Figure 2.

Subretinal Delivery of AAV2/8.CMV.hCLN6 Did Not Rescue Photoreceptors in Cln6^nclf Mice

(A) Scotopic ERG a-wave amplitudes of Cln6^nclf mice that received subretinal injections with AAV2/8.CMV.hCLN6 at P9–P11. Wild-type: n = 5–7 eyes, N = 2; Cln6^nclf untreated: n = 14–17 eyes, N = 5; null: n = 3–4 eyes, N = 1; 1.5 × 10⁸ vg/eye: n = 6 eyes, N = 1; 1.5 × 10⁹ vg/eye: n = 7 eyes, N = 2; 1.5 × 10¹⁰ vg/eye: n = 6–8 eyes, N = 2. ***p < 0.001 (2-way ANOVA with Bonferroni post-test). (B–D) hCLN6 staining confirmed transgene expression in photoreceptors at 6 months after transduction with 1.5 × 10⁸ vg/eye (B) and high magnification (B’), 1.5 × 10⁹ vg/eye (C) and high magnification (C’), and 1.5 × 10¹⁰ vg/eye (D) and high magnification (D’). Scale bar, 25 μm.