Recent estimates suggest approximately 80% of the human genome is transcribed, but only 3% of transcripts code for protein sequences.  Advances in both the structure and function of RNA have revealed major roles for noncoding RNA and DNA in regulating gene expression and in disease.  However, the majority of modern medicinal chemistry focuses only on protein targets for small molecule drugs.  Our laboratory uses a small molecule microarray (SMM) approach to rapidly screen and profile small molecules that bind directly to RNA and alternatively folded DNA.  In this seminar, I will discuss our use of the SMM screening platform and how it has been leveraged to identify druglike small molecules (including FDA approved drugs) that bind to nucleic acids and perturb gene expression and other processes such as alternative splicing.  Recent successes include the identification of selective G-quadruplex binders, inhibitors of microRNAs, and viral RNAs.

Targeting structurally and functionally diverse nucleic acids with druglike small molecules

254th National Meeting (2017)

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