Figure 6.

Comparison of retinal structure and function in GC1KO mice treated with the GMP vector versus the vector used in GLP tox studies 1 month p.i.

Photopic b wave amplitudes were significantly higher in GC1KO mouse eyes treated with 1.5 × 10¹¹ vg/mL (1.5 × 10⁸ vg/eye) of AAV5-GUCY2D (relative to untreated controls), regardless of whether treatment was performed with GMP or Tox lots. At a lower dose of 3.3 × 10¹⁰ vg/mL (3.3 × 10⁷ vg/eye), photopic improvements were not significant, but genuine waveforms were observed in a similar number of animals treated with the GMP lot (6 of 20) and Tox lot (7 of 18) vectors (left). No significant improvements in scotopic b wave amplitudes were observed in vector-treated eyes (center). ∗p < 0.05, ∗∗∗∗p < 0.0001 as determined by one-way ANOVA with Tukey’s post-test analysis. No significant difference in mean ONL thickness was observed in mice treated with the GMP versus Tox lot vector (right). ∗∗p < 0.01, ∗∗∗p < 0.001, ∗∗∗∗p < 0.0001, as determined by two-way ANOVA with Tukey’s post-test analysis.