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Synonyms: AD-51547 | ALN-TTRSC
Compound class:
Nucleic acid
Comment: Revusiran is a double-stranded small interfering ribonucleic acid (siRNA) class ligand. It is proposed to reduce hepatic transthyretin (TTR) production, as a mechanism to treat hereditary transthyretin-mediated (hATTR) amyloidosis-related cardiomyopathy.
Nucleic acid bases are chemically modified to improve stability and GalNAc-conjugation is used to promote delivery of the drug to the liver [1]- this is annotated as 'R1' in the sequence provided here. We have been unable to locate a full chemical SMILES for the ligand, or its HELM notation. |
| References |
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1. Holm A, Løvendorf MB, Kauppinen S. (2021)
Development of siRNA Therapeutics for the Treatment of Liver Diseases. Methods Mol Biol, 2282: 57-75. [PMID:33928570] |
