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Figure 4: RT-PCR analysis of pseudo-exon 47 skipping in PMOtreated patient cells (n=2). (a) Each PMO was tested at 1000 nM, 800 nM, 400 nM, 200 nM and 100 nM (shown left to right). The full-length patient transcript product is 650bp in length, and skipping of the pseudo-exon generates a 578 bp amplicon. UT: untreated patient samples; -ve: no template RT-PCR control. (b) Densitometry was used to analyse the relative amount of mutant and normal transcripts in treated and untreated cells. |