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Antisense Oligonucleotide: Basic Concepts and Therapeutic Application in Inflammatory Bowel Disease

机译:反义寡核苷酸:炎症性肠病中的基本概念和治疗应用

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Several molecular technologies aimed at regulating gene expression that have been recently developed as a strategy to combat inflammatory and neoplastic diseases. Among these, antisense technology is a specific, rapid, and potentially high-throughput approach for inhibiting gene expression through recognition of cellular RNAs. Advances in the understanding of the molecular mechanisms that drive tissue damage in different inflammatory diseases, including Crohn’s disease (CD) and ulcerative colitis (UC), the two major inflammatory bowel diseases (IBDs) in humans, have facilitated the identification of novel druggable targets and offered interesting therapeutic perspectives for the treatment of patients. This short review provides a comprehensive understanding of the basic concepts underlying the mechanism of action of the oligonucleotide therapeutics, and summarizes the available pre-clinical and clinical data for oligonucleotide-based therapy in IBD.
机译:旨在调节最近被制定为对抗炎症和肿瘤疾病的策略的基因表达的几种分子技术。其中,反义技术是通过识别细胞RNA来抑制基因表达的特异性,快速和潜在的高通量方法。理解促进不同炎症性疾病中的组织损伤的分子机制的进展,包括克罗恩病(CD)和溃疡性结肠炎(UC),人类的两个主要炎症性肠病(IBDS),促进了新型可毒靶的鉴定并为治疗患者提供有趣的治疗性观点。这次简短的评论对寡核苷酸治疗剂的作用机制的基本概念提供了全面的了解,并总结了IBD中基于寡核苷酸的疗法的可用前临床和临床数据。

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