Torres-Vanegas Julian D, Cruz Juan C, Reyes Luis H
Grupo de Diseño de Productos y Procesos (GDPP), Department of Chemical and Food Engineering, Universidad de los Andes, Bogotá 111711, Colombia.
Department of Biomedical Engineering, Universidad de los Andes, Bogotá 111711, Colombia.
Pharmaceutics. 2021 Mar 22;13(3):428. doi: 10.3390/pharmaceutics13030428.
Gene therapy has been used as a potential approach to address the diagnosis and treatment of genetic diseases and inherited disorders. In this line, non-viral systems have been exploited as promising alternatives for delivering therapeutic transgenes and proteins. In this review, we explored how biological barriers are effectively overcome by non-viral systems, usually nanoparticles, to reach an efficient delivery of cargoes. Furthermore, this review contributes to the understanding of several mechanisms of cellular internalization taken by nanoparticles. Because a critical factor for nanoparticles to do this relies on the ability to escape endosomes, researchers have dedicated much effort to address this issue using different nanocarriers. Here, we present an overview of the diversity of nanovehicles explored to reach an efficient and effective delivery of both nucleic acids and proteins. Finally, we introduced recent advances in the development of successful strategies to deliver cargoes.
基因治疗已被用作解决遗传疾病和遗传性疾病诊断与治疗的一种潜在方法。在这方面,非病毒系统已被开发为递送治疗性转基因和蛋白质的有前景的替代方案。在本综述中,我们探讨了非病毒系统(通常是纳米颗粒)如何有效克服生物屏障,以实现货物的高效递送。此外,本综述有助于理解纳米颗粒所采用的几种细胞内化机制。由于纳米颗粒实现这一目标的关键因素依赖于逃离内体的能力,研究人员已投入大量精力使用不同的纳米载体来解决这一问题。在此,我们概述了为实现核酸和蛋白质的高效递送而探索的纳米载体的多样性。最后,我们介绍了成功递送货物策略开发方面的最新进展。