Lipid-modified double-stranded rna having potent rna...

A - Human Necessities – 61 – K

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A61K 47/48 (2006.01) C12N 15/113 (2010.01) A61K 31/713 (2006.01) A61K 48/00 (2006.01) C07H 21/02 (2006.01)

Patent

CA 2703496

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

L'invention concerne un nouvel ARN double brin qui présente une résistance de la nucléase élevée et une efficacité d'assimilation cellulaire élevée, et qui peut présenter un excellent effet d'interférence ARN. L'invention concerne un ARN double brin modifié par un lipide comprenant un brin sens présentant une séquence de nucléotides complémentaire d'une séquence cible, et un brin antisens présentant une séquence complémentaire du brin sens, l'ARN double-brin pouvant inhiber l'expression du gène cible, le brin sens comportant un lipide fixé, directement ou par l'intermédiaire d'un lieur, sur au moins un des six premiers nucléotides de la terminaison 5'.

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