Fig. 1.

Structure of human dystrophin protein and strategies for targeting the mutant human DMD gene. a Schematic illustration of the human dystrophin protein structure and the reading frame of the DMD gene derived from a DMD patient harboring a deletion of exon 51 and a termination codon in exon 52 (red asterisk). H, Hinge; R, Repeat; E, Exon. b Strategy I: deletion of exons 45–55 (∆45–55) joins exon 44 to exon 56 and restores the reading frame. E: Exon. c Strategy II: deletion of exons 46–54 (∆46–54) joins exon 45 to exon 55 and restores the reading frame. E, exon. d Strategy III: introduction of random indels in exon 50 (INDEL50) has a chance of restoring the reading frame