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Figure 3: Nested RT-PCR analysis of dystrophin expression in DMD patient samples treated with AOs targeting the pseudo-exon. (a) The mutant transcript containing the pseudo-exon is 650 bp in length, and skipping of the pseudo-exon represents in a 578 bp amplicon. AOs were tested at 400 nM, 200 nM, 100 nM, 50 nM, 25 nM and 10 nM (shown left to right). UT: untreated patient samples. +ve: PCR amplification of normal dystrophin gene transcript. -ve: no template RT-PCR control. (b) Densitometry was used to analyse the relative amounts of mutant and normal transcripts in treated and untreated cells. |