Extended Data Fig. 7: ASO dose-dependent exon 5 skipping in mouse cells.
From: Therapeutic efficacy of antisense oligonucleotides in mouse models of CLN3 Batten disease
a, Sequence alignment of the most active ASO to the target Cln3 region. Cln3 exonic and intronic nucleotides are displayed as capital and lowercase letters, respectively. b, RT-PCR analysis of exon 5 splicing from RNA extracted from homozygous mCln3Δex7/8 cells transfected with increasing concentrations of ASO-26 (0.391 nM to 200 nM). Spliced products are indicated at left of gel image and size markers (bp) are shown at right of gel. Graph shows results of a single experiment. Untreated (UT) and mock (M) treated controls are included. The graph displays the percent of exon 5 skipped [Δ578/(Δ578 + Δ78) x 100] in relationship to the log of the dose. The half-maximal effective concentration (EC50) was calculated after fitting the data using nonlinear regression, variable slope.
