Figure 1.

Antisense-mediated exon skipping rationale for DMD. Patients with Duchenne muscular dystrophy have mutations which disrupt the open-reading frame of the dystrophin pre-mRNA. In this example, exon 50 is deleted, creating an out-of-frame mRNA and leading to the synthesis of a truncated non-functional or unstable dystrophin (left panel). An antisense oligonucleotide directed against exon 51 can induce effective skipping of exon 51 and restore the open reading frame, therefore generating an internally deleted but partly functional dystrophin (right panel).