Chemical and CRISPR/Cas9 tools for functional characterization of RNA helicases

Abstract

RNA helicases are remodeling proteins implicated in all aspects of RNA biology. Several gene expression pathways, such as ribosome biogenesis, splicing, and translation, are mis-regulated in cancer, making the helicases involved in maintaining expression flux through these networks putative drug targets. It is thus important to better understand the normal regulatory constraints of these proteins, as well as develop small molecule tools that can be used to probe their function. CRISPR/Cas9 can make a significant impact by helping to probe RNA helicase function, determining druggability, and linking the biological activity of small molecules to perturbation of RNA helicase activity.