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2-terminal epitope tag from influenza virus hemagglutin and nuclear localization signal from SV40 large T antigen (4, 16). Reporter construct AAV.Z12mEpo3S3 contains the 12 ZFHD1-binding sites (4), a minimal interleukin-2 promoter (4), a chimeric intron from pCl vector (Promega), a murine Epo cDNA, an SV40 late gene 3′ UTR, and a 2.7-kb stuffer containing a 1367-bp internal portion of human placental alkaline phosphatase coding sequence followed by a 626-bp portion of the human growth hormone 3′ UTR and a 720-bp portion of the 3′ rabbit β-globin intron and PAS (4). Reporter construct AAV.Z12rmEpo2S2 is similar except that the Epo cDNA is derived from a rhesus monkey Epo and the vector lacks the chimeric intron and the 720-bp rabbit β-globin portion of the stuffer. Reporter construct AAV.CM-VrmEpo3 contains a human CMV enhancer-promoter, a chimeric intron from pCl vector, a rhesus monkey Epo cDNA, and an SV40 late gene 3′ UTR. Recombinant AAV was generated by cloning CMVTF1, Z12mEpo3S3, Z12rmEpo2S2, and CMVrmEpo3 expression cassettes into Xba I-restricted pSub 201 backbone (17). The resulting plasmids were used to produce recombinant AAV by a triple transfection method (18) or by an Ad/AAV hybrid system (17).
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13 genomes per kilogram) peak Epo levels of 560 and 120 mU/ml.
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Antibodies to the influenza virus hemagglutinin tag were detected in serum of the nonhuman primate 3 months after gene transfer, although it is unclear whether their presence predicts functional cellular immune responses.
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Funding was provided by grants from the NIH [P30 DK47757-05 (J.M.W.) and P01 AR/NS43648-03 (H. L. Sweeney)], the Muscular Dystrophy Association, ARIAD Pharmaceuticals, and Genovo, a company J.M.W. founded and in which he holds equities. Technical support from N. Courage was greatly appreciated. W. Xiao provided plasmids for AAV production.
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