Transcriptional activators play an essential role in the regulatory network that controls gene-specific transcription. The mis-regulation of this complex event cascade is correlated with a growing number of human diseases. Several difficulties have been encountered in the use and analysis of the transcriptional activation systems; for example, overexpression of some transcriptional activators will squelch other known activators or randomly activate other genes. Hence, developing artificial transcriptional activators would be an enormously powerful tool for pharmaceutical applications as well as for basic research.
This invention describes the design and synthesis of small molecule activation domain compositions that activate transcription at levels comparable to a natural transcription factor. This invention also provides methods of regulating gene expression using the novel activation domains.
Applications and Advantages
- This technology will be useful as a rapid-nl-screening assay for small molecule ligands-nl-of a protein of interest.
- This technology provides a new method to-nl-lead to protein overexpression and for-nl-turning specific gene on to study gene function
- This technology provides a method for drug-nl-discovery of transcription-based therapeutics.
- This invention offers a powerful alternative for overcoming the current need to incorporate specific exogenous promoters to control the expression of the targeted gene.