| Chemical Properties | Back Directory | [form ]
Solid | [color ]
White to off-white | [Sequence]
RNA, [P-deoxy-P-(dimethylamino)](2',3'-dideoxy-2',3'-imino-2',3'-seco)(2'a→5')(G-m5U-m5U-G-C-C-m5U-C-C-G-G-m5U-m5U-C-m5U-G-A-A-GG-m5U-G-m5U-m5U-C), 5'-[P-[4-[[2-[2-(2-hydroxyethoxy)ethoxy]ethoxy]carbonyl]-1-piperazinyl]-N,Ndimethylphosphonamidate] |
| Hazard Information | Back Directory | [Uses]
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]. | [References]
[1] Heo YA. Golodirsen: First Approval. Drugs. 2020 Feb;80(3):329-333. DOI:10.1007/s40265-020-01267-2
[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. DOI:10.1186/s40478-020-01106-1 |
|
|