High Throughput Screening (HTS) Assay

In viruses, RNPs are integral components of many regulatory systems including transcription, nuclear export, stability, localization, and translation. There is an increasing appreciation that RNPs are suitable drug targets. This stems in part from the fact that RNA can adopt complex tertiary structures, with binding pockets for ligands or proteins that structurally resemble those found in enzyme active sites and at protein-protein interfaces. While the chemical character of RNA binding sites may be somewhat different from those of proteins, often involving highly charged surfaces, interactions can occur with very high affinities and specificities.

We have developed a proprietary cell-based High Throughput Screening platform specifically designed to target Ribonucleoproteins (RNPs). Cell-based assays provide the most realistic environment for the presentation of the RNP. These assays have the additional advantage of presenting multiple steps of the RNA pathway, any one of which may potentially be inhibited. In contrast, in vitro assays present only a snapshot of an RNP in a single conformation and may fail to capture the most "druggable" target state. Cell-based assays also filter out many compounds with poor solubility, uptake, localization, or toxicity.