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
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Abstract
Methods and compositions are provided for modulating, e.g., reducing, viral coding sequence expression in mammals and mammalian cells. 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 introduced into a target cell, e.g., by being administered to a mammal that includes the target cell, 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.
Claims37
A method of reducing expression of a target RNA sequence in a liver of a non-embryonic mammal, said method comprising:
- administering to a non-embryonic mammal, an effective amount of an siRNA to reduce expression of a target RNA sequence in liver cells in the mammal in vivo, wherein the siRNA is specific for the target RNA sequence and comprises a duplex structure having a length in a range of from 19 to 29 base pairs,
- wherein said administering is subcutaneous administration and wherein the siRNA is not administered using a virus or using a template that encodes the siRNA.
A method of reducing expression of a target RNA sequence in a liver of a non-embryonic mammal, said method comprising:
- administering to a non-embryonic mammal, an effective amount of an siRNA to reduce expression of a target RNA sequence in liver cells in the mammal in vivo, wherein the siRNA is specific for the target RNA sequence and comprises a duplex structure that has a length in a range of from 21 to 29 base pairs,
- wherein said administering is subcutaneous administration and wherein the siRNA is not administered using a virus or using a template that encodes the siRNA.
A method of reducing expression of a target RNA sequence in a liver of a non-embryonic mammal, said method comprising:
- administering to a non-embryonic mammal, an effective amount of an siRNA to reduce expression of a target RNA sequence in liver cells in the mammal in vivo, wherein the siRNA is specific for the target RNA sequence and comprises a duplex structure of 21 base pairs in length,
- wherein said administering is subcutaneous administration and wherein the siRNA is not administered using a virus or using a template that encodes the siRNA.
A method of reducing expression of a target RNA sequence in a liver of a non-embryonic mammal, said method comprising:
- administering to a non-embryonic mammal, an effective amount of an siRNA to reduce expression of a target RNA sequence in liver cells in the mammal in vivo, wherein the siRNA is specific for the target RNA sequence and comprises a duplex structure having a length in a range of from 19 to 29 base pairs,
- wherein said administering is intravenous administration and wherein the siRNA is not administered using a virus or using a template that encodes the siRNA.
A method of reducing expression of a target RNA sequence in a liver of a non-embryonic mammal, said method comprising:
- administering to a non-embryonic mammal, an effective amount of an siRNA to reduce expression of a target RNA sequence in liver cells in the mammal in vivo, wherein the siRNA is specific for the target RNA sequence and comprises a duplex structure of 19 base pairs in length,
- wherein said administering is intravenous administration and wherein the siRNA is not administered using a virus or using a template that encodes the siRNA.
Dependencies are inferred from the claim text.