Abstract
RNA therapeutics are emerging as a unique opportunity to drug currently
“undruggable” molecules and diseases. While their advantages over
conventional, small molecule drugs, their therapeutic implications and
the tools for their effective in vivo delivery have been extensively
reviewed, little attention has been so far paid to the technological
platforms exploited for the discovery of RNA therapeutics. Here, we
provide an overview of the existing platforms and ex vivo assays for RNA
discovery, their advantages and disadvantages, as well as their main
fields of application, with specific focus on RNA therapies that have
reached either phase 3 or market approval.