纳米技术作为siRNA递送帕金森病的潜在工具

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.

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