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Viltolarsen
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Catalog No. T39455Cas No. 2055732-84-6
Alias NS-065, NS065, NCNP-01, NCNP01
Viltolarsen (NS-065/NCNP-01) is a phosphorodiamidate morpholino antisense oligonucleotide that specifically binds exon 53 of the dystrophin mRNA precursor, restoring the amino acid open-reading frame through exon 53 skipping and resulting in the production of a shortened but functionally relevant dystrophin protein. Viltolarsen provides a mechanistically precise experimental agent for investigating exon-skipping therapies and molecular pathology in Duchenne muscular dystrophy research.
Viltolarsen
Copy Product Info😃Good
Catalog No. T39455Alias NS-065, NS065, NCNP-01, NCNP01Cas No. 2055732-84-6
Viltolarsen (NS-065/NCNP-01) is a phosphorodiamidate morpholino antisense oligonucleotide that specifically binds exon 53 of the dystrophin mRNA precursor, restoring the amino acid open-reading frame through exon 53 skipping and resulting in the production of a shortened but functionally relevant dystrophin protein. Viltolarsen provides a mechanistically precise experimental agent for investigating exon-skipping therapies and molecular pathology in Duchenne muscular dystrophy research.
| Pack Size | Price | USA Warehouse | Global Warehouse | Quantity |
|---|---|---|---|---|
| 1 mg | $252 | 2-4 weeks | 2-4 weeks | |
| 5 mg | $646 | 2-4 weeks | 2-4 weeks | |
| 10 mg | $1,050 | 2-4 weeks | 2-4 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.
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| Description | Viltolarsen (NS-065/NCNP-01) is a phosphorodiamidate morpholino antisense oligonucleotide that specifically binds exon 53 of the dystrophin mRNA precursor, restoring the amino acid open-reading frame through exon 53 skipping and resulting in the production of a shortened but functionally relevant dystrophin protein. Viltolarsen provides a mechanistically precise experimental agent for investigating exon-skipping therapies and molecular pathology in Duchenne muscular dystrophy research. |
| In vitro | In in vitro studies using DMD patient-derived cells, Viltolarsen induced exon 53 skipping with an EC50 of 0.63 μM in cells harboring an exon 45-52 deletion and an EC50 of 2.3 μM in cells with an exon 48-52 deletion [1]. |
| Synonyms | NS-065, NS065, NCNP-01, NCNP01 |
| Cas No. | 2055732-84-6 |
| 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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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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Please see Inhibitor Handling Instructions for more frequently ask questions. Topics include: how to prepare stock solutions, how to store products, and cautions on cell-based assays & animal experiments, etc
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