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Blood, 1 May 2004, Vol. 103, No. 9, pp. 3300-3302. Prepublished online as a Blood First Edition Paper on December 24, 2003; DOI 10.1182/blood-2003-11-3852. This Article Full Text (PDF) All Versions of this Article: 2003-11-3852v1 103/9/3300    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 Gao, G. Articles by Wilson, J. M Search for Related Content PubMed PubMed Citation Articles by Gao, G. Articles by Wilson, J. M Related Collections Related Articles in Blood Online Social Bookmarking                   What's this? Submitted November 20, 2003 Accepted December 14, 2003 Erythropoietin gene therapy leads to autoimmune anemia in macaques Guangping Gao, Corinna Lebherz, Daniel J Weiner, Rebecca Grant, Roberto Calcedo, Adam Bagg, Yi Zhang, and James M Wilson* Gene Therapy Program, University of Pennsylvania, Philadelphia, PA, USA; The Wistar Institute, Philadelphia, PA, USA Department of Pathology and Laboratory Medicine, University of Pennsylvania, Philadelphia, PA, USA Division of Hematology/Oncology, University of Pennsylvania, Philadelphia, PA, USA Division of Pulmonary Medicine, Children's Hospital of Philadelphia, Philadelphia, PA, USA * Corresponding author; email: wilsonjm{at}mail.med.upenn.edu. Gene therapy is being considered for the delivery of therapeutic proteins. We evaluated the delivery of the hormone erythropoietin into cynomolgus macaques via intramuscularly administered adeno-associated virus (AAV) vectors. As expected, the animals developed supraphysiologic levels of Epo and polycythemia. However, a severe anemia ensued in some animals due to an autoimmune response to endogenous and transgene derived Epo. This is the first example of gene therapy leading to inadvertent autoimmunity in primates. CiteULike    Connotea    Del.icio.us    Digg    Reddit    Technorati    What's this? Related Articles in Blood Online: Inadvertent autoimmunity in EPO gene transfer Els Verhoeyen and François-Loïc Cosset Blood 2004 103: 3248-3249. [Full Text] [PDF] Autoimmune anemia in macaques following erythropoietin gene therapy Pierre Chenuaud, Thibaut Larcher, Joseph E. Rabinowitz, Nathalie Provost, Yan Cherel, Nicole Casadevall, Richard J. Samulski, and Philippe Moullier Blood 2004 103: 3303-3304. [Abstract] [Full Text] [PDF] This article has been cited by other articles: Y. Lu and S. 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Galipeau Erythropoietin Delivery by Genetically Engineered Bone Marrow Stromal Cells for Correction of Anemia in Mice with Chronic Renal Failure J. Am. Soc. Nephrol., June 1, 2006; 17(6): 1576 - 1584. [Abstract] [Full Text] [PDF] Y. Lippin, M. Dranitzki-Elhalel, E. Brill-Almon, C. Mei-Zahav, S. Mizrachi, Y. Liberman, A. Iaina, E. Kaplan, E. Podjarny, E. Zeira, et al. Human erythropoietin gene therapy for patients with chronic renal failure Blood, October 1, 2005; 106(7): 2280 - 2286. [Abstract] [Full Text] [PDF] V. R. Arruda, H. H. Stedman, T. C. Nichols, M. E. Haskins, M. Nicholson, R. W. Herzog, L. B. Couto, and K. A. High Regional intravascular delivery of AAV-2-F.IX to skeletal muscle achieves long-term correction of hemophilia B in a large animal model Blood, May 1, 2005; 105(9): 3458 - 3464. [Abstract] [Full Text] [PDF] J. Schuettrumpf, R. W. Herzog, A. Schlachterman, A. Kaufhold, D. W. Stafford, and V. R. Arruda Factor IX variants improve gene therapy efficacy for hemophilia B Blood, March 15, 2005; 105(6): 2316 - 2323. [Abstract] [Full Text] [PDF] V. M. Rivera, G.-p. Gao, R. L. Grant, M. A. Schnell, P. W. Zoltick, L. W. Rozamus, T. Clackson, and J. M. Wilson Long-term pharmacologically regulated expression of erythropoietin in primates following AAV-mediated gene transfer Blood, February 15, 2005; 105(4): 1424 - 1430. [Abstract] [Full Text] [PDF] P. R. Johnson, B. C. Schnepp, M. J. Connell, D. Rohne, S. Robinson, G. R. Krivulka, C. I. Lord, R. Zinn, D. C. Montefiori, N. L. Letvin, et al. Novel Adeno-Associated Virus Vector Vaccine Restricts Replication of Simian Immunodeficiency Virus in Macaques J. Virol., January 15, 2005; 79(2): 955 - 965. [Abstract] [Full Text] [PDF] P. Chenuaud, T. Larcher, J. E. Rabinowitz, N. Provost, Y. Cherel, N. Casadevall, R. J. Samulski, and P. Moullier Autoimmune anemia in macaques following erythropoietin gene therapy Blood, May 1, 2004; 103(9): 3303 - 3304. [Abstract] [Full Text] [PDF]

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