Rugonersen (sodium)
Rugonersen sodium is a locked-nucleic acid (LNA) - modified antisense oligonucleotides (ASOs), and results in reduction of ubiquitin-protein ligase E3A (UBE3A) silencing. Angelman syndrome (AS) sodium is a severe neurodevelopmental disorder caused by the loss of neuronal E3 ligase UBE3A, Rugonersen has been used for AS reasearch[1][2].
Product Specifications
CAS Number
2591588-58-6
Product Name Alternative
RG6091 (sodium) ; RO7248824 (sodium)
UNSPSC
12352208
Target
E1/E2/E3 Enzyme
Type
Oligonucleotides
Related Pathways
Metabolic Enzyme/Protease
Applications
Metabolism-protein/nucleotide metabolism
Field of Research
Others
Assay Protocol
https://www.medchemexpress.com/rugonersen-sodium.html
Purity
97.47
Solubility
10 mM in H2O
Smiles
[Rugonersen(sodium)]
Molecular Formula
C204H235N63Na19O109P19S19
Molecular Weight
6530.3 (free acid)
References & Citations
[1]World Health Organization · 2021: WHO Drug Information.|[2]R Jagasia, et al. Angelman syndrome patient neuron screen identifies a potent and selective clinical ASO targeting UBE3A-ATS with long lasting effect in cynomolgus monkey. bioRxiv, 2022-06-12.
Shipping Conditions
Blue Ice
Storage Conditions
-20°C (Powder, sealed storage, away from moisture)
Scientific Category
Oligonucleotides
Clinical Information
Phase 1
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