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RNA-Mediated Disease Mechanisms in Neurodegenerative Disorders

机译：RNA介导的神经退行性疾病的疾病机制

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摘要

RNA is accurately entangled in virtually all pathways that maintain cellular homeostasis. To name but a few, RNA is the "messenger" between DNA encoded information and the resulting proteins. Furthermore, RNAs regulate diverse processes by forming DNA::RNA or RNA::RNA interactions. Finally, RNA itself can be the scaffold for ribonucleoprotein complexes, for example, ribosomes or cellular bodies. Consequently, disruption of any of these processes can lead to disease. This review describes known and emerging RNA-based disease mechanisms like interference with regular splicing, the anomalous appearance of RNA protein complexes and uncommon RNA species, as well as non-canonical translation. Due to the complexity and entanglement of the above-mentioned pathways, only few drugs are available that target RNA-based disease mechanisms. However, advances in our understanding how RNA is involved in and modulates cellular homeostasis might pave the way to novel treatments. (C) 2019 Elsevier Ltd. All rights reserved.
机译：在维持细胞稳态的几乎所有途径几乎是完全缠结的RNA。只要少数，RNA是DNA编码信息和所得蛋白质之间的“信使”。此外，RNA通过形成DNA :: RNA或RNA :: RNA相互作用来调节不同的过程。最后，RNA本身可以是核糖核蛋白复合物的支架，例如核糖体或细胞体。因此，任何这些过程的破坏都会导致疾病。本综述描述了已知的和新出现的基于RNA的疾病机制，如干扰定期剪接，RNA蛋白复合物的异常外观和罕见的RNA物种，以及非典型翻译。由于上述途径的复杂性和纠缠，只有少量药物可用于靶向基于RNA的疾病机制。然而，我们理解RNA如何参与并调节细胞稳态可能会铺平新的治疗方法。 （c）2019 Elsevier Ltd.保留所有权利。

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