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COMPOSITIONS AND METHODS FOR ENHANCING DELIVERY OF NUCLEIC ACIDS INTO CELLS AND FOR MODIFYING EXPRESSION OF TARGET GENES IN CELLS

机译:用于增强核酸向细胞内传递和修饰靶基因表达的组合物和方法

摘要

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.
机译:将多核苷酸递送增强多肽与核酸混合或复合或缀合至核酸,以增强核酸向细胞中的递送。转运的核酸在靶细胞中具有作为抑制靶核酸表达的小抑制核酸(siNA)的活性,至少部分地由RNA干扰(RNAi)介导。本发明的siNA /多肽组合物和方法提供了有效的工具来调节基因表达并改变哺乳动物细胞中的表型,包括通过以消除疾病症状或改变潜在靶细胞或对象个体中疾病症状或改变疾病潜能的方式改变表型。施用多肽组合物。

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