Institute of Biomaterials and Biomedical Engineering, University of Toronto, Toronto, ON, Canada.
J Biol Eng. 2012 Jun 11;6(1):7. doi: 10.1186/1754-1611-6-7.
Controlling gene expression via small interfering RNA (siRNA) has opened the doors to a plethora of therapeutic possibilities, with many currently in the pipelines of drug development for various ocular diseases. Despite the potential of siRNA technologies, barriers to intracellular delivery significantly limit their clinical efficacy. However, recent progress in the field of drug delivery strongly suggests that targeted manipulation of gene expression via siRNA delivered through nanocarriers can have an enormous impact on improving therapeutic outcomes for ophthalmic applications. Particularly, synthetic nanocarriers have demonstrated their suitability as a customizable multifunctional platform for the targeted intracellular delivery of siRNA and other hydrophilic and hydrophobic drugs in ocular applications. We predict that synthetic nanocarriers will simultaneously increase drug bioavailability, while reducing side effects and the need for repeated intraocular injections. This review will discuss the recent advances in ocular siRNA delivery via non-viral nanocarriers and the potential and limitations of various strategies for the development of a 'universal' siRNA delivery system for clinical applications.
通过小干扰 RNA（siRNA）控制基因表达为众多治疗方法开辟了道路，目前许多方法都处于各种眼部疾病药物开发的管道中。尽管 siRNA 技术具有潜力，但细胞内递送至的障碍极大地限制了它们的临床疗效。然而，药物输送领域的最新进展强烈表明，通过纳米载体递送至的 siRNA 进行靶向基因表达操纵可对改善眼科应用的治疗效果产生巨大影响。特别是合成纳米载体已证明它们适合作为一种可定制的多功能平台，可用于靶向递送至眼内的 siRNA 以及其他亲水性和疏水性药物。我们预测，合成纳米载体将同时提高药物的生物利用度，同时减少副作用和需要重复眼内注射。本综述将讨论通过非病毒纳米载体进行眼部 siRNA 递送至的最新进展，以及为临床应用开发“通用”siRNA 递送系统的各种策略的潜力和局限性。
