Fig. 11.

Efficiency of 2′-OMeUtaPS and dTtaPSat inducing the excision of exon 23 from the mdx mouse dystrophin pre-mRNA upon trans-fection of the PMO or PNA sequence 14 or 15, respectively, in mdx mouse myotubes. The concentration of 2′-OMeUtaPS or dTtaPS was kept at 2 µM in serum containing medium. Lipofectamine® 2000 (LF) was used as a transfection reagent in serum free-medium at a concentration recommended by the supplier. Total RNA was extracted from transfected myotubes and amplified by nested RT-PCR using appropriate sets of DNA primers (see Experimental section). Two major PCR products were separated by electrophoresis on an agarose gel; the larger 633 bp and shorter 420 bp secondary PCR products correspond to the unspliced and correctly spliced pre-mRNA exon 23, respectively. SM, size marker.