Extended Data Fig. 7: In vivo activity of Xon in brain.
From: Regulated control of gene therapies by drug-induced splicing
a, Representative photomicrographs (5 mice per group) showing eGFP expression from mice treated intravenously 4 weeks earlier with AAVPHPeB-Xon-eGFP, and 24 h after treatment with vehicle or LMI070 at 5 or 50 mg kg−1. Thalamus (Th), hippocampus (Hc), cerebellum (Cb) and facial motor nucleus (VII), cortex (Cx), striatum (Str), substantia nigra (SN), and medial vestibular nucleus (MV) are shown. Scale bar, 100 μm; inset scale bar, 25 μm. In the hippocampus, * and ** denote the polymorphic and CA3 areas, respectively. In the cerebellum, * denotes the deep cerebellar nuclei. b, Splicing assays for exon inclusion in the cortex and the hippocampus of mice injected with AAVPHPeB-Xon-eGFP (3 mice per group). c, RT–qPCR of human progranulin expression. Data are mean ± s.e.m. of 5 mice per group, ****P < 0.0001 vehicle versus AAV-treated groups, one-way ANOVA followed by Bonferroni’s post-hoc test. d, Splicing assays for exon inclusion in cortex samples of mice injected with AAVPHPeB-Xon-PGRN (3 mice per group).
