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基因治疗靶向角膜炎症:角膜炎的新兴策略。

Targeting corneal inflammation by gene therapy: Emerging strategies for keratitis.

机构信息

Pharmacokinetic, Nanotechnology and Gene Therapy Group (PharmaNanoGene), Faculty of Pharmacy, Centro de investigación Lascaray ikergunea, University of the Basque Country UPV/EHU, Paseo de la Universidad 7, Vitoria-Gasteiz, Spain.

Pharmacokinetic, Nanotechnology and Gene Therapy Group (PharmaNanoGene), Faculty of Pharmacy, Centro de investigación Lascaray ikergunea, University of the Basque Country UPV/EHU, Paseo de la Universidad 7, Vitoria-Gasteiz, Spain.

出版信息

Exp Eye Res. 2018 Nov;176:130-140. doi: 10.1016/j.exer.2018.07.006. Epub 2018 Jul 4.

DOI:10.1016/j.exer.2018.07.006
PMID:29981344
Abstract

Inflammation is the underlying process of several diseases within the eye, specifically in the cornea. Current treatment options for corneal inflammation or keratitis, and related neovascularization, are restricted by limited efficacy, adverse effects, and short duration of action. Gene therapy has shown great potential for the treatment of diseases affecting the ocular surface, and major efforts are being targeted to inflammatory mediators and neovascularization, in order to develop potential treatments for corneal inflammation. Gene therapy to treat ocular disorders is still starting, and current therapies are primarily experimental, with most human clinical trials still in research state, although some of them have already shown encouraging results. In this review, we focus on the progress and challenges of gene therapy to treat corneal inflammation. After introducing the inflammation process, we present the main nucleic acid delivery systems, including viral and non-viral vectors, and the most studied strategies to address the therapy: control of neovascularization and regulation of pro- and anti-inflammatory cytokines.

摘要

炎症是眼部几种疾病(尤其是角膜)的潜在发病机制。目前针对角膜炎症或角膜炎及相关新生血管形成的治疗选择受到疗效有限、不良反应和作用持续时间短的限制。基因治疗在治疗影响眼表面的疾病方面显示出巨大潜力,主要致力于炎症介质和新生血管形成,以开发治疗角膜炎症的潜在疗法。治疗眼部疾病的基因治疗仍处于起步阶段,目前的治疗方法主要是实验性的,大多数人类临床试验仍处于研究阶段,尽管其中一些已经显示出令人鼓舞的结果。在这篇综述中,我们重点介绍了治疗角膜炎症的基因治疗的进展和挑战。在介绍炎症过程之后,我们介绍了主要的核酸传递系统,包括病毒和非病毒载体,以及研究最多的解决该疗法的策略:控制新生血管形成和调节促炎和抗炎细胞因子。

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