Novel therapeutic approaches are being developed to tackle neurodegenerative diseases, due to the lack of efficiency of the known druggable targets. For Huntington's disease, a promising approach is the regulation of the RNA product. This target would allow for a selective and effective inhibition of the toxic effects exerted by the final nucleic product and the coded protein. In this review, the current state of the art of RNA regulation is discussed, with a brief but insightful view on novel plausible targets. After this, an emphasis on successful computational and experimental approaches tailored in modeling and regulating RNA aberrant behavior are extensively presented. Finally, the application and limitations of current computational methods are discussed, and possible avenues for improvement are outlined.

中文翻译：

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亨廷顿氏病中RNA调节的挑战：计算研究的见解

由于缺乏已知的可治疗靶标的效率，正在开发新颖的治疗方法来应对神经退行性疾病。对于亨廷顿氏病，一种有前途的方法是调节RNA产物。该靶标将允许选择性和有效地抑制由最终核酸产物和编码的蛋白质产生的毒性作用。在这篇综述中，我们对RNA调节的最新技术进行了讨论，并对新颖的可能靶标进行了简短而有见地的见解。在此之后，重点介绍了针对建模和调节RNA异常行为而量身定制的成功计算和实验方法。最后，讨论了当前计算方法的应用和局限性，并概述了可能的改进途径。