Extended Data Fig. 10: In vivo ABCA4 base editing in non-ocular tissues from subretinally injected mice and NHPs.
From: High-efficiency base editing in the retina in primates and human tissues
A) In vivo base-editing efficiencies in mouse tissues at the A7 and A8 site in Abca4 gDNA and mRNA in the retina and RPE/choroid/sclera as well as in Abca4 gDNA of different tissues of the visual pathway, cortex, cerebellum and peripheral organs. Tissues were harvested 5 weeks after subretinal injection. Results were obtained from two biological replicates (eyes) and one replicate (non-occular tissues). Results are presented as mean. B) In vivo base-editing efficiencies in NHPs at the A8 site in ABCA4 gDNA and mRNA in the retina and RPE/choroid as well as in ABCA4 gDNA of different tissues of the visual pathway, cortex, cerebellum and peripheral organs. Tissues were harvested 20 weeks after subretinal injection. Results were obtained from four biological replicates (eyes) and two biological replicates (non-occular tissues). Results are presented as mean ± s.d. Illustrations in a and b created with BioRender.com.
