Development of computational and chemical tools to improve methods for targeting RNA with small molecules
Seedhouse, Steven J.
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The ubiquitous role of RNA in living systems is becoming more and more apparent each year. Historically RNA was treated as a passive carrier of genetic information from its more stable chemical brother DNA, to a more functionally diverse but less structurally similar cousin, protein. Now though, it is known that RNA has a plethora of other essential roles beyond the relay of genetic information from the nucleus to the cytoplasm. Lagging behind our knowledge of RNA structure and function though, is a complementary knowledgebase describing how to effectively design drugs that target RNA. Most current therapeutics target protein, therefore targeting RNA represents a challenge and opportunity for medicinal chemists, and could potentially lead to treatments for cancer, muscular dystrophy, and HIV among other diseases. A unique approach combining experimental and computational tools was developed and utilized to improve our current understanding of how to effectively target RNA with small molecules. The tools that were developed and the research described herein also lay a foundation for future progress in this area.