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. 2018 May 3;3(9):e99357. doi: 10.1172/jci.insight.99357

Figure 3. 2OMePS-mediated exon skipping of the SGCG transcript.

Figure 3

(A) Correction of the SGCG 521ΔT frameshift mutation (red triangle) requires a multi-AON exon-skipping strategy targeting exons 4, 5, 6, and 7 (yellow boxes) to generate the reading frame–corrected Mini-Gamma transcript encoded by exons 2, 3, and 8. Arrows denote the location of the RT-PCR primers, and the expected amplicon lengths are indicated. Myogenic cells were exposed to a multiexon–skipping cocktail of 2OMePS AONs targeting exons 4, 5, 6, and 7. RT-PCR analysis demonstrated the expression of the 521ΔT transcript (black arrowhead), along with PCR products representing the skipping of 1, 2, and 3 exons (white arrowheads). A PCR product representing the 4-AON–mediated generation of the Mini-Gamma was observed at the expected size (red arrowhead). (B) Treatment of reprogrammed normal control fibroblasts with 2OMePS AONs targeting exons 4, 5, 6, and 7 also induced exon skipping. RT-PCR analysis demonstrated the expression of the SGCG transcript (black arrowhead), along with PCR products representing the skipping of 1, 2, and 3 exons (white arrowheads). No PCR product representing the 4-AON–mediated generation of the Mini-Gamma was observed at the expected size (red arrowhead).