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Fig. 7. Scale-up of AAV vector–mediated liver gene transfer of secretable GAA to nonhuman primates. Scale-up of AAV vector–mediated liver gene transfer.

Published byLester Casey Modified over 5 years ago

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Presentation on theme: "Fig. 7. Scale-up of AAV vector–mediated liver gene transfer of secretable GAA to nonhuman primates. Scale-up of AAV vector–mediated liver gene transfer."— Presentation transcript:

1 Fig. 7. Scale-up of AAV vector–mediated liver gene transfer of secretable GAA to nonhuman primates.
Scale-up of AAV vector–mediated liver gene transfer of secretable GAA to nonhuman primates. (A to C) Male cynomolgus monkeys (n = 3) were treated with AAV8-hAAT-sp7-Δ8-coGAA (AAV-GAA) (2 × 1012 vg/kg) and followed up for 90 days. (A) Western blot analysis of plasma using an anti-GAA antibody. rhGAA was used as loading control. NHP, nonhuman primate. (B) GAA activity in plasma. Basal, endogenous GAA activity in monkeys #1 to #3 before injection and five additional control monkeys (n = 8). Statistical analysis: one-way ANOVA with Tukey’s post hoc. Error bars represent the SD of the mean. (C) GAA activity in liver, heart, diaphragm, biceps, and triceps. Endogenous GAA activity was measured in six monkeys treated with an unrelated AAV vector [control (Ctrl)]. Statistical analysis: multiple t test. Francesco Puzzo et al., Sci Transl Med 2017;9:eaam6375 Published by AAAS

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