Columbia RNA Initiative

The mission of the Columbia RNA Initiative is to create a hub to bring together researchers and labs across Columbia University for events and opportunities that strengthen our community, stir local collaborations, and advance basic and applied RNA research and education.  

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      In addition to recent landmark discoveries and inventions of mRNA vaccines and CRISPR-based tools for programmed DNA and RNA editing, some exciting, current themes in RNA biology include the transcription of thousands of noncoding RNAs—many with expanding roles in genome regulation, myriad small RNA pathways (e.g RNAi, siRNAs, microRNAs, piRNAs), and RNAs with explicit links to disease. Recent years have also revealed the complexity of 3D structures formed by RNAs and RNA-protein complexes, many of which rival those of proteins. RNAs have been successfully employed as therapeutics for over ten years, including Macugen (Pegaptanib), the first RNA aptamer therapeutic. Now, a growing number of cellular RNAs are increasingly recognized to be opportune drug targets. In 2022, the first small-molecule drug targeting RNA, Evrysdi (Risdiplam), won FDA approval to treat patients with spinal muscular atrophy.  Evrysdi works by binding pre-mRNA to modulate mRNA splicing. Given the opportunity to target the vast real estate of coding and non-coding RNAs, it is only a matter of time before there are more RNA-targeting small molecule drugs for other diseases.  

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           Evolving views of the role of RNA in precision medicine, from marker to sculptor of genetic information, reflect a paradigm shift in genome biology. Our vision of RNA biology at Columbia is grounded in both basic and disease biology, especially cancer and personalized medicine, aspiring to understand and to develop new routes to target RNAs associated with disease and to augment our fundamental understanding of the basic RNA molecule, and its wide range of capacities and roles.