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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Autori principali:	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
Natura:	Artigo
Lingua:	Inglês
Pubblicazione:	BMC 2023-06-01
Serie:	Journal of Hematology & Oncology
Soggetti:	miRNA therapeutics microRNA miRNA RNA therapeutics Non-coding RNA therapeutics miR-221
Accesso online:	https://doi.org/10.1186/s13045-023-01468-8
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