Fig. 1

A novel RPE-specific AAV vector. (A) Schematic diagram of AAV vectors screened for specific targeting of RPE cells. (B) Whole mounts of mice retina and RPE-Choroid-Sclera after intravitreal injection of AAV2-GFP or AAV206-GFP for 14 days. GFP expression (green) indicates positively transduced cells. RPE cells are marked by ZO-1 (red). Scale bar: 1000 μm (left panel), 20 μm (right panel). (C, D) Statistical results of GFP fluorescence areas in RPE (C) and retinal (D) tissues from (B). n = 8 eyes, ****P < 0.0001 by Student’s t-test; values shown as mean ± SD. (E) Frozen sections of mice retina 14 days post-transduction with AAV2-GFP or AAV206-GFP. n = 6 eyes, Scale bar: 1000 μm (low magnification), 40 μm (high magnification). (F) Quantification of GFP fluorescence intensity in (E) using ImageJ. (G) CNV was examined in vivo by OCT after the induction of CNV for 7 days. Scale bar: 200 μm. (H, I) Measurements of CNV lesion length (yellow dotted line) and thickness (red dotted line) from OCT. n = 8 eyes, ****P < 0.0001 by one-way ANOVA; values shown as mean ± SD. (J) FFA to detect CNV lesion leakage, with representative color fundus photographs (CFP) and FFA images for each group after the induction of CNV for 7 days. (K) Distribution of CNV lesion grades. *P < 0.05, ****P < 0.0001 by the Kruskal-Wallis test. (L) RPE-choroid-sclera whole mounts stained with IB4 (red) to measure CNV areas after the induction of CNV for 7 days, with microglia marked by iba1 (green). Scale bar: 1000 μm (low magnification), 100 μm (high magnification). (M) Quantification of CNV areas in (L) using ImageJ. n = 8 eyes, **P < 0.01, ****P < 0.0001 by one-way ANOVA; values shown as mean ± SD