Project Title:

Small molecule approaches to targeted RNA degradation

Project Summary:

Tools that target and degrade or edit nucleic acids are already having a huge impact on both basic and transnational research, with several such systems being approved as therapeutics and many more in the clinical trials. The major drawback of currently available system - they are either genetic or nucleic acid-based, which limits their scope of applicability.  To circumvent these issues, I went on to design novel small molecule systems that target and degrade RNA. I explored their use as therapeutics and designed degradation-based systems for mapping of RNA modifications. As the targetable RNA space greatly exceeds the targetable protein space, this approach can target many more disease-relevant targets than traditional therapeutic strategies. I am hopeful that these small molecule gene silencers will find use both for basic research and development of new drugs.