Universal target sequences for sirna gene silencing

C - Chemistry – Metallurgy – 12 – N

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C12N 15/11 (2006.01) A61K 31/713 (2006.01)

Patent

CA 2562673

The present invention provides methods for designing a sequence for efficient short interference RNA molecules. In particular, the present invention defines a universal target for siRNA derived from the consensus sequence of the polyadenylation signal in conjunction with unique sequences for gene silencing and inhibition of viral replication in a eukaryotic host cell. The present invention further provides methods for the treatment and prevention of diseases and disorders by silencing a gene of a virus, an oncogene, genes encoding transcription factors and many other diseases related genes.

La présente invention se rapporte à des procédés de conception d'une séquence permettant l'obtention de molécules efficaces de petits ARN interférents (siARN). En particulier, la présente invention définit une cible universelle pour le siARN dérivé de la séquence de consensus du signal de polyadénylation en association à des séquences uniques pour le silençage de gènes et l'inhibition de la réplication virale dans une cellule hôte eukaryote. La présente invention se rapporte en outre à des procédés permettant le traitement et la prévention de maladies et de troubles par silençage d'un gène d'un virus, d'un oncogène, de gènes codant des facteurs de transcription et de nombreux autres gènes associés à des maladies.

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