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Blood, 1 January 2007, Vol. 109, No. 1, pp. 383-385. Prepublished online as a Blood First Edition Paper on September 21, 2006; DOI 10.1182/blood-2006-05-025072. This Article Full Text (PDF) All Versions of this Article: blood-2006-05-025072v1 109/1/383    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 Rao, A. Articles by Shenoy, S. Search for Related Content PubMed PubMed Citation Articles by Rao, A. Articles by Shenoy, S. Social Bookmarking                   What's this?  Previous Article  |  Next Article  Submitted May 23, 2006 Accepted August 7, 2006 Successful bone marrow transplantation for IPEX syndrome following reduced intensity conditioning Aarati Rao, Naynesh Kamani, Alexandra Filipovich, Susan Molleran Lee, Stella Davies, Jignesh Dalal, and Shalini Shenoy* Washington University School of Medicine and St. Louis Children's Hospital, St. Louis, MO Children's National Medical Center, Washington D.C. Children's Hospital Medical Center, Cincinnati, OH Children's Mercy Hospital, Kansas City, MO * Corresponding author; email: shenoy{at}wustl.edu. IPEX syndrome (immune dysregulation, polyendocrinopathy, enteropathy, X-linked disease) is a rare, fatal autoimmune disorder caused by mutations in the FOXP3 gene leading to disruption of signaling pathways involved in regulatory T lymphocyte function. Life-long multi-agent immunosuppression is necessary to control debilitating autoimmune manifestations such as colitis and food allergies. Allogeneic hematopoietic stem cell transplantation (HSCT) can restore T cell regulatory function but has been previously associated with poor outcome. We describe successful HSCT in 4 patients with IPEX syndrome using a novel reduced-intensity conditioning regimen that resulted in stable donor engraftment, reconstitution of FOXP3+ T regulatory CD4+ cells, and amelioration of gastrointestinal symptoms. CiteULike    Connotea    Del.icio.us    Digg    Reddit    Technorati    What's this? This article has been cited by other articles: S. Di Nunzio, M. Cecconi, L. Passerini, A. N. McMurchy, U. Baron, I. Turbachova, S. Vignola, E. Valencic, A. Tommasini, A. Junker, et al. Wild-type FOXP3 is selectively active in CD4+CD25hi regulatory T cells of healthy female carriers of different FOXP3 mutations Blood, November 5, 2009; 114(19): 4138 - 4141. [Abstract] [Full Text] [PDF] A. R. Redding, D. B. Lew, M. E. Conley, and E. K. Pivnick An Infant With Erythroderma, Skin Scaling, Chronic Emesis, and Intractable Diarrhea Clinical Pediatrics, November 1, 2009; 48(9): 978 - 980. [PDF] O. Rubio-Cabezas, J. A.L. Minton, R. Caswell, J. P. Shield, D. Deiss, Z. Sumnik, A. Cayssials, M. Herr, A. Loew, V. Lewis, et al. Clinical Heterogeneity in Patients With FOXP3 Mutations Presenting With Permanent Neonatal Diabetes Diabetes Care, January 1, 2009; 32(1): 111 - 116. [Abstract] [Full Text] [PDF] H. Zhan, J. Sinclair, S. Adams, C. M. Cale, S. Murch, L. Perroni, G. Davies, P. Amrolia, and W. Qasim Immune Reconstitution and Recovery of FOXP3 (Forkhead Box P3)-Expressing T Cells After Transplantation for IPEX (Immune Dysregulation, Polyendocrinopathy, Enteropathy, X-linked) Syndrome Pediatrics, April 1, 2008; 121(4): e998 - e1002. [Abstract] [Full Text] [PDF]

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