INTRACELLULAR EXPRESSION AND DELIVERY OF siRNAs IN MAMMALIAN CELLS

摘要

The present invention relates to compositions and methods for intracellular expression and delivery of siRNAs in mammalian cells. The siRNA is transcribed intracellularly as a double stranded RNA of about 18 to about 25 base pairs long from an expression cassette. Intracellular expression of siRNA is effective at reducing or eliminating expression of the targeted genes, and is applicable to reverse genetic analysis of genes and to genetic therapy, as for example to inhibiting expression of pathogenic genes and oncogenes.