Single ICM delivery of tcDNA or PMO. (A) Carboxyfluorescein-conjugated PMO detected 48 h post-ICM delivery. Representative images showing a sagittal section (scale bar 2 mm), cerebellum lobules IV-V (CBL), hippocampus dentate gyrus (HIPPO), and the lateral amygdala (LA) (scale bars 50 µm); n = 3. (B) Quantification of exon 51-skipping levels via RT-qPCR in different brain regions (CBL: cerebellum, CX: cortex, HIPPO: hippocampus, and SC: spinal cord) after a single ICM administration of tcDNA or PMO. Results are expressed as means ± SEMs; n = 3 mdx52-PMO and n = 4 mdx52-tcDNA. RM two-way ANOVA: structure effect p = 0.1810; ASO chemistry effect p = 0.2623.