School of Dentistry, The University of Queensland, Brisbane, Queensland 4006, Australia.
Curr Gene Ther. 2020;20(5):356-372. doi: 10.2174/1566523220666201005110726.
In recent years, RNA interference technology has been extensively studied for its therapeutic potential against a wide variety of diseases. It aims to silence the expression of undesired genes associated with the target disease by the administration of RNA interference agents. However, these agents (nucleic acids) are unstable in the circulatory system and lack target specificity. Drug delivery systems are, therefore, crucial for the successful practice of the technique. A wide array of delivery systems has been developed to conquer these challenges, such as viral vectors, inorganic drug carriers, polymeric carriers and lipid-based carriers, with, however, significant limitations. In addition to the existing technologies, novel, innovative drug delivery systems, such as the configurable xenobot, are emerging at a rapid pace and have the potential to take the realm of biomedicine to the next level. This review summarizes technical difficulties in the development of drug delivery systems and current technologies developed for delivering RNAi agents with a discussion on their limitations.
近年来，RNA 干扰技术因其在治疗多种疾病方面的治疗潜力而受到广泛研究。它旨在通过给予 RNA 干扰剂来沉默与目标疾病相关的不想要的基因的表达。然而，这些（核酸）药物在循环系统中不稳定，缺乏靶向特异性。因此，药物输送系统对于该技术的成功实践至关重要。已经开发了广泛的输送系统来克服这些挑战，例如病毒载体、无机药物载体、聚合物载体和基于脂质的载体，但它们具有显著的局限性。除了现有的技术外，新型创新的药物输送系统，如可配置的 xenobot，正在迅速涌现，并有可能将生物医学领域提升到一个新的水平。本综述总结了药物输送系统发展中的技术难点和目前开发的用于递送 RNAi 药物的技术，并讨论了它们的局限性。
