METHODS FOR TARGETING RNA MOLECULES

摘要

Described are methods for identifying targets in RNA molecules based on differences in RNA secondary structure related to sequence variances between different allelic forms. Also described are methods for identifying or developing small molecules which can be used to modulate a target RNA by creation of protein-small-molecule-RNA complexes, as well as compositions which include such small molecules and methods of using those small molecules and compositions.

主权项

1. A method for identifying a potential small molecule inhibitor which inhibits an activity of a cellular RNA by inducing formation of a complex with a said RNA and a cellular protein, wherein said potential small molecule inhibitor comprises a first moiety and a second moiety and a linker joining said first moiety and said second moiety, said method comprising:
a) identifying a said first moiety which binds to at least a portion of a selected cellular protein; and b) identifying a said second moiety which binds to at least a fragment of a target cellular RNA; wherein a small molecule consisting essentially of said first moiety and said second moiety and said linker connecting said first and second moieties is a said potential small molecule inhibitor.