For “simple” RNA targets such as helices and stemloops, sequence-selective recognition has most frequently been achieved only with relatively large molecules. While effective in vitro, these compounds typically have bioavailability issues that limit their utility in cellular assays and in vivo. This issue manifests as a large discrepancy between the measured dissociation constant (KD) for RNA binding, and the effective concentration (EC50) in a cellular assay. This talk will discuss our efforts to develop general strategies for narrowing the gap between affinity and efficacy, and will focus on two primary targets: a stemloop responsible for regulating frameshifting in HIV, and pre-miRNAs associated with a range of cancers.

Narrowing the gap between affinity and efficacy with RNA-targeted peptidomimetics

255th National Meeting (2018)

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