Compositions and methods for enhancing delivery of nucleic...

A - Human Necessities – 61 – K

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A61K 48/00 (2006.01)

Patent

CA 2565685

Polynucleotide delivery-enhancing polypeptides are admixed or complexed with, or conjugated to, nucleic acids for enhancing delivery the nucleic acids into cells. The transported nucleic acids are active in target cells as small inhibitory nucleic acids (siNAs) that modulate expression of target genes, mediated at least in part by RNA interference (RNAi). The siNA/polypeptide compositions and methods of the invention provide effective tools to modulate gene expression and alter phenotype in mammalian cells, including by altering phenotype in a manner that eliminates disease symptoms or alters disease potential in targeted cells or subject individuals to which the siNA/polypeptide compositions are administered.

La présente invention a trait à des polypeptides assurant une amélioration de polynucléotides additionnés ou complexés avec, ou conjugués à, des acides nucléiques pour l'amélioration de l'administration d'acides nucléiques dans des cellules. Les acides nucléiques transportés sont actifs dans des cellules cibles en tant que petits acides nucléiques inhibiteurs qui réalisent la modulation de l'expression des gènes cibles, par l'intermédiaire au moins partiellement d'ARN interférence (ARNi). Les compositions à base de petits acides nucléiques inhibiteurs /polypeptides et les procédés de l'invention fournissent des outils efficaces pour la modulation de l'expression générique et la modification de phénotype dans des cellules mammaliennes, y compris la modification de phénotype d'une manière permettant l'élimination de symptômes de maladies et de modification de potentiel de maladies dans des cellules cibles ou chez des sujets individuels auxquels les compositions à base de petits acides nucléiques inhibiteurs/polypeptides sont administrés

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