| Bioactivity | Golodirsen (SRP-4053) is a phosphorodiamidate morpholino oligomer (PMO) that specifically targets exon 53 of dystrophin pre-mRNA. Golodirsen can be used for the research of Duchenne muscular dystrophy (DMD)[1][2]. |
| Invitro | Golodirsen specifically targets exon 53 of dystrophin pre-mRNA, resulting in its exclusion from the final mRNA product. Skipping of exon 53 results in restoration of the mRNA reading frame and leads to the production of a partially internally deleted dystrophin protein with intact C and N-terminal regions[2]. |
| Name | Golodirsen |
| CAS | 1422959-91-8 |
| Molar Mass | 8647.00 |
| Appearance | Solid |
| Transport | Room temperature in continental US; may vary elsewhere. |
| Storage | -20°C, stored under nitrogen, away from moisture *In solvent : -80°C, 6 months; -20°C, 1 month (stored under nitrogen, away from moisture) |
| Reference | [1]. Heo YA. Golodirsen: First Approval. Drugs. 2020 Feb;80(3):329-333. [2]. Scaglioni D, et, al. The administration of antisense oligonucleotide golodirsen reduces pathological regeneration in patients with Duchenne muscular dystrophy. Acta Neuropathol Commun. 2021 Jan 6;9(1):7. |