Department of Chemical Engineering, University of Waterloo, Waterloo, Ontario, N2L 3G1, Canada.
Curr Med Chem. 2012;19(2):197-208. doi: 10.2174/092986712803414141.
Despite their relatively lower efficiency, nonviral approaches are emerging as safer alternatives in gene therapy to viral vectors. Delivery of nucleic acids to the target site is an important factor for effective gene expression (plasmid DNA) or knockdown (siRNA) with minimal side effects. Direct deposition at the target site by physical methods, including ultrasound, electroporation and gene gun, is one approach for local delivery. For less accessible sites, the development of carriers that can home into the target tissue is required. Cationic peptides, lipoplexes, polyplexes and nanoplexes have been used as carriers for delivery of nucleic acids. Targeting ligands, such as cell targeting peptides, have also been applied to decorate delivery vehicles in order to enhance their efficacy. This review focuses on delivery strategies and recent progress in non-viral carriers and their modifications to improve their performance in targeting and transfection.
尽管非病毒方法的效率相对较低，但它们作为基因治疗中病毒载体的更安全替代品正在出现。将核酸递送到靶位是实现有效基因表达（质粒 DNA）或敲低（siRNA）的重要因素，同时副作用最小。通过物理方法（包括超声、电穿孔和基因枪）直接在靶位沉积是局部递送的一种方法。对于较难到达的部位，需要开发能够靶向目标组织的载体。阳离子肽、脂质体、多聚物和纳米复合物已被用作递送核酸的载体。靶向配体，如细胞靶向肽，也已被应用于修饰递药系统，以提高其靶向和转染效率。本文综述了非病毒载体的递药策略和最新进展，以及对其进行修饰以改善靶向和转染性能的研究进展。
