Reprogramming RNA processing: an emerging therapeutic landscape

The production of a mature mRNA requires coordination of multiple processing steps, which ultimately control its content, localization, and stability. These steps include some of the largest macromolecular machines in the cell, which were, until recently, considered undruggable due to their biological complexity. Building from an expanded understanding of the underlying mechanisms that drive these processes, a new wave of therapeutics is seeking to target RNA processing. With a focus on impacting gene regulation at the RNA level, such modalities offer potential for sequence-specific resolution in drug design. Here, we review our current understanding of RNA-processing events and their role in gene regulation, with a focus on the therapeutic opportunities that have emerged within this landscape.

Automated summary

The production of mature mRNA requires coordination of multiple processing steps, which control its content, localization, and stability. Recent advances in understanding the underlying mechanisms have led to a new wave of therapeutics seeking to target RNA processing, offering the potential for sequence-specific resolution in drug design.