RNAi EXPRESSION CONSTRUCTS
AssigneeBenitec, Inc.
InventorsPetrus W. Roelvink, David A. Suhy, Alexander A. Kolykhalov, Linda Couto
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Abstract
The present invention provides compositions and methods suitable for expressing 1-x RNAi agents against a gene or genes in cells, tissues or organs of interest in vitro and in vivo so as to treat diseases or disorders.
Claims6
A genetic construct capable of modifying expression of one or more genes, the genetic construct comprising an RNAi expression cassette comprising a promoter and two or more stem-loop structures separated by one or more spacer regions, the stem-loop structures being RNAi agents when transcribed from the promoter.
A 1-x RNA expression cassette comprising a promoter and two or more stem-loop structures separated from one another by one or more spacer regions, the cassette encoding two or more RNAi agents for modifying target gene expression.
A method of modifying the expression of one or more genes, comprising:
- introducing to a cell, tissue, or organ of interest a genetic construct comprising an RNAi expression cassette comprising a promoter and two or more stem-loop structures separated from one another by one or more spacer regions, the stem-loop structures being RNAi agents when transcribed from the promoter.
The method of claim 13 wherein the stem-loop structures target one or more sequences as set forth in any of SEQ ID NO:
- 1-56.
Dependencies are inferred from the claim text.