Drisapersen
Based on 1 Customer Validation
Drisapersen (Kyndrisa) is a 2'-O-methyl phosphorothioate RNA antisense oligonucleotide that induces exon 51 skipping. Drisapersen induces skipping of exon 51 during Dystrophin pre-mRNA splicing, allowing the synthesis of partially functional Dystrophin. Drisapersen can be used in research related to Duchenne muscular dystrophy.
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: 97.92%
- CAS 番号: 1251830-50-8
- 分子式: C211H275N76O119P19S19
- 分子量:6977.62
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保管条件:
-20°C, sealed storage, away from moisture
* In solvent : -80°C, 6 months; -20°C, 1 month (sealed storage, 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
DNA/RNA Synthesis アイソフォーム固有の製品をすべて表示
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生物活性
Drisapersen induces exon 51 skipping during Dystrophin pre-mRNA splicing, leading to the synthesis of Dystrophin, and is used for research related to Duchenne muscular dystrophy[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 1251830-50-8
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性状 Solid
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分子量 6977.62
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分子式 C211H275N76O119P19S19
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Color White to off-white
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SMILES
CO[C@@H]1[C@H](O)[C@@H](COP(S)(O[C@@H]2[C@@H](COP(S)(O[C@@H]3[C@@H](COP(S)(O[C@@H]4[C@@H](COP(S)(O[C@@H]5[C@@H](COP(S)(O[C@@H]6[C@@H](COP(S)(O[C@@H]7[C@@H](COP(S)(O[C@@H]8[C@@H](COP(S)(O[C@@H]9[C@@H](COP(S)(O[C@@H]%10[C@@H](COP(S)(O[C@@H]%11[C@@H](COP(S)(O[C@@H]%12[C@@H](COP(S)(O[C@@H]%13[C@@H](COP(S)(O[C@@H]%14[C@@H](COP(S)(O[C@@H]%15[C@@H](COP(S)(O[C@@H]%16[C@@H](COP(S)(O[C@@H]%17[C@@H](COP(S)(O[C@@H]%18[C@@H](COP(S)(O[C@@H]%19[C@@H](COP(S)(O[C@@H]%20[C@@H](CO)O[C@H]([C@@H]%20OC)N%21C=CC(NC%21=O)=O)=O)O[C@H]([C@@H]%19OC)N%22C=CC(N)=NC%22=O)=O)O[C@H]([C@@H]%18OC)n%23c%24ncnc(N)c%24nc%23)=O)O[C@H]([C@@H]%17OC)n%25c%26ncnc(N)c%26nc%25)=O)O[C@H]([C@@H]%16OC)n%27cnc%28c%27N=C(N)NC%28=O)=O)O[C@H]([C@@H]%15OC)n%29cnc%30c%29N=C(N)NC%30=O)=O)O[C@H]([C@@H]%14OC)n%31c%32ncnc(N)c%32nc%31)=O)O[C@H]([C@@H]%13OC)n%33c%34ncnc(N)c%34nc%33)=O)O[C@H]([C@@H]%12OC)n%35cnc%36c%35N=C(N)NC%36=O)=O)O[C@H]([C@@H]%11OC)n%37c%38ncnc(N)c%38nc%37)=O)O[C@H]([C@@H]%10OC)N%39C=CC(NC%39=O)=O)=O)O[C@H]([C@@H]9OC)n%40cnc%41c%40N=C(N)NC%41=O)=O)O[C@H]([C@@H]8OC)n%42cnc%43c%42N=C(N)NC%43=O)=O)O[C@H]([C@@H]7OC)N%44C=CC(N)=NC%44=O)=O)O[C@H]([C@@H]6OC)n%45c%46ncnc(N)c%46nc%45)=O)O[C@H]([C@@H]5OC)N%47C=CC(NC%47=O)=O)=O)O[C@H]([C@@H]4OC)N%48C=CC(NC%48=O)=O)=O)O[C@H]([C@@H]3OC)N%49C=CC(NC%49=O)=O)=O)O[C@H]([C@@H]2OC)N%50C=CC(N)=NC%50=O)=O)O[C@H]1N%51C=CC(NC%51=O)=O
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別名
Kyndrisa; GSK2402968A; PRO051
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配列
RNA, (P-thio)(Um-Cm-Am-Am-Gm-Gm-Am-Am-Gm-Am-Um-Gm-Gm-Cm-Am-Um-Um-Um-Cm-Um)
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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, sealed storage, away from moisture
* In solvent : -80°C, 6 months; -20°C, 1 month (sealed storage, away from moisture)
溶剤 & 溶解度
H2O : 100 mg/mL (14.33 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 (sealed storage, 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 (sealed storage, 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)
純度とドキュメンテーション
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データシート (271 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]. Goemans NM, et al. Long-Term Efficacy, Safety, and Pharmacokinetics of Drisapersen in Duchenne Muscular Dystrophy: Results from an Open-Label Extension Study. PLoS One. 2016;11(9):e0161955. Published 2016 Sep 2. [Content Brief]
[2]. Flanigan KM, et al. Pharmacokinetics and safety of single doses of drisapersen in non-ambulant subjects with Duchenne muscular dystrophy: results of a double-blind randomized clinical trial. Neuromuscul Disord. 2014;24(1):16-24. [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 (sealed storage, 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 | 0.1433 mL | 0.7166 mL | 1.4332 mL | 3.5829 mL |
| 5 mM | 0.0287 mL | 0.1433 mL | 0.2866 mL | 0.7166 mL | |
| 10 mM | 0.0143 mL | 0.0717 mL | 0.1433 mL | 0.3583 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.
Drisapersen 関連分類
HY-145724 関連分類
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