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Eteplirsen
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Catalog No. T38554Cas No. 1173755-55-9
Alias AVI-4658, AVI4658, AVI 4658
Eteplirsen (brand name Exondys 51) is an antisense oligonucleotide therapy designed to induce exon 51 skipping in the dystrophin gene, enabling partial restoration of dystrophin protein expression in patients with Duchenne muscular dystrophy carrying specific exon 51 mutations, Eteplirsen is extensively applied in genetic disease research, RNA-targeted therapy development, and translational studies of muscular dystrophy.
Eteplirsen
😃Good
Catalog No. T38554Alias AVI-4658, AVI4658, AVI 4658Cas No. 1173755-55-9
Eteplirsen (brand name Exondys 51) is an antisense oligonucleotide therapy designed to induce exon 51 skipping in the dystrophin gene, enabling partial restoration of dystrophin protein expression in patients with Duchenne muscular dystrophy carrying specific exon 51 mutations, Eteplirsen is extensively applied in genetic disease research, RNA-targeted therapy development, and translational studies of muscular dystrophy.
| Pack Size | Price | USA Warehouse | Global Warehouse | Quantity |
|---|---|---|---|---|
| 1 mg | $143 | 2-4 weeks | 2-4 weeks | |
| 5 mg | $333 | 2-4 weeks | 2-4 weeks | |
| 10 mg | $570 | 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 | Eteplirsen (brand name Exondys 51) is an antisense oligonucleotide therapy designed to induce exon 51 skipping in the dystrophin gene, enabling partial restoration of dystrophin protein expression in patients with Duchenne muscular dystrophy carrying specific exon 51 mutations, Eteplirsen is extensively applied in genetic disease research, RNA-targeted therapy development, and translational studies of muscular dystrophy. |
| In vitro | Eteplirsen functions by enhancing dystrophin synthesis, thereby treating Duchenne muscular dystrophy (DMD). It achieves this by selectively skipping exon 51 in mutated gene variants, which restores the translational reading frame necessary for proper protein production[1]. |
| Synonyms | AVI-4658, AVI4658, AVI 4658 |
| Cas No. | 1173755-55-9 |
| Sequence | RNA, [P-deoxy-P-(dimethylamino)](2',3'-dideoxy-2',3'-imino-2',3'-seco)(2'a→5')(C-m5U-C-C-A-A-C-A-m5U-C-A-A-G-G-A-A-G-A-m5U-G-G-C-A-m5U-m5U-m5U-C-m5U-A-G), 5'-[P-[4-[[2-[2-(2-hydroxyethoxy)ethoxy]ethoxy]carbonyl]-1-piperazinyl]-N,N-dimethylphosphonamidate] |
| Storage | keep away from moisture | In solvent: -80°C for 1 year | Shipping with blue ice/Shipping at ambient temperature. |
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In Vivo Formulation Calculator (Clear solution)
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.
This example is provided solely to demonstrate the use of the In Vivo Formulation Calculator and does not constitute a recommended formulation for any specific compound. Please select an appropriate dissolution and formulation strategy based on your experimental model and route of administration.
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