Regional Centre for Biotechnology, NCR Biotech Science Cluster, 3rd Milestone, Faridabad-Gurugram Expressway, Faridabad 121001, India.
Genes (Basel). 2022 Oct 23;13(11):1930. doi: 10.3390/genes13111930.
RNA molecules, in one form or another, are involved in almost all aspects of cell physiology, as well as in disease development. The diversity of the functional roles of RNA comes from its intrinsic ability to adopt complex secondary and tertiary structures, rivaling the diversity of proteins. The RNA molecules form dynamic ensembles of many interconverting conformations at a timescale of seconds, which is a key for understanding how they execute their cellular functions. Given the crucial role of RNAs in various cellular processes, we need to understand the RNA molecules from a structural perspective. Central to this review are studies aimed at revealing the regulatory role of conformational equilibria in RNA in humans to understand genetic diseases such as cancer and neurodegenerative diseases, as well as in pathogens such as bacteria and viruses so as to understand the progression of infectious diseases. Furthermore, we also summarize the prior studies on the use of RNA structures as platforms for the rational design of small molecules for therapeutic applications.
RNA 分子以一种或另一种形式参与细胞生理学的几乎所有方面,以及疾病的发展。RNA 的功能作用的多样性来自于其内在的能力,即采用复杂的二级和三级结构,与蛋白质的多样性相媲美。RNA 分子在秒的时间尺度上形成许多相互转化构象的动态集合,这是理解它们如何执行细胞功能的关键。鉴于 RNA 在各种细胞过程中的关键作用,我们需要从结构的角度来理解 RNA 分子。这篇综述的核心是旨在揭示人类 RNA 中构象平衡的调节作用的研究,以了解癌症和神经退行性疾病等遗传疾病,以及细菌和病毒等病原体中的作用,从而了解传染病的进展。此外,我们还总结了先前关于将 RNA 结构用作合理设计小分子治疗应用平台的研究。