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.
Claims19
A 1-x RNAi expression cassette encoding x stem-loop structures, wherein x is two or more, and the stem-loop structures are separated from one another by one or more spacer regions, wherein the stem-loop structures are RNAi agents and at least one of the RNAi agents is encoded by SEQ ID NO:42.
A method of modifying the expression of one or more genes, comprising:
- introducing to a cell, tissue, or organ of interest a 1-x RNAi expression cassette encoding x stem-loop structures, wherein x is two or more and the stem-loop structures are separated from one another by one or more spacer regions, wherein the stem-loop structures are RNAi agents and at least one of the RNAi agents is encoded by SEQ ID NO:42.
A method of modifying the expression of one or more genes, comprising:
- introducing to a cell, tissue, or organ of interest an RNAi sequence that is an in vitro transcription product of a 1-x RNAi expression cassette wherein x is two or more and the stem-loop structures are separated from one another by one or more spacer regions, wherein the stem-loop structures are RNAi agents and at least one of the RNAi agents is encoded by SEQ ID NO:
- 42.
Dependencies are inferred from the claim text.