Figure 2

Mutation in dystrophin gene of mdx^5cv mice and correcting ODNs. Mdx^5cv mice have a single point mutation (A to T) in the dystrophin gene, which is both bolded and underlined in the gene sequence above. This single base mutation will create a donor splice site within exon 10 and cause a 53 base portion of this exon to be spliced out of the mature transcript as depicted by the dashed line in the diagram. A frameshift results in a stop codon in exon 11 (*) and will ultimately result in the production of a truncated dystrophin protein. Because the natural donor site is still present in the transcript there is a possibility that some full-length message, containing exon 10 is made and this is shown as the solid line. The ODNs, mdx47T and mdx47NT, shown above, were designed to correct the substitution mutation and the target base is underlined.