Figure 5

Evaluation of AAV1-U7ex23 benefit in cardiotoxin regenerated and mdx muscles. Wt, wt + CTX and mdx TAs were injected with AAV1-U7ex23 and the mice were sacrificed 3 weeks later. (A) Level of U7ex23 RNA estimated by relative qPCR and expressed as a percentage of U7ex23 present in wt muscles. Non inj., wt non-AAV injected TA. (B) Quantification of AAV genomes per nucleus by absolute qPCR in wt, wt + CTX and mdx muscles injected with AAV- U7ex23 vector and corrected by the correcting factors. AAV genome content is expressed as the AAV genome number relative to the value obtained for wt muscles. (C) U7ex23 RNA amount normalised by the AAV genome numbers and expressed as a percentage of wt RNAs. (D) Level of exon 23 skipping estimated by nested RT-PCR. The 901 bp PCR product corresponds to full-length dystrophin transcripts whereas the 688 bp product corresponds to transcripts lacking exon 23. (E) Quantification of exon 23 skipping by relative qPCR and expressed as a percentage of total dystrophin transcripts. (F) Quantification of dystrophin (dys) transcripts by relative qPCR. Relative amount of dys transcripts is expressed as a percentage of wt RNAs. The data represent the mean values of four TAs per group ± SEM. Student’s t-test: n.s. non-significant, *P ≤ 0.05, **P ≤ 0.01.