XRN1 Inhibitors

XRN1 is a critical enzyme in cellular RNA metabolism, playing a pivotal role in RNA decay, processing, and quality control mechanisms. It functions primarily in the degradation of mRNA molecules following deadenylation, thereby regulating gene expression by controlling the turnover of mRNA. XRN1 is also involved in the processing of rRNA, snRNA, and snoRNA, as well as in the degradation of aberrant RNAs and the resolution of structured RNA intermediates in various cellular pathways. Its activity is essential for maintaining RNA homeostasis, influencing post-transcriptional gene regulation, and modulating the cellular response to environmental stimuli. Through its involvement in the exosomal pathway, XRN1 helps to ensure the fidelity of gene expression and prevents the accumulation of harmful RNA species, highlighting its importance in both normal cellular function and stress responses.

The inhibition of XRN1's function can disrupt the delicate balance of RNA species within the cell, leading to the accumulation of mRNA transcripts and aberrant RNAs that can affect cellular homeostasis and viability. Mechanisms of XRN1 inhibition can include direct binding of small molecule inhibitors to the active site of the enzyme, interfering with its catalytic activity. Additionally, post-translational modifications of XRN1 or alterations in its interacting partners can modulate its activity, leading to a reduction in RNA degradation capacity. Genetic mutations that result in the production of a non-functional XRN1 protein can also inhibit its activity, contributing to the dysregulation of RNA metabolism and associated cellular processes.