Figure 7.

Promoter 770En_454P(hGRM6) Drives Stable, Functional, and Widespread Opn1mw Expression in Late Degenerated rd1 Mice

(A) AAV2(7m8)-770En_454P(hGRM6)-Opn1mw-IRES2-TurboFP635-injected rd1 mice were tested for visual acuity determined by the optomotor reflex between 28 and 38 weeks of age (n = 3) and compared to their untreated littermates (n = 5). Optogenetically treated mice showed significant restoration of visual acuity with a restoration of 65% of the visual acuity of wild-type mice (C57BL/6; n = 10). (B and C) Retinas of the treated animals were immunohistochemically evaluated for their expression profiles, as indicated in the whole mounts of (B) at 41 weeks of age and (C) at 26 weeks of age. Scale bars: 1,000 μm in (B) and 50 μm in (C). (D) 770En_454P(hGRM6) had a significant specificity for ON-bipolar cells (OBCs) also in late degenerated tissue at 26 weeks of age. INL, inner nuclear layer; GCL, ganglion cell layer. Statistical analysis by one-way ANOVA with post hoc Tukey HSD test: ∗p < 0.05; ∗∗∗p < 0.001. Data are represented as mean ± SD of biological replicates.