Viltolarsen (Synonyms: NS-065/NCNP-01) |
رقم الكتالوجGC63876
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يستهدف Viltolarsen (NS-065 / NCNP-01) ، وهو أليغنوكليوتيد مضاد للفوسفورودياميديت مورفولينو ، تضفير exon 53 في جين ديستروفين
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Cas No.: 2055732-84-6
Sample solution is provided at 25 µL, 10mM.
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Viltolarsen (NS-065/NCNP-01), a phosphorodiamidate morpholino antisense oligonucleotide, targets the splicing of exon 53 in the dystrophin gene. Viltolarsen can be used for the research of the Duchenne muscular dystrophy (DMD)[1].
Duchenne muscular dystrophy (DMD) is one of the most common lethal muscle-wasting disorders affecting young boys caused by mutations in the DMD gene. Exon skipping has emerged as a promising therapy for DMD. Antisense oligonucleotides (AONs) are designed to induce the skipping of exon(s), in order to restore the reading frame, and therefore, allow for dystrophin expression[1].
[1]. Dzierlega K, et al. Optimization of antisense-mediated exon skipping for Duchenne muscular dystrophy. Gene Ther. 2020;27(9):407-416.
| Cas No. | 2055732-84-6 | SDF | |
| المرادفات | NS-065/NCNP-01 | ||
| Formula | M.Wt | ||
| الذوبان | H2O : 100 mg/mL (14.44 mM; Need ultrasonic) | Storage | Store at -20°C |
| General tips | Please select the appropriate solvent to prepare the stock solution according to the
solubility of the product in different solvents; once the solution is prepared, please store it in
separate packages to avoid product failure caused by repeated freezing and thawing.Storage method
and period of the stock solution: When stored at -80°C, please use it within 6 months; when stored
at -20°C, please use it within 1 month. To increase solubility, heat the tube to 37°C and then oscillate in an ultrasonic bath for some time. |
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| Shipping Condition | Evaluation sample solution: shipped with blue ice. All other sizes available: with RT, or with Blue Ice upon request. | ||
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Method for preparing in vivo formulation: Take μL DMSO master liquid, next addμL PEG300, mix and clarify, next addμL Tween 80, mix and clarify, next add μL saline, mix and clarify.
Method for preparing in vivo formulation: Take μL DMSO master liquid, next add μL Corn oil, mix and clarify.
Note: 1. Please make sure the liquid is clear before adding the next solvent.
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Quality Control & SDS
- View current batch:
- Purity: >98.00% Appearance: A solid
- COA (Certificate of Analysis)
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Average Rating: 5 (Based on Reviews and 10 reference(s) in Google Scholar.)