Negishi Yoichi, Endo-Takahashi Yoko, Maruyama Kazuo
Department of Drug Delivery and Molecular Biopharmaceutics, School of Pharmacy, Tokyo University of Pharmacy and Life Sciences.
Drug Discov Ther. 2016 Nov 28;10(5):248-255. doi: 10.5582/ddt.2016.01063. Epub 2016 Oct 28.
Gene therapy is promising for the treatment of many diseases including cancers and genetic diseases. From the viewpoint of safety, ultrasound (US)-mediated gene delivery with nano/ microbubbles was recently developed as a novel non-viral vector system. US-mediated gene delivery using nano/microbubbles are able to produce transient changes in the permeability of the cell membrane after US-induced cavitation while reducing cellular damage and enables the tissue-specific or the site-specific intracellular delivery of gene both in vitro and in vivo. We have recently developed novel lipid nanobubbles (Lipid Bubbles). These nanobubbles can also be used to enhance the efficacy of the US-mediated genes (plasmid DNA, siRNA, and miRNA etc.) delivery. In this review, we describe US-mediated delivery systems combined with nano/microbubbles and discuss their feasibility as non-viral vector systems.
基因治疗在治疗包括癌症和遗传疾病在内的多种疾病方面前景广阔。从安全性的角度来看，最近开发了一种利用纳米/微泡的超声（US）介导的基因递送方法，作为一种新型的非病毒载体系统。使用纳米/微泡的US介导的基因递送能够在超声诱导的空化作用后使细胞膜通透性产生瞬时变化，同时减少细胞损伤，并能够在体外和体内实现基因的组织特异性或位点特异性细胞内递送。我们最近开发了新型脂质纳米泡（脂质泡）。这些纳米泡也可用于提高US介导的基因（质粒DNA、siRNA和miRNA等）递送的效率。在这篇综述中，我们描述了与纳米/微泡相结合的US介导的递送系统，并讨论了它们作为非病毒载体系统的可行性。
