Composition and Method for In Vivo and In Vitro Attenuation of Gene Expression Using Double Stranded RNA

Abstract

Introduction of double stranded RNA into cells, cell culture, organs and tissues, and whole organisms, particularly vertebrates, specifically attenuates gene expression.

1 . A method for attenuating the expression of a target gene in a cell comprising introducing into the cell a double stranded RNA in an amount sufficient to attenuate expression of the target gene, wherein the double stranded RNA comprises a nucleotide sequence that is essentially identical to the nucleotide sequence of at least a portion of the target gene. 
     
     
         2 . The method of  claim 1  wherein the target gene is an endogenous gene. 
     
     
         3 . The method of  claim 1  wherein the target gene is a foreign gene. 
     
     
         4 . The method of  claim 1  wherein the targeted gene is a chromosomal gene. 
     
     
         5 - 7 . (canceled) 
     
     
         8 . The method of  claim 1  wherein the cell is a vertebrate cell. 
     
     
         9 . (canceled) 
     
     
         10 . The method of  claim 8  wherein the vertebrate cell is a mammalian cell. 
     
     
         11 - 16 . (canceled) 
     
     
         17 . The method of  claim 1  wherein the double stranded RNA is administered in an amount sufficient to completely inhibit expression of the target gene. 
     
     
         18 . The method of  claim 1  in which the double stranded RNA comprises one strand which is self-complementary. 
     
     
         19 . The method of  claim 1  in which the double stranded RNA comprises two separate complementary strands. 
     
     
         20 . The method of  claim 1  wherein the cell is an embryo. 
     
     
         21 . (canceled) 
     
     
         22 . (canceled) 
     
     
         23 . The method of  claim 1  wherein the cell is present in a cell culture, a tissue, an organ, or an organism. 
     
     
         24 . The method of  claim 23  wherein the cell is present in an organism, and the double stranded RNA is introduced into a body cavity or interstitial space of the organism. 
     
     
         25 . The method of  claim 23  wherein the cell is present in an organism, and wherein the double stranded RNA delivered to the organism via oral, topical, parenteral, vaginal, rectal, intranasal, ophthalmic, or intraperitoneal administration. 
     
     
         26 . The method of  claim 23  wherein the cell is present in a cell culture or a tissue explant, and wherein introduction of the double stranded RNA into the cell comprises incubating the cell culture or tissue explant in a solution comprising the double stranded RNA. 
     
     
         27 - 29 . (canceled) 
     
     
         30 . The method of  claim 1  wherein the function of the target gene is unknown. 
     
     
         31 - 48 . (canceled) 
     
     
         49 . A method for treating or preventing a disease or infection in a mammal comprising: identifying a target gene, wherein expression of the target gene is associated with the disease or infection; and administering to the mammal a double stranded RNA in an amount sufficient to attenuate expression of the target gene; wherein the double stranded RNA comprises a nucleotide sequence that is essentially identical the nucleotide sequence of at least a portion of the target gene. 
     
     
         50 . The method of  claim 49  for treating or preventing a viral infection, wherein the double stranded RNA is an antiviral double stranded RNA that attenuates the expression of a viral gene. 
     
     
         51 . The method of  claim 49  for treating or preventing cancer, wherein the double stranded RNA is an antitumor double stranded RNA. 
     
     
         52 . The method of  claim 49  for treating an autosomal dominant genetic disease wherein the double stranded RNA attenuates the expression of an allele of a gene that is associated with the disease. 
     
     
         53 - 55 . (canceled) 
     
     
         56 . A method for reducing or preventing the rejection response to transplant tissue comprising administering to the transplant tissue a double stranded RNA that attenuates the expression of a gene in the transplant tissue that can elicit an immune response in the recipient. 
     
     
         57 - 61 . (canceled)