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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 (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 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 Related Article in Blood Online Social Bookmarking                   What's this?  Previous Article  |  Next Article  Submitted December 20, 2004 Accepted January 14, 2005 Failure of SCID-X1 gene therapy in older patients Adrian J Thrasher, Salima Hacein-Bey-Abina, H B Gaspar, Stephane Blanche, E G Davies, Kathryn Parsley, Kimberly Gilmour, Douglas King, Steven Howe, Joanna Sinclair, Christophe Hue, Frederique Carlier, Christof von Kalle, Genevieve de Saint Basile, Francoise le Deist, Alain Fischer, and Marina Cavazzana-Calvo* Molecular Immunology Unit, Institute of Child Health, London, United Kingdom; Immunology Unit, Great Ormond Street Hospital for Children, London, United Kingdom Department of Biotherapy, Hopital Necker, Paris, France; Cincinnati Children's Research Foundation, Cincinnati, OH, USA Unite d'Immunologie et d'Hematologie Pediatriques, Hopital Necker, Paris, France Immunology Unit, Great Ormond Street Hospital for Children, London, United Kingdom Molecular Immunology Unit, Institute of Child Health, London, United Kingdom Department of Biotherapy, Hopital Necker, Paris, France Cincinnati Children's Research Foundation, Cincinnati, OH, USA Inserm Unit 429, Hopital Necker, Paris, France Laboratoire d'Immunologie Pediatrique, Hopital Necker, Paris, France Unite d'Immunologie et d'Hematologie Pediatriques, Hopital Necker, Paris, France; Inserm Unit 429, Hopital Necker, Paris, France Department of Biotherapy, Hopital Necker, Paris, France; Inserm Unit 429, Hopital Necker, Paris, France * Corresponding author; email: m.cavazzana{at}nck.ap-hop-paris.fr. 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 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? 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