Viltolarsen
Based on 1 Customer Validation
Viltolarsen (NS-065/NCNP-01) is a phosphorodiamidate morpholino antisense oligonucleotide. Viltolarsen binds to exon 53 of the dystrophin mRNA precursor and restores the amino acid open-reading frame by skipping exon 53, resulting in the production of a shortened dystrophin protein that contains essential functional portions. Viltolarsen has the potential for Duchenne muscular dystrophy (DMD) research.
商品は「研究用試薬」です。人や動物の医療用・臨床診断用・食品用の製品ではありません。
研究用途以外に使用した場合、当社は一切の責任を負いかねます。
- 純度: 95.25%
- CAS 番号: 2055732-84-6
- 分子式: C244H381N113O88P20
- 分子量:6924.82
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保管条件:
-20°C, stored under nitrogen, away from moisture
* In solvent : -80°C, 6 months; -20°C, 1 month (stored under nitrogen, away from moisture)
To place orders, for customer services and technical support, please contact: MedChemExpress USA
Tel: 609-228-6898 E-mail: [email protected] [email protected]
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生物活性
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化学情報
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溶剤 & 溶解度
- 純度とドキュメンテーション
- 参考文献
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Help & FAQs
生物活性
Viltolarsen (NS-065/NCNP-01; 2 days) induces exon 53 skipping in cells from a DMD model with a deletion of exons 45-52 with an EC50 value of 0.63 μM and in cells with a deletion of exons 48-52 with an EC50 value of 2.3 μM[2].
Viltolarsen (10 μM; 3 days) causes the dystrophin protein expression in cells from a DMD model[2].
MedChemExpress (MCE) has not independently confirmed the accuracy of these methods. They are for reference only.
| NCT Number | Sponsor | Condition | Start Date |
Phase
|
|---|---|---|---|---|
| NCT01329991 | Plexxikon| | 2011-05 | PHASE1 |
化学情報
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CAS 番号 2055732-84-6
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性状 Solid
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分子量 6924.82
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分子式 C244H381N113O88P20
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Color White to off-white
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SMILES
OC[C@H]1O[C@@H](n(ccc(N)n2)c2=O)CN(P(N(C)C)(OC[C@H]3O[C@@H](n(ccc(N)n4)c4=O)CN(P(N(C)C)(OC[C@H]5O[C@@H](n(cc(C)c(=O)[nH]6)c6=O)CN(P(N(C)C)(OC[C@H]7O[C@@H](n(ccc(N)n8)c8=O)CN(P(N(C)C)(OC[C@H]9O[C@@H](n(ccc(N)n%10)c%10=O)CN(P(N(C)C)(OC[C@H]%11O[C@@H](n(cnc%12%13)c%12nc(N)[nH]c%13=O)CN(P(N(C)C)(OC[C@H]%14O[C@@H](n(cnc%15%16)c%15nc(N)[nH]c%16=O)CN(P(N(C)C)(OC[C@H]%17O[C@@H](n(cc(C)c(=O)[nH]%18)c%18=O)CN(P(N(C)C)(OC[C@H]%19O[C@@H](n(cc(C)c(=O)[nH]%20)c%20=O)CN(P(N(C)C)(OC[C@@H]%21CN(P(N(C)C)(OC[C@H]%22O[C@@H](n(cc(C)c(=O)[nH]%23)c%23=O)CN(P(N(C)C)(OC[C@H]%24O[C@@H](n(cnc%25%26)c%25nc(N)[nH]c%26=O)CN(P(N(C)C)(OC[C@H]%27O[C@@H](n%28c%29ncnc(N)c%29nc%28)CN(P(N(C)C)(OC[C@H]%30O[C@@H](n%31c%32ncnc(N)c%32nc%31)CN(P(N(C)C)(OC[C@H]%33O[C@@H](n(cnc%34%35)c%34nc(N)[nH]c%35=O)CN(P(N(C)C)(OC[C@H]%36O[C@@H](n(cnc%37%38)c%37nc(N)[nH]c%38=O)CN(P(N(C)C)(OC[C@H]%39O[C@@H](n(cc(C)c(=O)[nH]%40)c%40=O)CN(P(N(C)C)(OC[C@H]%41O[C@@H](n(cnc%42%43)c%42nc(N)[nH]c%43=O)CN(P(N(C)C)(OC[C@H]%44O[C@@H](n(cc(C)c(=O)[nH]%45)c%45=O)CN(P(N(C)C)(OC[C@H]%46O[C@@H](n(cc(C)c(=O)[nH]%47)c%47=O)CN(P(N(C)C)(OC[C@@H]%48CNC[C@H](n(ccc(N)n%49)c%49=O)O%48)=O)C%46)=O)C%44)=O)C%41)=O)C%39)=O)C%36)=O)C%33)=O)C%30)=O)C%27)=O)C%24)=O)C%22)=O)C[C@H](n(ccc(N)n%50)c%50=O)O%21)=O)C%19)=O)C%17)=O)C%14)=O)C%11)=O)C9)=O)C7)=O)C5)=O)C3)=O)C1
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別名
NS-065/NCNP-01
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配列
RNA, [P-deoxy-P-(dimethylamino)](2',3'-dideoxy-2',3'-imino-2',3'-seco)(2'a→5')(C-C-m5U-C-C-G-G-m5U-m5U-C-m5U-G-A-A-G-G-m5U-Gm5U-m5U-C)
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輸送条件
Room temperature in continental US; may vary elsewhere.
