Nucleic acid compounds for inhibiting akt gene expression...

Details Nucleic acid compounds for inhibiting akt gene expression... Nucleic acid compounds for inhibiting akt gene expression...

C - Chemistry, Metallurgy

12

N

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

Patent

CA 2679387

The present disclosure provides meroduplex ribonucleic acid molecules (mdRNA) capable of decreasing or silencing AKT 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 an AKT mRNA. In addition, the meroduplex may have at least one uridine substituted with a 5-methyluridine, a nucleoside replaced with a locked nucleic acid, or optionally other modifications, and any combination thereof. Also provided are methods of decreasing expression of an AKT gene in a cell or in a subject to treat an AKT -related disease.

La présente invention concerne des molécules d'acide ribonucléique méroduplex (ARNmd) qui peuvent diminuer ou étouffer l'expression de gène AKT. 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 ARNm AKT. De plus, le méroduplex peut avoir au moins une uridine substituée par une 5-méthyluridine, un nucléoside remplacé par un acide nucléique bloqué, ou facultativement d'autres modifications, et n'importe quelle combinaison de ceux-ci. L'invention concerne également des procédés de diminution de l'expression d'un gène AKT dans une cellule ou chez un sujet pour traiter une maladie liée à AKT.

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