The Steve and Cindy Rasmussen Institute for Genomic Medicine, Nationwide Children's Hospital, Columbus, OH, USA.
Institute of Molecular Biology and Biotechnology, The University of Lahore, Lahore, Pakistan.
Methods Mol Biol. 2025;2867:331-344. doi: 10.1007/978-1-0716-4196-5_19.
An appealing strategy for the treatment of several diseases is the therapeutic targeting of noncoding RNAs (ncRNAs), such as microRNAs (miRNAs) and long noncoding RNAs (lncRNAs). Many antisense oligonucleotides and small interfering RNAs have been tested in clinical studies over the past 10 years, and several of these have received FDA approval. However, trial results have thus far been mixed, with some studies reporting strong effects and others showing low effectiveness or side effects, including toxicity. Clinical trials for alternative entities like antimiRNAs are underway, and interest in lncRNA-based therapies is constantly growing. From this perspective, we discuss the basic overview of ncRNAs, their significant role as therapeutic biomarkers against different diseases, and the role of secondary structure in noncoding RNAs.
针对多种疾病的一种有吸引力的治疗策略是针对非编码 RNA（ncRNA）的治疗靶向，例如 microRNAs（miRNAs）和长非编码 RNA（lncRNAs）。在过去的 10 年中，已经在临床研究中测试了许多反义寡核苷酸和小干扰 RNA，其中一些已获得 FDA 批准。然而，迄今为止试验结果喜忧参半，一些研究报告了强烈的效果，而另一些则显示出低有效性或副作用，包括毒性。针对抗 miRNA 等替代实体的临床试验正在进行中，基于 lncRNA 的治疗方法的兴趣也在不断增长。从这个角度来看，我们讨论了 ncRNA 的基本概述、它们作为针对不同疾病的治疗生物标志物的重要作用，以及二级结构在非编码 RNA 中的作用。
