For research use only. Not for therapeutic Use.
Rugonersen(Cat No.:I042882)is an investigational antisense oligonucleotide designed to inhibit the production of the progranulin protein, which plays a role in neurodegenerative diseases. It specifically targets the GRN gene responsible for producing progranulin. Low levels of progranulin are linked to frontotemporal dementia (FTD) and other neurodegenerative conditions. By increasing the production of functional progranulin, Rugonersen aims to address these disorders at the molecular level, potentially slowing or halting disease progression. It is currently undergoing clinical trials to assess its safety and efficacy in treating these conditions.
CAS Number | 2591587-57-2 |
Purity | ≥95% |
Reference | [1]. 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. [2]. World Health Organization · 2021: WHO Drug Information. |