Despite the fact that some 85% of the human genome is transcribed into RNA, just 3% of these sequences code for proteins. Moreover, just 15% of the human proteome is thought to be “druggable” with current methods. Coupled with extensive genome-wide association studies that indicate RNA as a driver of diverse diseases, these observations lead to the idea that small molecules capable of targeting RNA could be powerful therapeutics for diverse diseases with no cure. In this seminar, I will discuss our laboratory’s efforts to understand targeting RNA with small molecules. This includes our work using a small molecule microarray (SMM) high throughput screening platform coupled with machine learning approaches to characterize RNA-binding chemical space, development of chemical probes and structure-guided design on RNA, and examples of how to target mRNAs of “undruggable” oncogenes.
