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Prepublished online as a Blood First Edition Paper on May 24, 2002; DOI 10.1182/blood-2002-03-0792. This Article Full Text Full Text (PDF) All Versions of this Article: 2002-03-0792v1 100/6/2026    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 Neff, T. Articles by Blau, C. A. Search for Related Content PubMed PubMed Citation Articles by Neff, T. Articles by Blau, C. A. Related Collections Gene Therapy Social Bookmarking                   What's this?  Previous Article  |  Table of Contents  |  Next Article  Blood, 15 September 2002, Vol. 100, No. 6, pp. 2026-2031 GENE THERAPY Pharmacologically regulated in vivo selection in a large animal Tobias Neff, Peter A. Horn, Victor E. Valli, Allen M. Gown, Scott Wardwell, Brent L. Wood, Christof von Kalle, Manfred Schmidt, Laura J. Peterson, Julia C. Morris, Robert E. Richard, Tim Clackson, Hans-Peter Kiem, and C. Anthony Blau From the Divisions of Hematology and Oncology, Department of Medicine, and the Department of Laboratory Medicine, University of Washington, Seattle; the Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, WA; the Department of Veterinary Pathobiology, University of Illinois, Urbana; PhenoPath Laboratories, Seattle, WA; ARIAD Gene Therapeutics, Cambridge, MA; and Department I of Internal Medicine and Institute for Molecular Medicine and Cell Research, University of Freiburg, Germany. The inefficiency of gene transfer has greatly hindered gene therapy. In vivo selection may increase the frequency of genetically modified cells, thereby circumventing this critical limitation. Here we demonstrate regulated in vivo selection in a large animal. CD34+ cells from 2 dogs were engineered to express a conditional derivative of the thrombopoietin receptor (F36Vmpl). Activation of the receptor through administration of a dimerizing drug, AP20187, produced reversible, drug-dependent rises in genetically modified red cells, white cells, and platelets in both animals, with minimal side effects. Cell growth switches could greatly enhance the efficacy and applicability of gene and cell therapy. © 2002 by The American Society of Hematology.   CiteULike    Connotea    Del.icio.us    Digg    Reddit    Technorati    What's this? This article has been cited by other articles: H. Abdel-Azim, Y. Zhu, R. Hollis, X. Wang, S. Ge, Q.-L. Hao, G. Smbatyan, D. B. Kohn, M. Rosol, and G. M. Crooks Expansion of multipotent and lymphoid-committed human progenitors through intracellular dimerization of Mpl Blood, April 15, 2008; 111(8): 4064 - 4074. [Abstract] [Full Text] [PDF] M. Allocca, U. Di Vicino, M. Petrillo, F. Carlomagno, L. Domenici, and A. 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