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保管条件
-20°C, stored under nitrogen, away from moisture
* In solvent : -80°C, 6 months; -20°C, 1 month (stored under nitrogen, away from moisture)
溶剤 & 溶解度
H2O : 100 mg/mL (14.44 mM; Need ultrasonic)
Please refer to the solubility information to select the appropriate solvent. Once prepared, please aliquot and store the solution to prevent product inactivation from repeated freeze-thaw cycles.
Storage method and period of stock solution: -80°C, 6 months; -20°C, 1 month (stored under nitrogen, away from moisture). When stored at -80°C, please use it within 6 months. When stored at -20°C, please use it within 1 month.
* Note: If you choose water as the stock solution, please dilute it to the working solution, then filter and sterilize it with a 0.22 μm filter before use.
Please refer to the solubility information to select the appropriate solvent. Once prepared, please aliquot and store the solution to prevent product inactivation from repeated freeze-thaw cycles.
Storage method and period of stock solution: -80°C, 6 months; -20°C, 1 month (stored under nitrogen, away from moisture). When stored at -80°C, please use it within 6 months. When stored at -20°C, please use it within 1 month.
* Note: If you choose water as the stock solution, please dilute it to the working solution, then filter and sterilize it with a 0.22 μm filter before use.
濃度 (開始) × 体積 (開始) = 濃度 (終了) × 体積 (終了)
For the following dissolution methods, please prepare the working solution directly:
It is recommended to prepare fresh solutions and use them promptly within a short period of time.
The percentages shown for the solvents indicate their volumetric ratio in the final prepared solution. If precipitation or phase separation occurs during preparation, heat and/or sonication can be used to aid dissolution.
Add each solvent one by one: PBS
Solubility: 50 mg/mL (7.22 mM); Clear solution; Need ultrasonic
純度とドキュメンテーション
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データシート (272 KB)
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SDS (252 KB)
- English - EN (252 KB)
- Français - FR (252 KB)
- Deutsch - DE (252 KB)
- Norwegian - NO (252 KB)
- Español - ES (252 KB)
- Swedish - SV (252 KB)
- Italian - IT (252 KB)
- Korean - KR (252 KB)
- Portuguese - PT (252 KB)
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取扱説明書 (2242 KB)
参考文献
[1]. Sohita Dhillon. Viltolarsen: First Approval. Drugs. 2020 Jul;80(10):1027-1031. [Content Brief]
[2]. Naoki Watanabe, et al. NS-065/NCNP-01: An Antisense Oligonucleotide for Potential Treatment of Exon 53 Skipping in Duchenne Muscular Dystrophy. Mol Ther Nucleic Acids. 2018 Dec 7:13:442-449. [Content Brief]
Complete Stock Solution Preparation Table
Please refer to the solubility information to select the appropriate solvent. Once prepared, please aliquot and store the solution to prevent product inactivation from repeated freeze-thaw cycles.
Storage method and period of stock solution: -80°C, 6 months; -20°C, 1 month (stored under nitrogen, away from moisture). When stored at -80°C, please use it within 6 months. When stored at -20°C, please use it within 1 month.
| Optional Solvent | Concentration Solvent Mass | 1 mg | 5 mg | 10 mg | 25 mg |
|---|---|---|---|---|---|
| H2O | 1 mM | 0.1444 mL | 0.7220 mL | 1.4441 mL | 3.6102 mL |
| 5 mM | 0.0289 mL | 0.1444 mL | 0.2888 mL | 0.7220 mL | |
| 10 mM | 0.0144 mL | 0.0722 mL | 0.1444 mL | 0.3610 mL |
* Note: If you choose water as the stock solution, please dilute it to the working solution, then filter and sterilize it with a 0.22 μm filter before use.
Viltolarsen 関連分類
HY-132586 関連分類
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