Regulating gene expression

Turning off disease-associated genes is an obvious strategy to treat conditions where targets have been identified. Ideally, gene-inactivating drugs would be small molecules that prevent synthesis of the protein encoded by the target gene.

Research recently published in Science may validate the use of small molecules to target mRNA and inhibit the activity of disease-associated genes. While the technology itself could be useful for gene therapy, the approach more importantly shows that finding small molecules to regulate endogenous genes is a feasible drug discovery strategy...