Background

Riboswitches recognize a small molecule through an RNA aptamer and respond by regulating gene expression. Various potential applications for synthetic riboswitches have been proposed and explored in many fields, including gene/cell therapy, regenerative medicine, vaccines, and biologics manufacturing. There have, however, been few reports of small molecules and their cognate aptamers demonstrated to function in mammalian cells, and even those typically require high concentrations of the trigger molecule (~100 μM or higher). A team of OIST researchers have developed a novel aptamer that binds to a nontoxic compound and works in mammalian cell culture and in vivo in mice at low concentrations. This technology represents a powerful addition to the currently limited toolbox for RNA-based regulation of gene expression.