Table 2.
Gene Functional Changes Induced by Exon Skipping.
| Function | Target exon | Gene | Inheritance | Disease |
|---|---|---|---|---|
| Restoration | Normal out-of-frame | DMD | Monogenetic | DMD |
| Mutated in-frame exon | DYSF | Monogenetic | LGMD2B | |
| Mutated out-of-frame exon | SGCG | Monogenetic | LGMD2C | |
| Mutated pseudo exon | CLRN1 | Monogenetic | USH3 | |
| Destruction | Out-of-frame exon | MSTN | Non monogenetic | Diabetes mellitus |
| Modulation | Normal in-frame exon | ITGA4 | Non monogenetic | Crohn’s disease |
DMD: Duchenne muscular dystrophy; LGMD2B: limb girdle muscular dystrophy 2B; LGMD2C: limb girdle muscular dystrophy 2C; USH3: Usher syndrome type III