Rnai expression constructs

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.

What is claimed is: 
     
         1 . 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. 
     
     
         2 . The genetic construct of  claim 1  wherein the stem-loop structures target one or more sequences as set forth in any of SEQ ID No: 1-56. 
     
     
         3 . 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. 
     
     
         4 . The cassette of  claim 3  wherein the stem-loop structures target one or more sequences as set forth in any of SEQ ID No: 1-56. 
     
     
         5 . 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.   
     
     
         6 . 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.