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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. |
Nucleic Acid Sequence  |
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(3’-5’)U-G-G-G-A-U-U-U-C-A-U-G-U-A-A-C-C-A-A-G-A-R1 (5’-3’)Cs-Us-A-C-C-C-U-A-A-A-G-U-A-C-A-U-U-G-G-U-U-C-U |
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