Oligonucleotide-Based Therapy for FTD/ALS Caused by the C9orf72 Repeat Expansion: A Perspective

Figure 3

Strategy for exon 51-skipping in Duchenne muscular dystrophy. Exon 51-skipping by appropriate PMO or 2′OMePS, indicated by a blue line, can restore the reading frame of dystrophin in a DMD patient, who lacks exon 52 in the mRNA of the DMD gene, leading to out-of-frame products. Dot line and Ex indicate introns and exons, respectively.