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. 2014 Nov 7;12(2):177–185. doi: 10.1111/jth.12471

Figure 1.

Figure 1

Experimental design and vectors. Schematic representation of the expression cassettes for the splicing-competent hFVII minigenes and for the modified U1+5a, which were used to create the pFVIIwt, pFVII+5A and pU1+5a plasmids, respectively. The splicing mutation under investigation (c.859+5G>A) and restriction sites used for cloning are indicated. Numbers indicate F7 exons. ITR, inverted terminal repeat; phAAT, chimeric promoter consisting of the human α1 antitrypsin promoter and human apolipoprotein E enhancer used for liver restricted expression of the transgene; SV40pA, polyadenylation site of SV40; pU1, endogenous promoter of the U1snRNA gene. The plasmids pFVII+5A and pU1+5a have been subsequently packaged into Adeno-Associated Virus (AAV) serotype 2 and 8, respectively.