Cancer and organ injury—such as that occurring in the perioperative period, including acute lung injury, myocardial infarction, and acute gut injury—are among the leading causes of death in the United States and impose a significant impact on quality of life. MicroRNAs (miRNAs) have been studied extensively during the last two decades for their role as regulators of gene expression, their translational application as diagnostic markers, and their potential as therapeutic targets for disease treatment. Despite promising preclinical outcomes implicating miRNA targets in disease treatment, only a few miRNAs have reached clinical trials. This likely relates to difficulties in the delivery of miRNA drugs to their targets to achieve efficient inhibition or overexpression. Therefore, understanding how to efficiently deliver miRNAs into diseased tissues and specific cell types in patients is critical. This review summarizes current knowledge on various approaches to deliver therapeutic miRNAs or miRNA inhibitors and highlights current progress in miRNA-based disease therapy that has reached clinical trials. Based on ongoing advances in miRNA delivery, we believe that additional therapeutic approaches to modulate miRNA function will soon enter routine medical treatment of human disease, particularly for cancer or perioperative organ injury.

中文翻译：

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调节 MicroRNA 功能治疗癌症或器官损伤的策略。


癌症和器官损伤（例如围手术期发生的损伤，包括急性肺损伤、心肌梗塞和急性肠道损伤）是美国的主要原因之一，对生活质量产生重大影响。 MicroRNA (miRNA) 在过去二十年中因其作为基因表达调节剂的作用、作为诊断标记的翻译应用以及作为疾病治疗靶点的潜力而得到了广泛的研究。尽管 miRNA 靶标在疾病治疗中的临床前结果很有希望，但只有少数 miRNA 进入了临床试验。这可能与将 miRNA 药物递送至靶标以实现有效抑制或过度表达的困难有关。因此，了解如何有效地将 miRNA 传递到患者的患病组织和特定细胞类型中至关重要。这篇综述总结了目前关于提供治疗性 miRNA 或 miRNA 抑制剂的各种方法的知识，并强调了基于 miRNA 的疾病治疗目前已达到临床试验的进展。基于 miRNA 递送的不断进展，我们相信调节 miRNA 功能的其他治疗方法将很快进入人类疾病的常规医学治疗，特别是癌症或围手术期器官损伤。