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Please use this identifier to cite or link to this item: https://hdl.handle.net/2445/67166

Synthesis and Tau RNA Binding Evaluation of Ametantrone-Containing Ligands

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We describe the synthesis and characterization of ametantrone-containing RNA ligands based on the derivatization of this intercalator with two neamine moieties (Amt-Nea,Nea) or with one azaquinolone heterocycle and one neamine (Amt-Nea,Azq) as well as its combination with guanidinoneamine (Amt-NeaG4). Biophysical studies revealed that guanidinylation of the parent ligand (Amt-Nea) had a positive effect on the binding of the resulting compound for Tau pre-mRNA target as well as on the stabilization upon complexation of some of the mutated RNA sequences associated with the development of tauopathies. Further studies by NMR revealed the existence of a preferred binding site in the stem-loop structure, in which ametantrone intercalates in the characteristic bulged region. Regarding doubly-functionalized ligands, binding affinity and stabilizing ability of Amt-Nea,Nea were similar to those of the guanidinylated ligand, but the two aminoglycoside fragments seem to interfere with its accommodation in a single binding site. However, Amt-Nea,Azq binds at the bulged region in a similar way than Amt-NeaG4. Overall, these results provide new insights on fine-tuning RNA binding properties of ametantrone by single or double derivatization with other RNA recognition motifs, which could help in the future design of new ligands with improved selectivity for disease-causing RNA molecules.

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ARTIGAS SOLÉ, Gerard and MARCHÁN SANCHO, Vicente. Synthesis and Tau RNA Binding Evaluation of Ametantrone-Containing Ligands. Journal of Organic Chemistry. 2015. Vol. 80, num. 4, pags. 2155-2164. ISSN 0022-3263. [consulted: 13 of June of 2026]. Available at: https://hdl.handle.net/2445/67166

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