Bioactivity | 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]. |
CAS | 2591588-58-6 |
Formula | C204H235N63Na19O109P19S19 |
Molar Mass | 6948.00 |
Transport | Room temperature in continental US; may vary elsewhere. |
Storage | Please store the product under the recommended conditions in the Certificate of Analysis. |
Reference | [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. |