Methods and compositions for RNAi mediated inhibition of gene expression in mammals
AssigneeThe Board of Trustees of the Leland Stanford Junior University
InventorsMark A. Kay, Anton McCaffrey
Life of this patent
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Abstract
Methods and compositions are provided for modulating, e.g., reducing, coding sequence expression in mammals. In the subject methods, an effective amount of an RNAi agent, e.g., an interfering ribonucleic acid (such as an siRNA or shRNA) or a transcription template thereof, e.g., a DNA encoding an shRNA, is administered to a non-embryonic mammal, e.g., via a hydrodynamic administration protocol. Also provided are RNAi agent pharmaceutical preparations for use in the subject methods. The subject methods and compositions find use in a variety of different applications, including academic and therapeutic applications.
Claims16
A method of reducing expression of a target mRNA in liver cells of a non-embryonic mammal in vivo, said method comprising:
- intravenously administering to said non-embryonic mammal an effective amount of an siRNA that is specific for said target mRNA to reduce expression of said target mRNA in said liver cells, wherein the siRNA comprises a 21 nucleotide (nt) sense strand and a 21 nt antisense strand that are annealed to form a 19 base pair duplex with a 2 nucleotide 3′ overhang on each end, wherein the two 3′-most nucleotides of the sense strand and the two 3′-most nucleotides of the antisense strand are deoxyribonucleotides,
- wherein the siRNA is not administered using a virus.
A method of reducing expression of a target mRNA in a liver cell of a non-embryonic mammal in vivo, said method comprising:
- intravenously administering to said non-embryonic mammal an effective amount of an siRNA specific for said target mRNA to reduce expression of said target mRNA in said liver cell, wherein said siRNA comprises a 21 nucleotide (nt) sense strand and a 21 nt antisense strand that are annealed to form a 19 base pair duplex with a 2 nucleotide 3′ overhang on each end, wherein the two 3′-most nucleotides of the sense strand and the two 3′-most nucleotides of the antisense strand are deoxyribonucleotides,
- wherein the siRNA is not administered using a virus.
Dependencies are inferred from the claim text.