2. Metabolic Enzyme/Protease
  3. E1/E2/E3 Enzyme
  4. Rugonersen

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.

The free form of the compound is prone to instability, it is advisable to consider the stable salt form (Rugonersen sodium) that retains the same biological activity.

For research use only. We do not sell to patients.

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)

Rugonersen Chemical Structure

CAS No. : 2591587-57-2

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Rugonersen Related Antibodies

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Description

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[1][2].

IC50 & Target

ubiquitin-protein ligase E3A (UBE3A)[1]
In Vitro

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.

Rugonersen Related Antibodies
In Vivo

Rugonersen (RO7248824) (24 mg/monkey; i.t.; for 8-85 d) is well tolerated without adverse in-life effects or tissue pathology and produced a robust, long lasting (up to 3 months) paternal reactivation of UBE3A mRNA/protein across key monkey brain regions[1].
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.

Animal Model: Male cynomolgus monkey[1]
Dosage: 24 mg per monkey
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
Result: Resulted a long duration of action on paternal UBE3A reactivation in NHP brains after IT delivery.
Animal Model: WT and AS Ube3a m-/p+ mice adult mice (10-12 weeks old)[1]
Dosage: 150 μg per mice
Administration: Intracerebroventricular injection; single dose; harvested at 2 weeks post injection
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.
Clinical Trial
Molecular Weight

6530.30

CAS No.
Appearance

Solid

Color

White to off-white

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)
SMILES

[Rugonersen]
Shipping

Room temperature in continental US; may vary elsewhere.
Storage

-20°C, sealed storage, away from moisture

*In solvent : -80°C, 6 months; -20°C, 1 month (sealed storage, away from moisture)

Solvent & Solubility
In Vitro: 

H2O : ≥ 100 mg/mL (15.31 mM)

*"≥" means soluble, but saturation unknown.

Preparing
Stock Solutions
Concentration Solvent Mass 1 mg 5 mg 10 mg
1 mM 0.1531 mL 0.7657 mL 1.5313 mL
5 mM 0.0306 mL 0.1531 mL 0.3063 mL
View the 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.

* 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.

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  • Dilution Calculator

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In Vivo Dissolution Calculator
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This product has good water solubility, please refer to the measured solubility data in water/PBS/Saline for details.
The concentration of the stock solution you require exceeds the measured solubility. The following solution is for reference only.If necessary, please contact MedChemExpress (MCE).
Purity & Documentation
References

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.

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Rugonersen Related Classifications

Help & FAQs
  • Do most proteins show cross-species activity?

    Species cross-reactivity must be investigated individually for each product. Many human cytokines will produce a nice response in mouse cell lines, and many mouse proteins will show activity on human cells. Other proteins may have a lower specific activity when used in the opposite species.

Keywords:

Rugonersen2591587-57-2RG6091 RO7248824RG 6091RG-6091RO7248824RO 7248824RO-7248824E1/E2/E3 EnzymeE1 activating enzymeE2 conjugating enzymeE3 ligating enzymeUbiquitin activating enzymeUbiquitin conjugating enzymeUbiquitin ligaseAngelman syndromeUBE3A-ATSlong lasting effectcynomolgus monkey AS human pluripotent stem cell neuronsInhibitorinhibitorinhibit

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