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Synonyms: ARO-APOC3
Compound class:
Nucleic acid
Comment: Plozasiran (ARO-APOC3) is a small interfering RNA (siRNA) class ligand. The molecule contains modified nucleotides to improve stability, and it is congugated with N-acetylgalactosamine (GalNAc; at the R1 position), which binds to asialoglycoprotein receptors expressed on hepatocyte membranes to promote delivery of the drug to the liver [4]. Once bound, the siRNA is internalised in lysosomes and is subsequently released into the cytosol. Plozasiran blocks synthesis of apolipoprotein C-III (APOC3), as a mechanism to reduce dyslipidemia that is linked to cardiovascular disease risk [1-2,5].
We have been unable to locate a full chemical SMILES for the ligand, or its HELM notation. |
| References |
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1. Akoumianakis I, Zvintzou E, Kypreos K, Filippatos TD. (2021)
ANGPTL3 and Apolipoprotein C-III as Novel Lipid-Lowering Targets. Curr Atheroscler Rep, 23 (5): 20. [PMID:33694000] |
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2. Carugo S, Sirtori CR, Gelpi G, Corsini A, Tokgozoglu L, Ruscica M. (2023)
Updates in Small Interfering RNA for the Treatment of Dyslipidemias. Curr Atheroscler Rep, 25 (11): 805-817. [PMID:37792132] |
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3. Gaudet D, Pall D, Watts GF, Nicholls SJ, Rosenson RS, Modesto K, San Martin J, Hellawell J, Ballantyne CM. (2024)
Plozasiran (ARO-APOC3) for Severe Hypertriglyceridemia: The SHASTA-2 Randomized Clinical Trial. JAMA Cardiol, 9 (7): 620-630. [PMID:38583092] |
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4. 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] |
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5. Tomlinson B, Wu QY, Zhong YM, Li YH. (2024)
Advances in Dyslipidaemia Treatments: Focusing on ApoC3 and ANGPTL3 Inhibitors. J Lipid Atheroscler, 13 (1): 2-20. [PMID:38299167] |
