Examples of therapeutic exon skipping in DMD patient-derived muscle cells.

 

DMD del.ex 45-54:                skip exon 44

DMD del. ex45:                      skip exon 46

DMD point mutation ex49:     skip exon 49

DMD del. ex48-50:                skip exon 51

DMD del. 52:                          skip exon 51 or 53

DMD point mutation ex 43:    double skip exon 43+44

DMD del. ex46-50:                double skip exon 45+51