Rugonersen
Based on 1 Customer Validation
Rugonersen (RG6091; RO7248824) is a locked-nucleic acid (LNA)- modified antisense oligonucleotides (ASOs), and results in reduction of ubiquitin-protein ligase E3A (UBE3A) silencing. Angelman syndrome (AS) is a severe neurodevelopmental disorder caused by the loss of neuronal E3 ligase UBE3A, Rugonersen has been used for AS reasearch.
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- Pureza: 98.50%
- No. CAS: 2591587-57-2
- Peso molecular:6530.30
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Almacenamiento:
-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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Actividad biológica
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Chemical Information
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Solvente y solubilidad
- Pureza y Documentación
- Referencias
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Help & FAQs
Actividad biológica
ubiquitin-protein ligase E3A (UBE3A)[1]
RO7248824 (0-10 μM) show a nanomolar potency against UBE3A-ATS (EC50=26.3 nM), UBE3A mRNA upregulation (EC50=15.4 nM) and UBE3A protein upregulation (EC50=24.8 nM) in Angelman syndrome (AS) neurons[1].
MedChemExpress (MCE) has not independently confirmed the accuracy of these methods. They are for reference only.
Male cynomolgus monkeys[1] Rugonersen (150 μg; i.c.v.; single dose) selectively and potently reduces UBE3A-ATS, while concomitantly upregulating the UBE3A mRNA and protein[1].
MedChemExpress (MCE) has not independently confirmed the accuracy of these methods. They are for reference only.
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Animal Model:Male cynomolgus monkey[1]
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Dosage:24 mg per monkey
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Administration:Intrathecal injection; single dose or twice dose with 2 weeks apart; sacrificed at 8, 15, 29, 57, and 85 days after the last dose
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Result:Resulted a long duration of action on paternal UBE3A reactivation in NHP brains after IT delivery.
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Animal Model:WT and AS Ube3a m-/p+ mice adult mice (10-12 weeks old)[1]
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Dosage:150 μg per mice
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Administration:Intracerebroventricular injection; single dose; harvested at 2 weeks post injection
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Result:Revealed a steep relationship between UBE3A-ATS knock-down and UBE3A mRNA/protein upregulation, whereby an almost 90% downregulation was needed to achieve a 50% upregulation, respectively.
Chemical Information
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No. CAS 2591587-57-2
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Appearance Solid
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Peso molecular 6530.30
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Color White to off-white
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SMILES
[Rugonersen]
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Synonyms
RG6091; RO7248824
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Sequence
DNA, d(P-thio)((2′-O,4′-C-methylene)m5rU-(2′-O,4′-C-methylene)m5rU-(2′-O,4′-C-methylene)rA-C-(2′-O,4′-C-methylene)rA-C-T-T-A-A-T-T-A-T-A-C-T-(2′-O,4′-C-methylene)m5rU-(2′-O,4′-C-methylene)m5rC-(2′-O,4′-C-methylene)m5rC)
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Envío
Room temperature in continental US; may vary elsewhere.
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Almacenamiento
-20°C, sealed storage, away from moisture
* In solvent : -80°C, 6 months; -20°C, 1 month (sealed storage, away from moisture)
Solvente y solubilidad
H2O : ≥ 100 mg/mL (15.31 mM)
* "≥" means soluble, but saturation unknown.
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.
Concentration (start) × Volume (start) = Concentration (final) × Volume (final)
Pureza y Documentación
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Ficha de datos (274 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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Instrucciones de manejo (2242 KB)
Referencias
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 | 0.1531 mL | 0.7657 mL | 1.5313 mL | 3.8283 mL |
| 5 mM | 0.0306 mL | 0.1531 mL | 0.3063 mL | 0.7657 mL | |
| 10 mM | 0.0153 mL | 0.0766 mL | 0.1531 mL | 0.3828 mL | |
| 15 mM | 0.0102 mL | 0.0510 mL | 0.1021 mL | 0.2552 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.
Rugonersen Related Classifications
HY-148130 Related Classifications
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