1. Multiple-exon skipping.
Multiple-exon skipping. The uppermost diagram is the intron/exon structure of the dystrophin gene. Blue boxes denote exons. The top box shows the golden retriever muscular dystrophy dog (GRMD) mutation and exon skipping for GRMD. A point mutation in intron 6 alters normal splicing, and the resulting transcript (gray) is out-of-frame. Skipping exons 6, 7 and 8 yields an in-frame transcript. The bottom box shows the mdx52 mutation and exon skipping in mdx52. Deletion of exon 52 disrupts the reading frame and results in a premature stop. Removing exons 45 to 55 from the mutated transcript generates an in-frame transcript.
Joe W. McGreevy et al. Dis. Model. Mech. 2015;8:
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