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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