METHODS AND COMPOSITIONS CAPABLE OF CAUSING POST-TRANSCRIPTIONAL SILENCING OF GENE EXPRESSION IN A SYNERGIC MANNER

The invention relates to compositions intended for post-transcriptional inhibition of gene expression through the combined use of: a modified U1 RNA directed against a pre-selected region in a target pre-mRNA-, and a siRNA-, shRNA and/or miRNA-type gene expression silencing agent, as well as to the...

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Hauptverfasser: MARIA PURIFICACION FORTES ALONSO, XABIER ABAD LLORET
Format: Patent
Sprache:eng ; spa
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Zusammenfassung:The invention relates to compositions intended for post-transcriptional inhibition of gene expression through the combined use of: a modified U1 RNA directed against a pre-selected region in a target pre-mRNA-, and a siRNA-, shRNA and/or miRNA-type gene expression silencing agent, as well as to the use of said combinations for the treatment of diseases caused by the unwanted overexpression of a protein. La invención se relaciona con composiciones para la inhibición post-transcripcional de la expresión génica mediante el uso combinado de un ARN U1 modificado dirigido contra una región preseleccionada en un pre-ARNm diana y de un agente silenciador de la expresión génica del tipo siRNA, shRNA y/o miRNA, así como al uso de dichas combinaciones para el tratamiento de enfermedades causadas por la sobre-expresión indeseadas de una proteína.