Figure 1: SMN splicing and protein rescue in SM25 transgenic SMA mice.
From: Therapeutic activity of modified U1 core spliceosomal particles
(a) Northern blot analysis of ExSpeU1 SM25 expression in Smn1 heterozygous mice at P40 (B, brain; H, heart; K, kidney; L, liver; M, muscle; SC, spinal cord). ExSpeU1 expression levels are normalized to U6 snRNA and brain is set to 1. Data are the mean±s.e.m. of four animals except for SC (three mice). ‘Neg.’ denotes ExSpeU1−/− mice. (b) SMN2 specific splicing assay in Smn1 heterozygous mice at P40. Data represent the mean±s.e.m. in eight ExSpeU1 +/− and nine ExSpeU1 −/− animals except for SC (three mice; Student’s t-test *P≤0.05, ***P≤0.001). Bars on the right represent SMN2 exon 7 inclusion and exclusion splicing isoforms. (c) SMN protein expression levels detected by western blotting in SMA-affected (Smn1−/−, SMN2+/+), SMA-rescued (Smn1−/−, SMN2 +/+; ExSpeU1 +/−), and control animals (Smn1+/−, SMN2+/+, ExSpeU1+/−) at P2–3.
