Nucleic acid compounds for inhibiting myc gene expression...

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

Category

C - Chemistry, Metallurgy

Subcategory

12

Subsubcategory

N

IPC codes

C12N 15/11 (2006.01) A61K 31/713 (2006.01) C07H 21/00 (2006.01)

Type

Patent

Patent number

CA 2679244

Description

The present disclosure provides meroduplex ribonucleic acid molecules (mdRNA) capable of decreasing or silencing MYC 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 a MYC 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 a MYC gene in a cell or in a subject to treat a MYC-related disease.

La présente invention concerne des molécules d'acide ribonucléique méroduplexe (ARNmd) capable de diminuer ou de rendre silencieuse l'expression du gène MYC. 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, un brin étant complémentaire d'un ARNm MYC. Par ailleurs, le méroduplexe peut posséder au moins une uridine substituée avec 5-méthyluridine, un nucléoside remplacé par un acide nucléique bloqué, ou éventuellement d'autres modifications, et n'importe quelle combinaison de ceux-ci. L'invention concerne aussi des procédés de diminution de l'expression d'un gène MYC dans une cellule ou chez un sujet afin de traiter une maladie liée à MYC.

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