基于 RNA 的治疗学：概述与展望。
RNA-based therapeutics: an overview and prospectus.
机构信息
Laboratory of Cancer Biology, Key Lab of Biotherapy in Zhejiang Province, Cancer Center of Zhejiang University, Sir Run Run Shaw Hospital, School of Medicine, Zhejiang University, Hangzhou, Zhejiang, China.
Department of Medical Oncology, Sir Run Run Shaw Hospital, School of Medicine, Zhejiang University, Hangzhou, Zhejiang, China.
出版信息
Cell Death Dis. 2022 Jul 23;13(7):644. doi: 10.1038/s41419-022-05075-2.
The growing understanding of RNA functions and their crucial roles in diseases promotes the application of various RNAs to selectively function on hitherto "undruggable" proteins, transcripts and genes, thus potentially broadening the therapeutic targets. Several RNA-based medications have been approved for clinical use, while others are still under investigation or preclinical trials. Various techniques have been explored to promote RNA intracellular trafficking and metabolic stability, despite significant challenges in developing RNA-based therapeutics. In this review, the mechanisms of action, challenges, solutions, and clinical application of RNA-based therapeutics have been comprehensively summarized.
对 RNA 功能及其在疾病中的关键作用的认识不断加深，促使人们将各种 RNA 应用于选择性地针对迄今为止“不可成药”的蛋白质、转录本和基因，从而可能扩大治疗靶点。已有几种基于 RNA 的药物获得批准用于临床应用，而其他药物仍在研究或临床前试验中。尽管在开发基于 RNA 的治疗方法方面存在重大挑战，但人们仍在探索各种技术来促进 RNA 的细胞内运输和代谢稳定性。在这篇综述中，全面总结了基于 RNA 的治疗方法的作用机制、挑战、解决方案和临床应用。
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