Safety and activity of the first-in-class locked nucleic acid (LNA) miR-221 selective inhibitor in refractory advanced cancer patients: a first-in-human, phase 1, open-label, dose-escalation study

Safety and activity of the first-in-class locked nucleic acid (LNA) miR-221 selective inhibitor in refractory advanced cancer patients: a first-in-human, phase 1, open-label, dose-escalation study

Abstract Background We developed a 13-mer locked nucleic acid (LNA) inhibitor of miR-221 (LNA-i-miR-221) with a full phosphorothioate (PS)-modified backbone. This agent downregulated miR-221, demonstrated anti-tumor activity against human xenografts in mice, and favorable toxicokinetics in rats and...

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Bibliographic Details
Main Authors: Pierfrancesco Tassone, Maria Teresa Di Martino, Mariamena Arbitrio, Lucia Fiorillo, Nicoletta Staropoli, Domenico Ciliberto, Alessia Cordua, Francesca Scionti, Bernardo Bertucci, Angela Salvino, Mariangela Lopreiato, Fredrik Thunarf, Onofrio Cuomo, Maria Cristina Zito, Maria Rosanna De Fina, Amelia Brescia, Simona Gualtieri, Caterina Riillo, Francesco Manti, Daniele Caracciolo, Vito Barbieri, Eugenio Donato Di Paola, Adele Emanuela Di Francesco, Pierosandro Tagliaferri
Format: Article
Language:English
Published: BMC 2023-06-01
Series:Journal of Hematology & Oncology
Subjects:
miRNA therapeutics
microRNA
miRNA
RNA therapeutics
Non-coding RNA therapeutics
miR-221
Online Access:https://doi.org/10.1186/s13045-023-01468-8

Internet

https://doi.org/10.1186/s13045-023-01468-8

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