Extended Data Fig. 10: Assessing off-target activity of ASO-26.
From: Therapeutic efficacy of antisense oligonucleotides in mouse models of CLN3 Batten disease
a, The predicted ASO-26 binding location in Zmynd8 pre-mRNA is shown. The arrows indicate the primer binding location. b, RT-PCR analysis of RNA extracted from the cortex of Cln3Δex7/8/Δex7/8 mice treated with either ASO-C or ASO-26. The region of the Zmynd8 transcript predicted to be affected by ASO-26 base pairing was amplified by RT-PCR and products were separated on a 6% polyacrylamide gel. Two alternatively spliced Zmynd8 isoforms were detected at equal levels in all samples. RT-PCR of samples were analyzed twice with similar results. c, Quantification of exon 3 skipped shows that ASO-26 treatment does not alter Zmynd8 alternative splicing. Bars are mean ± s.e.m. n = 2 mice.
