siRNA滴眼液对眼部疾病的潜在治疗作用

Yuhong Nie , Zhujuan Pan , Kaili Wu
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引用次数: 5

摘要

近年来,研究人员对开发RNA干扰(RNAi)技术用于治疗各种疾病的基因抑制表达了持续的兴趣。动物模型的临床前研究和细胞培养研究表明,RNAi技术是治疗多种眼部疾病的有效实验工具,一些小干扰RNA (small interference RNA, siRNA)药物已进入I期和II期临床试验。然而,在这些研究中,sirna通过全身或结膜下/玻璃体内注射进入眼部组织,如果重复,这是侵入性和有害的。基于这一证据,我们假设局部应用siRNA滴眼液可能是一种安全有效的治疗选择,用于暂时改变基因表达的眼表疾病。此外,针对不同基因的siRNA滴眼液可能同时治疗几种眼表疾病。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
The potential therapeutic of siRNA eye drops in ocular diseases

In recent years, researchers have expressed an ongoing interest in developing RNA interference (RNAi) technology for therapeutic gene suppression in various diseases. Preclinical studies in animal models and cultured cell studies indicated that RNAi technology was an effective experimental tool against a variety of ocular diseases, and some small interference RNA (siRNA) drugs have been entered into clinical trials in Stage I and Stage II. However, in these studies siRNAs were delivered into ocular tissues via either systemic or subconjunctival/intravitreous injection, which is invasive and harmful if repeated. Based on this evidence, we hypothesize that topical application of siRNA eye drops may be a safe and effective therapeutic option in ocular surface diseases with temporary changes of gene expression. Furthermore, siRNA eye drops targeting different genes may simultaneously treat several ocular surface diseases.

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