Double-stranded lipid-modified rna having high rna...

Details Double-stranded lipid-modified rna having high rna... Double-stranded lipid-modified rna having high rna...

C - Chemistry, Metallurgy

12

N

C12N 15/09 (2006.01) A61K 31/7088 (2006.01) A61K 48/00 (2006.01)

Patent

CA 2719963

An object of the present invention is to provide a novel double-stranded RNA that has high resistance to nuclease and cellular uptake efficiency and that can produce an excellent RNA interference effect. The present invention provides a double-stranded lipid-modified RNA comprising a sense strand having a nucleotide sequence complementary to a target sequence in a target gene, and an antisense strand having a nucleotide sequence complementary to the sense strand, the double-stranded RNA being capable of suppressing the expression of the target gene, and the sense strand having a double-stranded lipid bound directly or via a linker to at least one of the first to sixth nucleotides from the 5' end.

L'invention porte sur un nouvel ARN double brin qui présente une résistance élevée aux nucléases, qui peut être introduit dans un cytoplasme à une vitesse élevée et qui peut présenter un excellent effet d'interférence ARN. Plus précisément, l'invention porte sur un ARN modifié par lipide double brin qui est composé d'un ARN de brin sens comportant une séquence nucléotidique complémentaire d'une séquence cible contenue dans un gène cible et un ARN de brin antisens comportant une séquence nucléotidique complémentaire de l'ARN de brin sens et qui peut inhiber l'expression du gène cible, un lipide double brin étant lié à au moins un nucléotide choisi parmi les 6premiers nucléotides à partir de l'extrémité terminale 5' de l'ARN de brin sens directement ou par l'intermédiaire d'un lieur.

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