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. 2016 Oct 20;23(12):846–856. doi: 10.1038/gt.2016.61

Figure 1.

Figure 1

(a, b) FXN gene expression in FRDA fibroblasts. (a) Human, (b) Mouse. qRT-PCR quantification of FXN transgene expression after pHR'SIN-cPPT-SFFV-FXN-WPRE infection of fibroblasts 2, 8 and 12 weeks post infection. Cells infected with pHR'SIN-cPPT-SFFV-eGFP-WPRE served as controls. After normalizing to endogenous GAPDH gene expression, FXN expression in the human and mouse FRDA cells reached 96- and 210-fold, respectively after infection compared with uninfected FRDA cells and 0.5- and 0.2-fold, respectively, compared with normal fibroblasts. Over time, this expression appeared to fall by ~50%, however FXN expression remained significantly greater than in untreated FRDA fibroblasts over the 12-week-period after gene delivery. Control normal human fibroblasts=GMO7492, FRDA fibroblasts=GMO3665. Control normal mouse model fibroblasts=Y47, FRDA fibroblasts=YG8sR. The data shown is the mean of two independent experiments where n=3/experiment. Error bars represent the standard error of the means s.e.m. Significance differences are represented by asterisks: *P<0.05, **P<0.01 and ***P<0.001.