Figure 4.

Intravitreal Administration of 7m8.CMV.hCLN6 Slowed Loss of Photoreceptor Function and Photoreceptor Cells in Cln6^nclf Mice

(A) Scotopic ERG a-wave amplitudes of Cln6^nclf mice after injection of 7m8.CMV.hCLN6 at P5–P6. Wild-type: n = 5–7 eyes, N = 2; Cln6^nclf untreated: n = 10–11 eyes (1–6 months), N = 4, n = 5 (9 months), N = 2; null: n = 3–6 eyes, N = 2; 1 × 10⁹ vg/eye: n = 11–13 eyes (1–6 months), N = 4, n = 5 (9 months), N = 2; 1 × 10¹⁰ vg/eye: n = 12 eyes (1–6 months), N = 4, n = 4 (9 months), N = 2. **p < 0.01 and ***p < 0.001 (2-way ANOVA with Bonferroni post-test). (B) Scotopic ERG traces of a Cln6^nclf mouse 9 months after the administration of 1 × 10⁹ vg 7m8.CMV.hCLN6 in one eye (red). Contralateral uninjected eye is in black. (C) hCLN6 staining (green) on treated and untreated eyes at 6 months. GFAP (white) and Rhodopsin (red) showed better morphology in treated eyes. (D) Quantification of photoreceptor rows and (E) PKCα⁺ cells at 6 months. ***p < 0.001 (Kruskal-Wallis test). (F) hCLN6 (green) and PKCα (red) staining on treated and untreated eyes at 9 months. (G) Quantification of photoreceptor rows and (H) PKCα⁺ cells at 9 months post-treatment. **p < 0.01 and *p < 0.05 (Kruskal-Wallis test). Scale bars, 25 μm.