Abstract: The invention provides a method of regulating U1 activity associated with its splicing role as well as its role in protecting pre-mRNAs from premature termination by cleavage and polyadenylation, thereby modulating expression of a gene or genes. In one embodiment, the invention includes compositions and methods for regulating gene expression and treating diseases associated with dysregulated gene expression.

Abstract: The invention provides a method of regulating U1 activity associated with its splicing role as well as its role in protecting pre-mRNAs from premature termination by cleavage and polyadenylation, thereby modulating expression of a gene or genes. In one embodiment, the invention includes compositions and methods for regulating gene expression and treating diseases associated with dysregulated gene expression.

Abstract: The present invention is based on the discovery of a cell-based system to identify novel modulators of splicing or splicing dependent processes. The cell-based system of the present invention utilizes a fast and highly sensitive reporter, that responds to defects in the splicing machinery itself and is sensitive to changes in the signals that regulate splicing dependent processes such as those that modulate the EJC, splicing-dependent export, localization or translation efficiency. The present invention further uses the cell-based screen to identify several small molecules that modulate both constitutive and alternative splicing. Accordingly, the present invention includes general or alternative splicing inhibitors identified using the assay described herein. The present invention also provides methods of treating a subject having a condition associated with aberrant target RNA expression.

Abstract: The present invention relates to the discovery of a novel global gene expression regulation mechanism whereby U1 snRNP (U1) protects pre-mRNAs. In one embodiment, the invention includes compositions and methods for regulating gene expression and treating diseases associated with dysregulated gene expression.

Abstract: The present invention is based on the discovery of a cell-based system to identify novel modulators of splicing or splicing dependent processes. The cell-based system of the present invention utilizes a fast and highly sensitive reporter, that responds to defects in the splicing machinery itself and is sensitive to changes in the signals that regulate splicing dependent processes such as those that modulate the EJC, splicing-dependent export, localization or translation efficiency. The present invention further uses the cell-based screen to identify several small molecules that modulate both constitutive and alternative splicing. Accordingly, the present invention includes general or alternative splicing inhibitors identified using the assay described herein. The present invention also provides methods of treating a subject having a condition associated with aberrant target RNA expression.