Figure 3: ExSpeU1 SM25 has a minimal genome-wide effect in spinal cord.
From: Therapeutic activity of modified U1 core spliceosomal particles
(a) Difference in total transcript expression in the spinal cord. Volcano plots for changes in gene expression between wild-type (Smn1+/−, SMN2 +/+; ExSpeU1−/−) and SM25 ExSpeU1 transgenic animals (Smn1+/−, SMN2+/+, ExSpeU1+/−; n=6 with three samples per group). Horizontal and vertical dashed lines indicate cutoff values (FDR value of 0.05 and absolute logarithmic fold change >2). Genes having a significantly altered expression are emphasized in red and green. (b) Difference in expression of individual exons in the spinal cord in Mus Musculus transcripts. The plot shows exon-level changes of all exons obtained from UCSC mm10 RefGene. The horizontal axis shows variation (log2-fold) in expression between wild-type (WT) and SM25 ExSpeU1 transgenic animals while the vertical axis refers to P value after Benjamini–Hochberg correction. (c) SMN2−specific splicing assay in the spinal cord of WT and transgenic animals. Bars on the right represent SMN2 exon 7 inclusion and exclusion splicing isoforms. (d) Data obtained from c represent the mean±s.e.m. of SMN2 splicing changes in three WT and three transgenic animals (Student’s t-test *P≤0.05).
