Methods and compositions capable of causing...

Details Methods and compositions capable of causing... Methods and compositions capable of causing...

C - Chemistry, Metallurgy

12

N

C12N 15/113 (2010.01) A61K 31/713 (2006.01) A61P 31/12 (2006.01) A61P 35/00 (2006.01)

Patent

CA 2713458

The invention relates to compositions for the post-transcriptional inhibition of gene expression by means of the combined use of a modified U1 snRNA targeted at a pre-selected region in a target pre-mRNA and of a gene expression-silencing agent of the siRNA, shRNA and/or miRNA type, as well as the use of said combinations for the treatment of diseases caused by the unwanted over-expression 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.

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