Figure 5.

AAV5 vectors containing the human GUCY2D or murine Gucy2e coding sequence, manufactured via TTx, and purified via 2 CC confer significant improvements in retinal function to GC1KO mice

(A) The AAV-GUCY2D and AAV5-Gucy2e vectors demonstrated stable and dose-responsive restoration of cone PR function for at least 3 months after injection. There were no significant differences in photopic b wave amplitudes between AAV5-GUCY2D- versus AAV5-Gucy2e-treated eyes at any equivalent dose or time point. By 2 months p.i., there were no significant differences in scotopic b wave amplitudes. A summary of all statistical comparisons can be found in Figures S7–S10. (B) No significant difference in mean ONL thickness was observed across treatment groups. ∗∗p < 0.01, ∗∗∗p < 0.001, ∗∗∗∗p < 0.0001, as determined by two-way ANOVA with Tukey’s post-test analysis. (C) Two representative photopic (cone-mediated) ERG waveforms from mice treated with 3.3 × 10¹⁰ vg/mL (3.3 × 10⁷ vg/eye) of AAV5-GUCY2D were made via TTx and purified via 2-column chromatography (2 CC). (D) Despite the barely visible ERG waveforms, these mice had near-normal cone-mediated behavior.