Figure 2.
Figure 2. Sex difference in rAAV expression is independent of transgene cDNA and promoter. (A) Schematic of rAAV-2 CAGG tsFlk-1 in which the hFIX cDNA of rAAV-2 CAGG-FIX has been replaced with a 2.3-kb tsFlk-1 cDNA (top panel). Stable serum levels of tsFlk-1 assessed by ELISA, 12 weeks after portal vein infusionof1 × 1011 rAAV-2 CAGG tsFlk-1 vector particles in cohorts (n = 6) of male (M) and female (F) B6.CB17-Prkdc-SzJ mice (bottom panel). (B) Schematic of rAAV LSP-FIX in which the CAGG promoter has been replaced with an LSP (top panel). Stable plasma hFIX levels 12 weeks after portal vein infusion of 1 × 1011 rAAV LSP-FIX vector particles in cohorts (n = 3) of male (M) and female (F) B6.CB17-Prkdc-SzJ mice (bottom panel). All transgene expression values are depicted as an average together with the standard error of the mean.

Sex difference in rAAV expression is independent of transgene cDNA and promoter. (A) Schematic of rAAV-2 CAGG tsFlk-1 in which the hFIX cDNA of rAAV-2 CAGG-FIX has been replaced with a 2.3-kb tsFlk-1 cDNA (top panel). Stable serum levels of tsFlk-1 assessed by ELISA, 12 weeks after portal vein infusionof1 × 1011 rAAV-2 CAGG tsFlk-1 vector particles in cohorts (n = 6) of male (M) and female (F) B6.CB17-Prkdc-SzJ mice (bottom panel). (B) Schematic of rAAV LSP-FIX in which the CAGG promoter has been replaced with an LSP (top panel). Stable plasma hFIX levels 12 weeks after portal vein infusion of 1 × 1011 rAAV LSP-FIX vector particles in cohorts (n = 3) of male (M) and female (F) B6.CB17-Prkdc-SzJ mice (bottom panel). All transgene expression values are depicted as an average together with the standard error of the mean.

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