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. 2021 Jul 14;29(1-2):81–93. doi: 10.1038/s41434-021-00258-6

Fig. 5. Complete correction of RPGR-ORF15 in AAV-treated mice.

Fig. 5

A Representative confocal images of CRISPR-targeted ORF15 (AAV CRISPR-ORF15) rd9 retinal cross sections. Staining with additional RPGR antibodies c100 and GT335 confirm RPGR-ORF15 expression in treated retina. B Confocal images of control C57Bl/6 retinal cross sections stained with RPGR-570 and AHI1 to demonstrate the assay used to quantify rescue percentages. In healthy control retina, RPGR-ORF15 (570) and AHI1 are near perfectly colocalized at the connecting cilium. C RPGR-570 and AHI1 staining in AAV CRISPR ORF15 treated rd9 retinal cross sections. All connecting cilia are positive for AHI1 and a subset are also positive for 570 indicating correction of RPGR-ORF15 in these cells D Quantification of rescue in four large fields of AAV CRISPR ORF15 treated retina. Injections were performed in 3–6 week old rd9 mice, and tissues were collected and analyzed 8–12 weeks post injection.