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Golodirsen
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Catalog No. T38860Cas No. 1422959-91-8
Alias SRP-4053, Golodirsen
Golodirsen (SRP-4053) is a phosphorodiamidate morpholino oligomer (PMO) designed to selectively bind to exon 53 of dystrophin pre-mRNA, used in the investigation of Duchenne muscular dystrophy (DMD).
Golodirsen
😃Good
Catalog No. T38860Alias SRP-4053, GolodirsenCas No. 1422959-91-8
Golodirsen (SRP-4053) is a phosphorodiamidate morpholino oligomer (PMO) designed to selectively bind to exon 53 of dystrophin pre-mRNA, used in the investigation of Duchenne muscular dystrophy (DMD).
| Pack Size | Price | Availability | Quantity |
|---|---|---|---|
| 25 mg | $1,520 | Backorder |
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Product Introduction
Bioactivity
Chemical Properties
| Description | Golodirsen (SRP-4053) is a phosphorodiamidate morpholino oligomer (PMO) designed to selectively bind to exon 53 of dystrophin pre-mRNA, used 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]. |
| Alias | SRP-4053, Golodirsen |
| Cas No. | 1422959-91-8 |
Storage & Solubility Information
| Storage | Powder: -20°C for 3 years | In solvent: -80°C for 1 year | Shipping with blue ice. |
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Please enter your animal experiment information in the following box and click Calculate to obtain the mother liquor preparation method and in vivo formula preparation method:
For example, your dosage is 10 mg/kg Each animal weighs 20 g, and the dosage volume is 100 μL . A total of 10 animals were administered, and the formula you used is 5% 
DMSO+30% PEG300+5% Tween 80+60% ddH2O. So your working solution concentration is 2 mg/mL。Mother liquor preparation method: 2 mg of drug dissolved in 50 μL DMSO (mother liquor concentration of 40 mg/mL), if you need to configure a concentration that exceeds the solubility of the product, please contact us first.
(mother liquor concentration of 40 mg/mL), if you need to configure a concentration that exceeds the solubility of the product, please contact us first.Preparation method for in vivo formula: Take 50 μL DMSO main solution, add 300 μLPEG300 mix well and clarify, then add 50 more μL Tween 80, mix well and clarify, then add 600 more μLddH2O mix well and clarify
main solution, add 300 μLPEG300 mix well and clarify, then add 50 more μL Tween 80, mix well and clarify, then add 600 more μLddH2O mix well and clarifyFor Reference Only. Please develop an appropriate dissolution method based on your laboratory animals and route of administration.
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