Fig. 1.

(A) Images of fundus photos compare the baseline (“Pre”) and d60 (“Post”) appearance and the predicted pre- and post-readministration visual field. There is extensive disease at baseline, with retinal pigment epithelial disturbance and geographical atrophy in the macula in patient CH12. Arrowheads indicate the lower border of the subretinal injection site, which was supratemporal and included the superior aspect of the macula in all three subjects. The lower border of the bleb was closer to the superior vascular arcade in CH12, whereas the lower borders for patients CH11 and NP01 were closer to the fovea. On the far right are the pre- and post-readministration visual fields. The predicted visual field changes based on the injection sites (and assuming a healthy retina) were similar for the three subjects (yellow shaded areas). Gray shaded areas denote scotomas (spots in the visual field in which vision is absent or decreased) that were altered in location at each different FO exam (only baseline scotomas are shown). (B) Full-field sensitivity threshold testing shows an increase in retinal light sensitivity (y axis shows sensitivity thresholds) in the left eyes of NP01 and CH11 by d30 persisting through the latest time point (d180), but no change in sensitivity of the previously injected eye for the three patients. There was no change in FST test results for either eye of patient CH12. (C) Improved PLR in the second eye to receive an injection of AAV2-hRPE65v2. Average pre-readministration PLR amplitudes of constriction are compared with those of post-readministration amplitudes (FOd30 to FOd180). PLR amplitudes were measured after illumination with light at 10 lux (CH12) or 0.4 lux (CH11 and NP01). *P = 0.08; **P = 0.009; ***P = 0.01.