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Authors: ALLEPUZ, Rubén D ARTERO; TROISI, María Beatriz LLAMUSÍ; HERREROS, Estefanía CERRO; COSTA, Juan M FERNÁNDEZ;
Patent
Publisher: Google Patents
Modulación de microRNAs contra la distrofia miotónica tipo 1 y antagonistas de microRNAs para ello. La invención proporciona el uso de inhibidores de microRNAs represores de los genes MBNL1 y/o MBNL2 para la fabricación de un medicamento para el tratamiento de la distrofia miotónica 1. Inhibir dichos microRNAs permite incrementar los niveles endógenos de las correspondientes proteínas MBNL1 y/o MBNL2, paliando con ello síntomas de la enfermedad, especialmente cuando se inhiben represores que se expresan en los principales órganos afectados: músculo esquelético, corazón u órganos del sistema ne
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