Exon Exchange Approach to Repair Duchenne Dystrophin Transcripts
Figure 5
Dosing study of AS2-E24-DISE-AS4 molecule.
(A) RT-PCR analysis using primers A and C of NIH3T3 cells cotransfected with 250 ng of dystrophin minigene and 100, 200, 400 and 750 ng of pSMD2-AS2-E24-DISE-AS4 plasmids. (B) Histogram showing numbers of parental and exchanged transcripts evaluated by absolute quantitative real-time RT-PCR illustrated in Fig. 4A. PCR1 corresponds to the parental transcripts whereas PCR2 to the repaired transcripts. PCR1+PCR2 corresponds to the totality of the dystrophin minigene transcripts. (C) ExChange efficiency of the dosing study estimated by calculating the ratio between levels obtained for repaired transcripts (PCR2) and whole dystrophin minigene transcripts (PCR1+PCR2). Representative results from two independent transfection experiments.