Extended Data Fig. 1: c.5882G>A haplotype categories and percent of patients who have a fully correctable allele by SABE.

Our analysis is based on published genetic data from 150 Stargardt patients mostly from European descent⁶. A) Possible haplotypes found in Stargardt patient with at least one allele including c.5882G>A. B) Counts and percentage of different haplotypes found in the published study. C) Fraction of patients who have at least one allele, which is fully or partially correctable by SABE. D) Pie chart showing fraction of STGD patients carrying the p.Gly1961Glu mutation who have at least one allele, which is fully (green) or partially (red) correctable by SABE. The calculation is based on Lee W et al.⁶ and was applied on a large number of patients analyzed by Cornelis SS et al⁵⁰. E) Depiction of possible ABCA4 genotypes with respect to SABE-mediated correction. Concerning patients with homozygous haplotype 1, we expect other yet unknown modifier(s) (labeled with question mark) since homozygosity for c.5882G>A alone was shown not to lead to disease⁵¹. Therefore, we considered haplotype 1 as “partially correctable”. VUS, variant of uncertain significance.