| Entry |
|
| Name |
|
| Type |
Loss of function
|
| Gene |
|
| Organism |
hsa_var Human gene variants (Homo sapiens)
|
| Variation |
mutations amenable to exon 51 skipping |
| Variation |
mutations amenable to exon 53 skipping |
| Variation |
mutations amenable to exon 45 skipping |
| Network |
nt06539 Cytoskeleton in muscle cells |
| Disease |
| H01963 | Duchenne muscular dystrophy |
|
| Drug target |
| Delandistrogene moxeparvovec: | D12633<US> |
| Zeleciment rostudirsen: | D13292 |
|
| Reference |
|
| Authors |
Lim KR, Maruyama R, Yokota T |
| Title |
Eteplirsen in the treatment of Duchenne muscular dystrophy. |
| Journal |
|
| Reference |
|
| Authors |
Rodrigues M, Yokota T |
| Title |
An Overview of Recent Advances and Clinical Applications of Exon Skipping and Splice Modulation for Muscular Dystrophy and Various Genetic Diseases. |
| Journal |
|
| LinkDB |
|