Fig. 4: Formation of functional rod CNG channel in Cnga1-/- mice after gene augmentation therapy. | Communications Medicine

Fig. 4: Formation of functional rod CNG channel in Cnga1-/- mice after gene augmentation therapy.

From: Gene augmentation therapy restores vision and preserves photoreceptors in a mouse model of CNGA1-retinitis pigmentosa

Fig. 4

A Map of gene expression cassette for subretinal delivery and the experimental timeline. The data shown in (B-E) are from mice at 3 months of age. B Representative confocal images showing the expression of correctly localized CNGA1 protein (red) and lack of cGMP accumulation in treated retina. Scale bar, 10 μm. C Representative scotopic (dark-adapted) ERG waveforms in response to a series of flashes from wild-type mouse (left), untreated Cnga1-/- mouse (middle) and treated Cnga1-/- mouse (right). D Quantification of a-wave amplitudes of untreated and treated Cnga1-/- mice. Mean ± SEM. **P < 0.01, ***P < 0.001; two-way ANOVA with Šídák’s multiple comparisons. P values are 0.0001 at −0.5, 0.0011 at 0, and <0.0001 at higher light intensities. E Quantification of b-wave amplitudes of untreated and treated Cnga1-/- mice. Mean ± SEM. ***P < 0.001; two-way ANOVA with Šídák’s multiple comparisons. P values are 0.0006 at −2, 0.0003 at −1.5, 0.0001 at −1, and <0.0001 at higher light intensities. Treated Cnga1-/-, n = 10 eyes; untreated Cnga1-/-, n = 10 eyes.

Back to article page