Intracellular expression and delivery of siRNAs in mammalian cells
OwnerDavid R. Engelke, Paul D. Good +1inventors
InventorsDavid R. Engelke, Paul D. Good, Cynthia P. Paul
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Abstract
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.
Claims31
A composition comprising an siRNA molecule, wherein the molecule comprises a first region, a second region, and a third region, wherein the first region is complementary to and paired to the second region forming a double stranded region such that the double stranded region comprises about 18 to about 25 nucleotide pairs, and wherein the third region links said first region to said second region.
An expression cassette comprising a promoter operably linked to a gene encoding an siRNA molecule, wherein the siRNA molecule comprises a first region and a second region, wherein the first region is complementary to and paired to the second region forming a double stranded region such that the double stranded region comprises about 18 to about 25 nucleotide pairs.
Dependencies are inferred from the claim text.