Small molecule replacements of transcriptional activation domains

ORGN 310

Anna K. Mapp, amapp@umich.edu, Department of Chemistry, University of Michigan, 930 N University, Ann Arbor, MI 48109-1055
Artificial transcriptional regulators are powerful tools for developing a more detailed picture of the protein-protein and protein-DNA interactions that govern gene expression. Given the range of human diseases linked to aberrant transcription patterns, artificial regulators also hold great promise for the long-term development of transcription targeted therapeutic agents. The greatest challenges are two-fold: 1. developing artificial transcriptional activators that function robustly in a cellular environment; and 2. the identification of small molecules that can function as transcriptional activation domains either in vitro or in cell culture. Towards that end, a mechanistic study of artificial transcriptional activation domains was used to delineate the relative importance of transcriptional activator characteristics that dictate functional potency, including target binding site(s), target affinity, and transcriptional activator lifetime. This data was used to design isoxazolidine-based small molecules that function as transcriptional activation domains in vitro and in S. cerevisiae with potencies comparable to an endogenous counterpart.