Molecular Therapy

FIGURE 3

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Helper-Independent Sleeping Beauty Transposon–Transposase Vectors for Efficient Nonviral Gene Delivery and Persistent Gene Expression in Vivo

Jacob Giehm Mikkelsen, Stephen R. Yant, Leonard Meuse, Zan Huang, Hui Xu and Mark A. Kay

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Figure 3.

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Improved transgene transposition with SB HITT vectors. Serum hAAT levels in mice receiving 24 mug pT/hAAT, 30 mug pT/hAAT.PGK-SB, or 30 mug pT/hAAT.MTH-SB (data from Fig. 2C) compared with 24 mug pT/hAAT + 20 mug pCMV-SB (1:1 ratio based on hAAT and SB transposase gene copy numbers) and 24 mug pT/hAAT + 0.8 mug pCMV-SB (25:1). When necessary, pUC19 "stuffer" DNA was included to ensure that each mouse was injected with the same total amount of DNA (44 mug). Gene copy numbers determined amounts of DNA in the two-component injections; hAAT and SB transposase genes were injected in equal copy numbers for HITT vector and 1:1 two-component injections. Average values plusminus standard deviation are shown. n = 5 mice per group.

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