Center for Pharmaceutical Biotechnology and Nanomedicine, Northeastern University, 360 Huntington Ave, Boston, MA 02115, USA.
Front Biosci (Landmark Ed). 2013 Jan 1;18(1):58-79. doi: 10.2741/4087.
The chance to selectively intervene and stop the development of any gene-dependent disease in different organs and pathologies makes siRNA an ideal therapeutic agent. However, serious issues should be addressed before the real therapeutic use of siRNA. The poor pharmacokinetic properties of siRNA, its short half-life, its low in vivo stability, its fast elimination by kidney filtration and its low transfection efficiency complicate the use of siRNA as a therapeutic molecule. In this review, we will describe the latest and most advanced approaches and strategies undertaken to address these limitations and improve siRNA delivery and its gene silencing efficacy as well as the prospects for its therapeutic applications.
siRNA 作为一种理想的治疗药物，有机会选择性干预和阻止不同器官和病变中任何依赖基因的疾病的发展。然而，在真正将 siRNA 用于治疗之前，还需要解决一些严重的问题。siRNA 的药代动力学性质差、半衰期短、体内稳定性低、肾脏过滤快速清除以及转染效率低，这些都使得 siRNA 的应用变得复杂。在这篇综述中，我们将描述为解决这些限制以及提高 siRNA 递呈和基因沉默效果而采取的最新和最先进的方法和策略，以及其治疗应用的前景。
