1422959-91-8
1422959-91-8 structure
- Name: Golodirsen
- Chemical Name: Golodirsen
- CAS Number: 1422959-91-8
- Molecular Formula:
- Molecular Weight:
- Catalog: Research Areas Others
- Create Date: 2022-02-12 08:30:59
- Modify Date: 2026-07-16 15:37:10
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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].
| Name | Golodirsen |
|---|
| Description | 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]. |
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| Related Catalog | |
| In Vitro | 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]. |
| References |
[1]. Heo YA. Golodirsen: First Approval. Drugs. 2020 Feb;80(3):329-333. |
| No Any Chemical & Physical Properties |