Saffari Mostafa, Moghimi Hamid Reza, Dass Crispin R
Department of Pharmaceutics and Nanotechnology, School of Pharmacy, Shahid Beheshti University of Medical Sciences, Tehran, Iran. ; Current Address: Department of Pharmaceutics, School of Pharmacy, Islamic Azad University, Tehran, Iran.
Department of Pharmaceutics and Nanotechnology, School of Pharmacy, Shahid Beheshti University of Medical Sciences, Tehran, Iran.
Iran J Pharm Res. 2016 Winter;15(Suppl):3-17.
Gene therapy is a therapeutic approach to deliver genetic material into cells to alter their function in entire organism. One promising form of gene delivery system (DDS) is liposomes. The success of liposome-mediated gene delivery is a multifactorial issue and well-designed liposomal systems might lead to optimized gene transfection particularly in vivo. Liposomal gene delivery systems face different barriers from their site of application to their target, which is inside the cells. These barriers include presystemic obstacles (epithelial barriers), systemic barriers in blood circulation and cellular barriers. Epithelial barriers differ depending on the route of administration. Systemic barriers include enzymatic degradation, binding and opsonisation. Both of these barriers can act as limiting hurdles that genetic material and their vector should overcome before reaching the cells. Finally liposomes should overcome cellular barriers that include cell entrance, endosomal escape and nuclear uptake. These barriers and their impact on liposomal gene delivery will be discussed in this review.
基因治疗是一种将遗传物质导入细胞以改变其在整个生物体中功能的治疗方法。基因递送系统（DDS）的一种有前景的形式是脂质体。脂质体介导的基因递送的成功是一个多因素问题，精心设计的脂质体系统可能会导致优化的基因转染，特别是在体内。脂质体基因递送系统从其应用部位到其靶标（细胞内部）面临不同的障碍。这些障碍包括全身前障碍（上皮屏障）、血液循环中的全身屏障和细胞屏障。上皮屏障因给药途径而异。全身屏障包括酶降解、结合和调理作用。这两种屏障都可能成为遗传物质及其载体在到达细胞之前应克服的限制障碍。最后，脂质体应克服包括细胞进入、内体逃逸和核摄取在内的细胞屏障。本综述将讨论这些屏障及其对脂质体基因递送的影响。
