Cojocaru Elena, Ghitman Jana, Stan Raluca
Advanced Polymer Materials Group, University Politehnica of Bucharest, 1-7 Gh Polizu Street, 011061 Bucharest, Romania.
Department of Organic Chemistry "C. Nenitzescu", University Politehnica of Bucharest, 1-7 Gh Polizu Street, 011061 Bucharest, Romania.
Polymers (Basel). 2022 Jun 29;14(13):2647. doi: 10.3390/polym14132647.
Gene-based therapy represents the latest advancement in medical biotechnology. The principle behind this innovative approach is to introduce genetic material into specific cells and tissues to stimulate or inhibit key signaling pathways. Although enormous progress has been achieved in the field of gene-based therapy, challenges connected to some physiological impediments (e.g., low stability or the inability to pass the cell membrane and to transport to the desired intracellular compartments) still obstruct the exploitation of its full potential in clinical practices. The integration of gene delivery technologies with electrospun fibrous architectures represents a potent strategy that may tackle the problems of stability and local gene delivery, being capable to promote a controlled and proficient release and expression of therapeutic genes in the targeted cells, improving the therapeutic outcomes. This review aims to outline the impact of electrospun-fibrous-architecture-mediated gene therapy drug delivery, and it emphatically discusses the latest advancements in their formulation and the therapeutic outcomes of these systems in different fields of regenerative medicine, along with the main challenges faced towards the translation of promising academic results into tangible products with clinical application.
基于基因的疗法代表了医学生物技术的最新进展。这种创新方法背后的原理是将遗传物质引入特定的细胞和组织，以刺激或抑制关键信号通路。尽管基于基因的疗法领域已经取得了巨大进展，但与一些生理障碍相关的挑战（例如，稳定性低或无法穿过细胞膜并运输到所需的细胞内区室）仍然阻碍了其在临床实践中充分发挥潜力。将基因递送技术与电纺纤维结构相结合是一种有效的策略，它可以解决稳定性和局部基因递送问题，能够促进治疗性基因在靶细胞中可控且高效地释放和表达，从而改善治疗效果。这篇综述旨在概述电纺纤维结构介导的基因治疗药物递送的影响，并着重讨论其制剂的最新进展以及这些系统在再生医学不同领域的治疗效果，以及将有前景的学术成果转化为具有临床应用价值的实际产品所面临的主要挑战。
