| Description | Eteplirsen (AVI 4658) is a synthetic antisense oligonucleotide utilized in research for Duchenne muscular dystrophy. |
| In vitro | Eteplirsen acts to promote dystrophin production by restoring the translational reading frame of Duchenne muscular dystrophy (DMD) through specific skipping of exon 51 in defective gene variants[1]. |
| Synonyms | 依特立生;依特普森, AVI 4658 |
| molecular weight | N/A |
| CAS | 1173755-55-9 |
| Storage | Powder: -20°C for 3 years | In solvent: -80°C for 1 year |
| References | 1. Kenji Rowel Q Lim, et al. Eteplirsen in the treatment of Duchenne muscular dystrophy. Drug Des Devel Ther. 2017 Feb 28;11:533-545. |