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Golodirsen

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Catalog No. T38860Cas No. 1422959-91-8
Alias SRP-4053, SRP4053

Golodirsen (SRP-4053) is an antisense oligonucleotide of the phosphorodiamidate morpholino oligomer (PMO) class that restores the dystrophin gene reading frame in Duchenne muscular dystrophy by modulating pre-mRNA splicing to induce skipping of exon 53, thereby enabling partial dystrophin expression and restoring anti-myostatin protein levels, which supports its application as a molecular tool for studying exon-skipping strategies and disease-modifying approaches in DMD research.

Golodirsen

Golodirsen

😃Good
Catalog No. T38860Alias SRP-4053, SRP4053Cas No. 1422959-91-8
Golodirsen (SRP-4053) is an antisense oligonucleotide of the phosphorodiamidate morpholino oligomer (PMO) class that restores the dystrophin gene reading frame in Duchenne muscular dystrophy by modulating pre-mRNA splicing to induce skipping of exon 53, thereby enabling partial dystrophin expression and restoring anti-myostatin protein levels, which supports its application as a molecular tool for studying exon-skipping strategies and disease-modifying approaches in DMD research.
Pack SizePriceUSA WarehouseGlobal WarehouseQuantity
1 mg$3912-4 weeks2-4 weeks
5 mg$9782-4 weeks2-4 weeks
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In Stock Estimated shipping dateUSA Warehouse[1-2 days] Global Warehouse[5-7 days]
With extensive experience in compound synthesis, we can provide rapid custom synthesis services for this product according to your research needs.All TargetMol products are for research purposes only and cannot be used for human consumption. We do not provide products or services to individuals. Please comply with the intended use and do not use TargetMol products for any other purpose.
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Product Introduction

Bioactivity
Description
Golodirsen (SRP-4053) is an antisense oligonucleotide of the phosphorodiamidate morpholino oligomer (PMO) class that restores the dystrophin gene reading frame in Duchenne muscular dystrophy by modulating pre-mRNA splicing to induce skipping of exon 53, thereby enabling partial dystrophin expression and restoring anti-myostatin protein levels, which supports its application as a molecular tool for studying exon-skipping strategies and disease-modifying approaches in DMD research.
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].
SynonymsSRP-4053, SRP4053
Chemical Properties
Cas No.1422959-91-8
SequenceRNA, [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]
Storage & Solubility Information
Storagekeep away from moisture | In solvent: -80°C for 1 year | Shipping with blue ice/Shipping at ambient temperature.
Solubility Information
H2O: 80 mg/mL, Sonication is recommended.

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Keywords

SRP 4053Golodirsen
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