Figure 3. LV-RPE65 treatment of Rpe65^R91W/R91W mice at P5 restores cone density comparable to wild type.

(A) Quantification of the number of outer segments expressing GNAT2 or S-opsin proteins in sections positioned in the middle of the transduced area was compared between LV-RPE65-treated, LV-GFP-treated and untreated animals. The LV-RPE65 group shows a clear improvement in the number of cells correctly expressing these cone markers (p<0.01, stars). Data are represented as mean ± standard error of the mean (SEM). (B) When we plot the transduced area to the number of GNAT2-labeled outer segments, a clear correlation shows that the wider the transgene expression area, the more GNAT2-positive outer segments. (C) Finally, we estimated the density of cones correctly expressing the GNAT2 in the outer segments in the middle of the transduced area of LV-RPE65-treated eyes. When compared to the density of cones in wild-type animals at the same position, we observed that LV-RPE65 injection at P5 allows to rescue 100% of the cones (p<0.001, star). Data are represented as mean ± SEM. 4m: 4 month old.