1] Department of Chemical Engineering, Massachusetts Institute of Technology, Cambridge, Massachusetts 02142, USA [2] David H. Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology, Cambridge, Massachusetts 02142, USA.
Nat Mater. 2013 Nov;12(11):967-77. doi: 10.1038/nmat3765.
RNA interference (RNAi) has broad potential as a therapeutic to reversibly silence any gene. To achieve the clinical potential of RNAi, delivery materials are required to transport short interfering RNA (siRNA) to the site of action in the cells of target tissues. This Review provides an introduction to the biological challenges that siRNA delivery materials aim to overcome, as well as a discussion of the way that the most effective and clinically advanced classes of siRNA delivery systems, including lipid nanoparticles and siRNA conjugates, are designed to surmount these challenges. The systems that we discuss are diverse in their approaches to the delivery problem, and provide valuable insight to guide the design of future siRNA delivery materials.
RNA 干扰 (RNAi) 具有作为一种治疗方法可逆沉默任何基因的广泛潜力。为了实现 RNAi 的临床潜力，需要输送材料将短干扰 RNA (siRNA) 输送到靶组织细胞的作用部位。本综述介绍了 siRNA 输送材料旨在克服的生物学挑战，以及讨论了最有效和临床最先进的 siRNA 输送系统类别，包括脂质纳米颗粒和 siRNA 缀合物，如何设计克服这些挑战。我们讨论的系统在输送问题的方法上是多种多样的，并为指导未来 siRNA 输送材料的设计提供了有价值的见解。
