Figure 2.
Mutation pattern of the CXMD/golden retriever muscular dystrophy (GRMD) dog models and exons 6–8 skipping strategy using AOs. (A) A point mutation in the acceptor splice site (ASS) in intron 6 in CXMD dogs leads to exon 7 being skipped from the dystrophic dog mRNA; (B) AOs are designed such that they bind to exons 6 and 8 causing them to be spliced out thereby correcting the reading frame. Exon 9 is spliced spontaneously along with exons 6 and 8.