Fig. 3: In vivo base editing in mice.
From: High-efficiency base editing in the retina in primates and human tissues
a, Experimental design. Dual AAV9-PHP.eB-SABE1 was delivered by subretinal injection. Eyes were harvested at 2, 4 or 8 weeks after injection and the retina and RPE/choroid/sclera were processed separately. Illustration in a and illustrations of the AAV ITRs and AAV capsid in a created with BioRender.com. b, Representative immunofluorescence images of ABE(N) and ABE(C) expression in the photoreceptor layer (scale bar, 12.5 µm) and RPE layer (scale bar, 25 µm) of Abca4ms1961G/G wild-type mice at 7 weeks after injection. Gray, Hoechst; cyan, ABE(N); yellow, ABE(C). c, In vivo base-editing efficiencies with the STGD-gRNA targeting the humanized allele in Abca4hu1961E/ms1961G(KO) mice in the retina and RPE/choroid/sclera at different time points after treatment. The analysis was performed on the humanized allele only. Results were obtained from four (week 2) and six (weeks 4 and 8) biological replicates (eyes) and are presented as the mean ± s.d. d, In vivo base-editing efficiencies with the ms-gRNA targeting the mouse alleles in Abca4ms1961G/G (wt) mice in the retina and RPE/choroid/sclera at 4 weeks after treatment. The analysis was performed on both mouse alleles. Results were obtained from four biological replicates (eyes) and are presented as mean ± s.d.
