Transcription‐Based Therapeutics

Abstract

Aberrant transcription patterns are associated with most human diseases. Therefore, enormous interest exists in the development of designer molecules that can be used to regulate directly the transcription of predetermined genes for the ultimate treatment of a wide range of disease states. One emerging strategy is to identify molecules that reconstitute one or more functions of the endogenous proteins that upregulate transcription, transcriptional activators. In doing so, they function as either inhibitors or activators of transcription. Using this approach, a variety of protein‐ and small molecule‐based transcriptional regulators have been developed, and at least one has reached clinical trials.