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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
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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 Size | Price | USA Warehouse | Global Warehouse | Quantity |
|---|---|---|---|---|
| 1 mg | $391 | 2-4 weeks | 2-4 weeks | |
| 5 mg | $978 | 2-4 weeks | 2-4 weeks |
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In Stock Estimated shipping dateUSA Warehouse[1-2 days] Global Warehouse[5-7 days]
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| 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]. |
| Synonyms | SRP-4053, SRP4053 |
| Cas No. | 1422959-91-8 |
| 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] |
| Storage | keep away from moisture | Powder: -20°C for 3 years | 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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Please enter your animal experiment information in the following box and click Calculate to obtain the stock solution preparation method and in vivo formula preparation method:
For example, if the intended dosage is 10 mg/kg for animals weighing 20 g , with a dosing volume of 100 μL per animal, and a total of 10 animals are to be administered, using a formulation of 
10% DMSO+ 40% PEG300+ 5% Tween 80+ 45% Saline/PBS/ddH2O , the resulting working solution concentration would be 2 mg/mL.Stock Solution Preparation:
Dissolve 2 mg of the compound in 100 μL DMSO to obtain a stock solution at a concentration of 20 mg/mL . If the required concentration exceeds the compound's known solubility, please contact us for technical support before proceeding.
Preparation of the In Vivo Formulation:
1) Add 100 μL of the DMSO stock solution to 400 μL PEG300 and mix thoroughly until the solution becomes clear.
2) Add 50 μL Tween 80 and mix well until fully clarified.
3) Add 450 μL Saline,PBS or ddH2O and mix thoroughly until a homogeneous solution is obtained.
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