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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

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 monkeys....

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Detalles Bibliográficos
Autores principales: Tassone, Pierfrancesco, Di Martino, Maria Teresa, Arbitrio, Mariamena, Fiorillo, Lucia, Staropoli, Nicoletta, Ciliberto, Domenico, Cordua, Alessia, Scionti, Francesca, Bertucci, Bernardo, Salvino, Angela, Lopreiato, Mariangela, Thunarf, Fredrik, Cuomo, Onofrio, Zito, Maria Cristina, De Fina, Maria Rosanna, Brescia, Amelia, Gualtieri, Simona, Riillo, Caterina, Manti, Francesco, Caracciolo, Daniele, Barbieri, Vito, Di Paola, Eugenio Donato, Di Francesco, Adele Emanuela, Tagliaferri, Pierosandro
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10294514/
https://www.ncbi.nlm.nih.gov/pubmed/37365583
http://dx.doi.org/10.1186/s13045-023-01468-8