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.