Figure 6. Assessment of efficacy of miRNA-regulated TetR-KRAB control of transgene expression in vivo.

(a) Schematic diagram of the AAV vectors used for in vivo studies. (b) GFP fluorescence was measured in live animals at 8 weeks after injection with recombinant AAVs. Mice received AAVs encoding the TetR-KRAB sequence without copies of miRNA target located downstream. Animals did or did not receive doxycycline in their drinking water. The color scale next to the images indicates the signal intensity. (c) & (d) Results at 8, 12 and 16 weeks post injection are expressed in counts/mm²/second. Data shown are mean and error bars indicate the SD. * Statistically significant differences P<0.05 (two-tailed, unpaired Student’s t-test), n = 4 per group.