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

Figure 4. Vivo-PMO–mediated exon skipping of the SGCG transcript.

Figure 4

(A) A multi-AON exon-skipping cocktail using vivo-PMO chemistry was employed to correct the SGCG 521ΔT reading frame in myogenic cells. RT-PCR analysis showed treatment with vivo-PMOs targeting exons 4, 5, 6, and 7 generated the Mini-Gamma reading frame–corrected transcript (red arrowhead) with minimal residual expression of the mutant 521ΔT transcript (black arrowhead). Arrows in schematic denote the location of the RT-PCR primers, and the expected amplicon lengths are indicated. (B) Normal control myogenic cells were treated with the same multiexon-skipping vivo-PMO cocktail. RT-PCR analysis showed expression of the Mini-Gamma transcript (red arrowhead), indicating that normal SGCG transcript could be skipped using vivo-PMOs. Expression of the SGCG transcript in untreated cells is indicated (black arrowhead). (C and D) Sequence analysis of PCR products confirmed the generation of the Mini-Gamma transcripts in each of the mutant cell lines, with the splicing of SGCG exons 3 and 8 demonstrating the removal of exons 4, 5, 6, and 7 from the mature transcripts.