Fig.1.

AON B efficiently skipped dysferlin exon 32 in human patient cells. (A) Efficiency of exon skipping using AON B was assessed by RT-PCR on cells from patient 2. PCR was used to amplify the region between exons 31 and 33 (293 bp with or 215 bp without exon 32). RT::negative control of reverse transcription. Mock: negative control of transfection. Scrambled: nonspecific AON sequence. AON B: single transfection of AON B. CTL: positive control, cells were transfected with dysferlin full-length (native dysferlin). MM: molecular marker. (B) Western Blot experiment performed with proteins from (AON B or scrambled) treated patient cells or control cells. Hamlet was used to detect dysferlin and GAPDH was used for normalization.