LSI Seminar Series: John 'Jay' Schneekloth, Jr., Ph.D., National Cancer Institute

Recent estimates indicate that some 85% of the human genome is transcribed into RNA, but just 3% of these transcripts code for protein sequences. Further, just ~15% of proteins are “druggable” by modern medicinal chemistry, while a far greater proportion of gene products have been implicated in human disease. Taken together, these observations indicate that the ability to target noncoding RNA or the mRNA of undruggable proteins with small molecules would be a transformative advance in the development of novel therapeutics for diseases that have no cure, including many cancers. However, many of the technologies to understand and control RNA structure fall short of comparable techniques to study proteins. In this talk, I will describe my group's efforts to understand RNA as a target for small molecules, using a combination of chemical biology, structual, biochemical, molecular, and artificial intelligence-based approaches.