ncRNAs 在治疗中的应用：基于核酸的药物递送的挑战和局限性。
ncRNAs in Therapeutics: Challenges and Limitations in Nucleic Acid-Based Drug Delivery.
机构信息
Department of Nephrology, Hospital Universitari de Bellvitge, 08907 L'Hospitalet de Llobregat, Spain.
Nephrology and Renal Transplantation Group, Infectious Disease and Transplantation Program, Institut d'Investigació Biomèdica de Bellvitge-IDIBELL, 08907 L'Hospitalet de Llobregat, Spain.
出版信息
Int J Mol Sci. 2021 Oct 27;22(21):11596. doi: 10.3390/ijms222111596.
Non-coding RNAs (ncRNAs) are emerging therapeutic tools but there are barriers to their translation to clinical practice. Key issues concern the specificity of the targets, the delivery of the molecules, and their stability, while avoiding "on-target" and "off-target" side effects. In this "ncRNA in therapeutics" issue, we collect several studies of the differential expression of ncRNAs in cardiovascular diseases, bone metabolism-related disorders, neurology, and oncology, and their potential to be used as biomarkers or therapeutic targets. Moreover, we review recent advances in the use of antisense ncRNAs in targeted therapies with a particular emphasis on their basic biological mechanisms, their translational potential, and future trends.
非编码 RNA（ncRNAs）正在成为有潜力的治疗工具，但将其转化为临床实践仍存在障碍。关键问题涉及靶标的特异性、分子的递送及其稳定性，同时要避免“靶内”和“靶外”的副作用。在本期“ncRNA 在治疗学中的应用”中，我们收集了一些关于 ncRNA 在心血管疾病、骨代谢相关疾病、神经学和肿瘤学中的差异表达的研究，以及它们作为生物标志物或治疗靶点的潜在应用。此外，我们还综述了反义 ncRNAs 在靶向治疗中的最新进展，特别强调了它们的基本生物学机制、转化潜力和未来趋势。
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