Extended Data Fig. 9: ASO activity in the CNS of ASO-treated mice.
From: Therapeutic efficacy of antisense oligonucleotides in mouse models of CLN3 Batten disease
a, RNA extracted from the cortex, thalamus, striatum, brain stem, spinal cord, and kidney of 19-week-old Cln3+/Δex7/8 (+/Δ78) and Cln3Δex7/8/Δex7/8 (Δ78/Δ78) mice treated at P1 or P2 with ASO-C or ASO-26. b, Quantification of exon 5 skipping in (a). Bars are mean ± s.e.m. n = 4 for all but Cln3+/Δex7/8 ASO-C striatum n = 3; *P < 0.05, ****P < 0.0001, one-way ANOVA, Tukey’s multiple comparisons test within each tissue. c, Brain map indicating regions sampled for SCMAS and GFAP immunohistochemistry. The regions numbered 1–5 are the somatosensory cortex, visual cortex, striatum, thalamus, and hippocampus, respectively.
