Compositions and methods for inhibiting expression of the...

A - Human Necessities – 61 – K

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A61K 31/713 (2006.01) A61K 48/00 (2006.01) C07H 21/02 (2006.01) C12N 15/11 (2006.01) C12N 15/57 (2006.01) C12N 15/63 (2006.01)

Patent

CA 2651839

The invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of the PCSK9 gene (PCSK9 gene), comprising an antisense strand having a nucleotide sequence which is less that 30 nucleotides in length, generally 19-25 nucleotides in length, and which is substantially complementary to at least a part of the PCSK9 gene. The invention also relates to a pharmaceutical composition comprising the dsRNA together with a pharmaceutically acceptable carrier; methods for treating diseases caused by PCSK9 gene expression and the expression of the PCSK9 gene using the pharmaceutical composition; and

La présente invention concerne un acide ribonucléique bicaténaire (ARNds) permettant l'inhibition de l'expression du gène PCSK9, comportant un brin antisens comprenant une séquence nucléotidique dont la longueur est inférieure à 30 nucléotides, généralement de 19 à 25 nucléotides, et qui est sensiblement complémentaire à au moins une partie du gène PCSK9. L'invention concerne également une composition pharmaceutique comportant l'ARNds conjointement avec un support pharmaceutiquement acceptable, des procédés de traitement de maladies provoquées par l'expression du gène PCSK9, des procédés d'utilisation des compositions pour inhiber l'expression du gène PCSK9 mettant en oeuvre la composition pharmaceutique; et des procédés d'utilisation des compositions pharmaceutiques pour traiter des maladies pouvant être modulées par la régulation à la baisse de l'expression du gène PCSK9.

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