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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 (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 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 Social Bookmarking                   What's this? Submitted March 14, 2002 Accepted May 6, 2002 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 A Blau* * Corresponding author; email: tblau{at}u.washington.edu. 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 two 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. 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