Histopathological Defects in Intestine in Severe Spinal Muscular Atrophy Mice Are Improved by Systemic Antisense Oligonucleotide Treatment
Fig 6
Systemic delivery of PMO25 increased SMN2 exon 7 inclusion and SMN protein expression in intestine.
(A) Representative image of reverse transcriptional polymerase chain reaction (PCR) showed the partial increase of full-length SMN2 in SMA mice after PMO25 treatment. (B) Quantitative real-time PCR of full-length SMN2 to Δ7 SMN2 transcript ratio. (C) Western blotting assay of human SMN protein in intestine tissues from SMA and PMO25 treated SMA mice. β–tubulin was used as loading control. (D) Semi-quantification of SMN protein relative to tubulin control. Data were normalized to the ratio of SMN/tubulin in untreated SMA mice. (N = 3, *P< 0.05)