Wullner Ulrich, Neef Inga, Tur Mehmet K, Barth Stefan
Department of Pharmaceutical Product Development, Fraunhofer Institute for Molecular Biology and Applied Ecology, Aachen, Germany.
Recent Pat Anticancer Drug Discov. 2009 Jan;4(1):1-8. doi: 10.2174/157489209787002506.
RNA interference (RNAi) is a powerful endogenous process initiated by short double stranded RNAs, which results in sequence-specific posttranscriptional gene silencing. The ability to block the expression of any disease-causing or disease-related protein emphasizes the huge therapeutic potential of this technology. In a clinical setting, however, the use of RNAi-based therapeutics is limited by their short serum half lives and poor uptake into cells. In this review, we provide an overview of recent patents in the field of short interfering RNA (siRNA) delivery and discuss recent progress in the development of efficient siRNA delivery vehicles enhancing the pharmacokinetic properties of RNAi-based therapeutics and promoting cellular uptake.
RNA干扰（RNAi）是一种由短双链RNA引发的强大的内源性过程，它会导致序列特异性的转录后基因沉默。阻断任何致病或与疾病相关蛋白质表达的能力凸显了这项技术巨大的治疗潜力。然而，在临床环境中，基于RNAi的疗法的应用受到其血清半衰期短和细胞摄取不良的限制。在这篇综述中，我们概述了短干扰RNA（siRNA）递送领域的近期专利，并讨论了高效siRNA递送载体开发方面的最新进展，这些载体可增强基于RNAi疗法的药代动力学特性并促进细胞摄取。
