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. 2014 Aug 12;22(9):1614–1624. doi: 10.1038/mt.2014.131

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Copyright © 2014 American Society of Gene & Cell Therapy

This work is licensed under a Creative Commons Attribution-NonCommercial-NoDerivs 3.0 Unported License The images or other third party material in this article are included in the article's Creative Commons license, unless indicated otherwise in the credit line; if the material is not included under the Creative Commons license, users will need to obtain permission from the license holder to reproduce the material. To view a copy of this license, visit http://creativecommons.org/licenses/by-nc-nd/3.0/

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Increased therapeutic efficiency using the hyperactive PB platform. CB17/IcrTac/Prkdc^scid mice were hydrodynamically transfected with (a,c,e) 500 ng or (b,d,f) 50 ng of PB-hFIXco (a–f), (c,d; triangle) PB-hFIXco/IR_micro or (e,f, triangle) PB-hFIXco/IR_mut16 transposon plasmids along with (a,c) 1,000 ng or (b,d) 100 ng mPB (triangle a,b) or hyPB-expressing plasmid (square a,b) or empty control plasmid (hatched lines). hFIX expression was measured on plasma samples collected at the indicated times by a specific enzyme-linked immunosorbent assay assay. Results were presented as mean ± SEM. n.s. indicates not significant; *P < 0.05; **P < 0.01; ***P < 0.001 (n = 3 mice/group).