B. Gaspar, et al. Adoptive immunotherapy with allodepleted donor T-cells improves immune reconstitution after haploidentical stem cell transplantation Blood, September 15, 2006; 108(6): 1797 - 1808. [Abstract] [Full Text] [PDF] J. A. Borghans, R. G. Bredius, M. D. Hazenberg, H. Roelofs, E. C. Jol-van der Zijde, J. Heidt, S. A. Otto, T. W. Kuijpers, W. E. Fibbe, J. M. Vossen, et al. Early determinants of long-term T-cell reconstitution after hematopoietic stem cell transplantation for severe combined immunodeficiency Blood, July 15, 2006; 108(2): 763 - 769. [Abstract] [Full Text] [PDF] T. Neff, B. C. Beard, and H.-P. Kiem Survival of the fittest: in vivo selection and stem cell gene therapy Blood, March 1, 2006; 107(5): 1751 - 1760. [Abstract] [Full Text] [PDF] O. Adjali, R. R. Vicente, C. Ferrand, C. Jacquet, C. Mongellaz, P. Tiberghien, K. Chebli, V. S. Zimmermann, and N. Taylor Intrathymic administration of hematopoietic progenitor cells enhances T cell reconstitution in ZAP-70 severe combined immunodeficiency PNAS, September 20, 2005; 102(38): 13586 - 13591. [Abstract] [Full Text] [PDF]

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