Figure 6. CAG-ASO treatment partially improves SCA7 retinal degeneration in mice.
A) Effect of CAG-ASO knock-down on visible aggregates in the retinas of SCA7 266Q knock-in mice. Mice received a single intravitreal injection of 50 μg of CAG-ASO in the right eye, and a single intravitreal injection of PBS in the left eye. Retinal sections were immunostained with an anti-Ataxin-7 antibody (green) and counterstained with Hoechst 33342 (blue) at 6 weeks after intravitreal injection. ONL = outer nuclear layer; INL = inner nuclear layer; GCL = ganglion cell layer. Scale bar = 20 μm B) Filter trap assay to quantify reduction of Ataxin-7 aggregates in the retinas of SCA7 266Q knock-in mice. Mice received a single intravitreal injection of 50 μg of CAG-ASO in the right eye, and a single intravitreal injection of PBS in the left eye. Here we see densitometry quantification of Ataxin-7 immunoreactivity in filter trap assays at 6 weeks post-injection. **P < 0.01; ***P < 0.001; ANOVA with post-hoc Tukey test. n = 5 mice / group. C) Quantification of mean maximal b-wave amplitudes for ERG analysis performed on untreated wild-type (non-transgenic) and SCA7 266Q mice at 4 weeks after intravitreal injection of 50 μg of CAG-ASO in the right eye, and intravitreal injection of PBS in the left eye. *P < 0.05, **P < 0.01; ***P < 0.001; ANOVA with post-hoc Tukey test. n = 8 – 11 mice / group. D) Quantification of mean maximal b-wave amplitudes for ERG analysis performed on untreated wild-type (non-transgenic) and SCA7 266Q mice at 6 weeks after intravitreal injection of 50 μg of CAG-ASO in the right eye, and intravitreal injection of PBS in the left eye. **P < 0.01; ***P <0.001; ANOVA with post-hoc Tukey test. n = 8 – 13 mice / group. Error bars = s.e.m.