Nucleic acid compounds for inhibiting erbb family gene...

C - Chemistry – Metallurgy – 12 – N

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C12N 15/11 (2006.01) A61K 31/713 (2006.01) A61P 29/00 (2006.01) A61P 35/00 (2006.01)

Patent

CA 2679757

The present disclosure provides meroduplex ribonucleic acid molecules (mdRNA) capable of decreasing or silencing one or more ERBB family gene expression. An mdRNA of this disclosure comprises at least three strands that combine to form at least two non-overlapping double-stranded regions separated by a nick or gap wherein one strand is complementary to one or more ERBB family mRNA. In addition, the meroduplex may have at least one uridine substituted with a 5-methyluridine and optionally other modifications or combinations thereof. Also provided are methods of decreasing expression of one or more ERBB family gene in a cell or in a subject to treat one or more ERBB family-related disease.

La présente invention concerne des molécules d'acide ribonucléique méroduplex (ARNmd) qui peuvent diminuer ou étouffer l'expression d'un ou plusieurs gènes de la famille ERBB. Un ARNmd de cette invention comprend au moins trois brins qui se combinent pour former au moins deux régions double brin non chevauchantes séparées par une encoche ou un espace dans lequel un brin est complémentaire à un ou plusieurs ARNm de la famille ERBB. De plus, le méroduplex peut avoir au moins une uridine substituée par une 5-méthyluridine et facultativement d'autres modifications ou combinaisons de ceux-ci. L'invention concerne également des procédés de diminution de l'expression d'un ou plusieurs gènes de la famille ERBB dans une cellule ou chez un sujet pour traiter une ou plusieurs maladies liées à la famille ERBB.

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