New paradigms in RNA biology are rapidly emerging from reports of pervasive transcription and RNA structure across genomes. Furthermore, decades of research demonstrate that RNA regulates many processes central to human health and disease. However, RNA remains a disproportionately under-explored target for drug discovery and basic chemical biology research. Progress in this area of biomedical research will require new interdisciplinary approaches at the forefront of chemistry and biology to identify and characterize RNA-small molecule interactions.
Work in my lab employs a two-phase methodology. First, we aim to develop new high-throughput screening assays for targeting structured RNAs with small molecules. These assays identify ligands that modulate RNA conformational changes. Second, we employ structural biology techniques to characterize RNA-ligand interactions, leading to the rational design of improved ligands. Iterations of this cross-discipline approach will result in advances toward novel therapeutic leads and synthetic biology tools.