Decoding pseudouridine: an emerging target for therapeutic development

摘要

Pseudouridine (LP) is the most abundant post-transcriptional RNA modification and is widespread in multiple RNA species. LP impacts various aspects of RNA biology, conferring distinct structural and functional properties to the RNA molecules that it decorates. However, aberrant pseudouridylation contributes to a variety of human diseases, including cancer and genetic disorders. Dysregulation of the LP epitranscriptome can arise from mutations and abnormal expression of pseudouridylation machinery, impacting protein translation and other cellular processes. With advancing understanding of LP roles in health and disease, efforts are now invested in developing therapeutic and diagnostic approaches targeting LP. Emerging reports indicate that LP and its installation machinery could be potential pharmacological targets for therapeutic development and may serve as biomarkers for human diseases.