Decoding pseudouridine: an emerging target for therapeutic development

作者：Jonas Cerneckis, Qi Cui, Chuan He, Chengqi Yi, Yanhong Shi · 发表于：Trends in Pharmacological Sciences · 年份：2022 · DOI：10.1016/j.tips.2022.03.008 · 被引用次数：154 · 研究领域：RNA modifications and cancer、RNA Research and Splicing、Cancer-related molecular mechanisms research

Pseudouridine (Ψ) is the most abundant post-transcriptional RNA modification and is widespread in multiple RNA species. Ψ 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 Ψ epitranscriptome can arise from mutations and abnormal expression of pseudouridylation machinery, impacting protein translation and other cellular processes. With advancing understanding of Ψ roles in health and disease, efforts are now invested in developing therapeutic and diagnostic approaches targeting Ψ. Emerging reports indicate that Ψ and its installation machinery could be potential pharmacological targets for therapeutic development and may serve as biomarkers for human diseases.