Name | ASO | Target exon | Phase | ||
---|---|---|---|---|---|
Nucleic acid | Size | Route | |||
Eteplirsen | PMO | 25 | IV | 51 | Approved |
Golodirsen | PMO | 25 | IV | 53 | Approved |
Viltolarsen | PMO | 21 | IV | 53 | Approved |
Casimersen | PMO | 25 | IV | 45 | Approval stage |
Renadirsen | ENA/2'RNA | 18 | SC | 45 | I/II |
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 |
Disease | Gene | Exon | Phase | Author, Year |
---|---|---|---|---|
LGMD2B | DYSF | 32 | P | Barthelemy et al. 2015 (26) |
Laminopathy | LMNA | 5 | P | Scharner et al. 2015 (27) |
Joubert syndrome | CEP290 | 41 | P | Molinari et al. 2019 (31) |
Alport syndrome | COL4A5 | 21 | P | Yamamura et al. 2020 (29) |
Dystrophic epidermolysis bullosa | COL7A1 | 73 | P | Bornert et al. 2020 (30) |
CADASIL | NOTCH3 | 9 | P | Gravesteijn et al. 2020 (32) |
Mucolipidosis II | GNPTAB | 19 | P | Matos et al. 2020 (33) |
Myotonic dystrophy type1 | DMPK | 15 | P | Stepniak et al. 2020 (34) |
Disease | Gene | Phase | Author, Year |
---|---|---|---|
Usher syndrome type III | CLRN1 | P | Panagiotopoulos et al. 2020 (41) |
LGMD2B | DYSF | P | Dominov et al. 2020 (42) |
Erythropoietic protoporphyria | FECH | P | Halloy et al. 2020 (43) |
Stargardt disease | ABCA4 | P | Sangermano et al. 2019 (44) |
Neuronal ceroid lipofuscinosis 7 | CLN7 | H | Kim et al. 2019 (45) |
Disease | Gene | Exon | Phase | Author, Year |
---|---|---|---|---|
Diabetes mellitus | MSTN | 2 | P | Eilers et al. 2021 (50) |
DMD | P | Kemaladewi et al. 2011 (47) | ||
Parkinson’s disease | LRRK2 | 41 | P | Korecka et al. 2020 (48) |
Prostate cancer | ERG | 4 | P | Li et al. 2020 (49) |
Muscle fibrosis | ALK5 | 6 | P | Kemaladewi et al. 2014 (51) |
Familial hypercholesterolemia | APOB | 27 | P | Disterer et al. 2013 (52) |
Disease | Gene | Exon | Lost domain | Phase | Author, Year |
---|---|---|---|---|---|
Crohn’s disease | ITGA4 | 27 | transmembrane | P | Aung-Htut 2019 (21) |
Dupuytren’s disease | ALK5 | 2 | ligand binding | P | Karkampouna et al. 2014 (53) |
Muscle fibrosis | P | Kemaladewi et al. 2014 (51) | |||
Hypertrophic scar | P | Raktoe et al. 2020 (54) | |||
Cancer | CD44 | v8 | ligand binding | P | Fukushima et al. 2020 (55) |