Farris Eric, Sanderfer Kari, Lampe Anna, Brown Deborah M, Ramer-Tait Amanda E, Pannier Angela K
Department of Biological Systems Engineering, University of Nebraska-Lincoln, Lincoln, NE 68583.
School of Biological Sciences, University of Nebraska-Lincoln, Lincoln, NE 68583.
Curr Opin Biomed Eng. 2018 Sep;7:51-57. doi: 10.1016/j.cobme.2018.09.003. Epub 2018 Sep 26.
Non-viral gene delivery via the oral route is a promising strategy for improving outcomes of DNA vaccination and gene therapy applications. Unlike traditional parenteral administration routes, the oral route is a non-invasive approach that lends itself to high patient compliance and ease of dosing. Moreover, oral administration allows for both local and systemic production of therapeutic genes or, in the case of DNA vaccination, mucosal and systemic immunity. However, the oral route presents distinct challenges and barriers to achieving successful gene delivery. Oral non-viral gene delivery systems must be able to survive the harsh and variable environments (e.g. acidic pH, degrading enzymes, mucus layer) encountered during transit through the gastrointestinal tract, while still allowing for efficient transgene production at sites of interest. These barriers present unique design challenges for researchers in material selection and in improving the transfection efficiency of orally delivered genes. This review provides an overview of advancements in the design of oral non-viral gene delivery systems, and highlights recent and important developments towards improving orally delivered genes for applications in gene therapy and DNA vaccination.
通过口服途径进行非病毒基因递送是一种很有前景的策略，可改善DNA疫苗接种和基因治疗应用的效果。与传统的肠胃外给药途径不同，口服途径是一种非侵入性方法，具有很高的患者依从性且易于给药。此外，口服给药既可以使治疗性基因在局部产生，也可以在全身产生，或者在DNA疫苗接种的情况下，诱导黏膜免疫和全身免疫。然而，口服途径在实现成功的基因递送方面存在独特的挑战和障碍。口服非病毒基因递送系统必须能够在通过胃肠道转运过程中遇到的恶劣且多变的环境（例如酸性pH值、降解酶、黏液层）中存活，同时仍能在目标部位高效产生转基因。这些障碍给研究人员在材料选择以及提高口服基因的转染效率方面带来了独特的设计挑战。本文综述了口服非病毒基因递送系统设计方面的进展，并重点介绍了在改善用于基因治疗和DNA疫苗接种的口服基因方面的最新重要进展。
