Fig 1: Antisense morpholino oligonucleotide-based pseudoexon skipping efficacy. (A) Diagram of the pseudoexon insertion caused by c.689+908G>A and the predicted effect of the antisense morpholino oligonucleotide (AON). Inset showing location and sequence of the 25mer AON. (B) Representative image of the RT-PCR product from mutant (Pt16) and wild type (CT) cells, non-transfected (-) and transfected with 10 µM of non-target control (#); or in the presence of different concentrations (0 to 30 µM) of GFM1-specific AON. (C) EFG1 rescue upon treatment with 10 µM of non-target control (#) or GFM1-specific AON.
Fig 2: Aberrant splicing of GFM1 in Pt16. (A) Diagram of GFM1 cDNA with primers (arrows) used to amplify the complete coding region in two overlapping (F1 and F2) fragments. Agarose gel showing the results of RT-PCR amplifications in control (CT) and patient (Pt) fibroblasts. (B) Cloning of F1 and F2 PCR products and Sanger sequencing of regions around the nucleotide variations detected. (C) Distribution of reads. Data represent the percentage of GFM1 transcript reads with exon 16 skipped (stripped bars) and full length (filled bars). Read numbers were 41,758 for Pt16, 13,581 for CT1 and 16,718 for CT2.
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