Drug Target Strategies
Recent estimates indicate that greater than 85% of the human genome is transcribed into RNA, yet just 3% of these transcripts code for protein sequences. Coupled with an increased knowledge of the noncoding functions of RNA and improved technologies for RNA structure determination, this information has given rise to interest in RNA as a therapeutic target for small molecules. In this presentation, I will discuss my group's recent efforts to understand RNA-small molecule interactions. We have developed a high throughput Small Molecule Microarray (SMM) screening platform, which we use to rapidly screen and profile RNA-binding small molecules. Further, I will discuss in detail several targets we have studied. I will also discuss our efforts toward structure-guided design of RNA-binding molecules and the potential for future development.