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US 8,008,468 B2Granted
RNAi expression constructs with liver-specific enhancer/promoter
AssigneeBenitec, Inc.
InventorsPetrus W. Roelvink, David A. Suhy, Alexander A. Kolykhalov, Mark A. Kay, Jeffery C. Giering
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Abstract
The present invention provides compositions and methods suitable for RNAi specifically in the liver so as to treat diseases or disorders.
Claims16
A DNA directed RNA interference (ddRNAi) expression construct comprising:
- one or more enhancer elements selected from the group consisting of ApoE enhancer elements and SynEnh enhancer elements;
- one or more liver-specific promoters;
- and
- one or more RNAi constructs that provide three or more RNAi agents;
- wherein the RNAi agents target hepatitis virus genes that are desired to be repressed in a liver cell or whole liver, and wherein at least one of the three or more RNAi agents is coded by SEQ ID NO:
- 42.
3 more elements
A method of modifying the expression of one or more hepatitis virus genes in the liver, comprising delivering to the liver a DNA directed RNA interference (ddRNAi) expression construct comprising:
- one or more enhancer elements selected from the group consisting of ApoE enhancer elements and SynEnh enhancer elements;
- one or more liver-specific promoters;
- and
- one or more RNAi constructs that provide three or more RNAi agents,
- wherein at least one of the three or more RNAi agents is coded by SEQ ID NO:
- 42.
3 more elements
Dependencies are inferred from the claim text.