Figure 5 | Scientific Reports

Figure 5

From: Spinal muscular atrophy phenotype is ameliorated in human motor neurons by SMN increase via different novel RNA therapeutic approaches

Figure 5

SMN upregulation in SMA iPSCs using TALE-TFs.

(A) A schematic representation of the interaction of a TALE-TF construct with SMN2 promoter. (B–D) TALE-TFs increased the transcription of the SMN2 gene in SMA iPSCs. Quantitative RT-PCR analysis on SMA iPSCs treated with various TALE-TF combinations demonstrated the transcriptional activation of the SMN2 gene in both the FL SMN and the SMNΔ7 transcripts and highlighted that TALE-TF combination 1 + 3 + 5 was the most effective of all tested combinations (*p < 0.01) (B). Accordingly, the immunocytochemistry assay documented a higher level of SMN protein expression (green) in SMA iPSCs transfected with TALE-TF combination 1 + 3 + 5 than in mock-transfected SMA iPSCs (C). Nuclei are labeled with DAPI (blue). TALE-TF-treated cells were more brightly fluorescent than mock-treated cells (*p < 0.01) in quantitative analysis (D). Values represent means ± s.e.m. from five independent experiments performed in triplicate. Scale bar: 75 μm.

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