摘要
背景:帕金森病(PD)缺乏彻底的治疗是医学中的一个关键问题,并促使人们寻求新的治疗方法。在所提出的方法中,基于小干扰RNA(siRNA)的疗法作为用于治疗PD的潜在方法引起了显着的关注;然而,siRNAs递送具有潜在的缺点,例如血液循环稳定性降低以及到达靶位点的能力低。 目的:本综述旨在探索针对PD的基于siRNA的方法,以及设计纳米颗粒的最新进展,这些纳米颗粒可有效地将siRNAs靶向作用位点,并保护这些物质免于血液循环中的降解。 结果:基于siRNA的方法为通过基因沉默来设计治疗PD的新策略提供了有趣的选择,所述基因的异常表达有助于疾病的病理生理学;然而,siRNA递送到大脑是迄今尚未解决的关键问题。目前的研究工作集中在设计有效转运和保护siRNA的载体上。在这方面,正在开发纳米粒子作为具有可控递送效率和低毒性概况的siRNA的载体,并且这些代表了常见载体的替代物。 结论:PD的siRNA治疗推定的基因靶点的确定为研究非病毒载体奠定了基础。然而,解决siRNA针对大脑所面临的挑战的技术方面是必不可少的。在这方面,在纳米颗粒中制备siRNA将避免有害的副作用,如免疫原性和致癌性缺陷。
关键词: 帕金森病治疗,siRNA疗法,基于纳米粒子的siRNA递送,siRNA药物靶向,血脑屏障,纳米技术。
图形摘要
Current Drug Targets
Title:Nanotechnology As Potential Tool for siRNA Delivery in Parkinson's Disease
Volume: 18 Issue: 16
关键词: 帕金森病治疗,siRNA疗法,基于纳米粒子的siRNA递送,siRNA药物靶向,血脑屏障,纳米技术。
摘要: Background: The lack of an outright treatment for Parkinson’s disease (PD) is a pivotal concern in medicine and has driven the search for novel alternatives for treating the disease. Among the proposed approaches, small interfering RNA (siRNA)-based therapy is attracting significant attention as a potential method for the treatment of PD; however, siRNAs delivery possesses potential drawbacks, such as reduced stability in blood circulation and low capacity for reaching the target site.
Objective: This review aims to explore siRNA-based approaches to PD and the latest advances for designing nanoparticles that effectively target siRNAs to the action site and that protect these against degradation in blood circulation.
Results: siRNA-based approaches provide an interesting option for designing new strategies for treating PD through the silencing of genes, whose abnormal expressions contribute to the pathophysiology of the disease; however, siRNA delivery to the brain is a key issue that remains unsolved to date. Current research efforts are focused on designing vectors that effectively transport and protect siRNAs. In this regard, nanoparticles are being developed as carriers for siRNAs with controlled delivery efficiency and low toxicity profiles, and these represent an alternative to common vectors.
Conclusion: Identification of putative gene targets for siRNA therapy of PD has set the pace for researching non-viral vectors; however, the technological aspects for tackling the challenge that siRNAs targeting to the brain represents are essentials. In this respect, the formulation of siRNAs in nanoparticles would avoid harmful side effects, such as immunogenic and oncogenic drawbacks.
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Cite this article as:
Nanotechnology As Potential Tool for siRNA Delivery in Parkinson's Disease, Current Drug Targets 2017; 18 (16) . https://dx.doi.org/10.2174/1389450118666170321130003
DOI https://dx.doi.org/10.2174/1389450118666170321130003 |
Print ISSN 1389-4501 |
Publisher Name Bentham Science Publisher |
Online ISSN 1873-5592 |
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