Figure 1. Transfection of cultured muscle cells with a fluorescently labeled AON. Differentiated muscle cells are multinucleated, while the undifferentiated myoblasts have a single nucleus. Most of the nuclei are brightly stained, while the cytoplasm is faintly stained, or not stained at all.
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Figure 2. Example of exon skipping analysis. (a) Ethidium bromide‐stained agarose gel. Ten different AONs targeting a single exon were compared. NT = nontreated control, C = negative water control, M = size marker; (b) Sequence analysis to confirm proper exon skipping; (c) Lab‐on‐a‐ chip analysis. The Agilent Lab‐Chip system allows the quantification of full‐length and skip fragments, and corrects for length. Using this system, exon skipping levels can be calculated and individual AONs compared; (d) Comparison of exon skipping levels. AONs 8, 9, and 10 outperform the other AONs. Before making a decision it is best to compare efficiency at different concentrations, and confirm the reproducibility.
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