Powder: -20°C for 3 years | In solvent: -80°C for 1 year
Golodirsen (SRP-4053) is a phosphorodiamidate morpholino oligomer (PMO) designed to selectively bind and target exon 53 of dystrophin pre-mRNA, with applications in the investigation of Duchenne muscular dystrophy (DMD).
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25 mg | Inquiry | $ 1,520.00 |
Description | Golodirsen (SRP-4053) is a phosphorodiamidate morpholino oligomer (PMO) designed to selectively bind and target exon 53 of dystrophin pre-mRNA, with applications in the investigation of Duchenne muscular dystrophy (DMD). |
In vitro | Golodirsen acts selectively on exon 53 of dystrophin pre-mRNA to cause its exclusion from the final mRNA sequence. This exon skipping restores the mRNA reading frame, facilitating the creation of a truncated yet functional dystrophin protein, which retains intact C and N-terminal regions[2]. |
Synonyms | Golodirsen, SRP-4053 |
Molecular Weight | N/A |
CAS No. | 1422959-91-8 |
Powder: -20°C for 3 years | In solvent: -80°C for 1 year
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Golodirsen 1422959-91-8 SRP 4053 SRP-4053 SRP4053 inhibitor inhibit