Kaygisiz Kübra, Synatschke Christopher V
Max Planck Institute for Polymer Research, Ackermannweg 10, 55128 Mainz, Germany.
Biomater Sci. 2020 Nov 21;8(22):6113-6156. doi: 10.1039/d0bm01367f. Epub 2020 Oct 7.
Therapeutic viral gene delivery is an emerging technology which aims to correct genetic mutations by introducing new genetic information to cells either to correct a faulty gene or to initiate cell death in oncolytic treatments. In recent years, significant scientific progress has led to several clinical trials resulting in the approval of gene therapies for human treatment. However, successful therapies remain limited due to a number of challenges such as inefficient cell uptake, low transduction efficiency (TE), limited tropism, liver toxicity and immune response. To adress these issues and increase the number of available therapies, additives from a broad range of materials like polymers, peptides, lipids, nanoparticles, and small molecules have been applied so far. The scope of this review is to highlight these selected delivery systems from a materials perspective.
治疗性病毒基因递送是一项新兴技术，其目的是通过向细胞引入新的遗传信息来纠正基因突变，从而纠正有缺陷的基因或在溶瘤治疗中引发细胞死亡。近年来，重大的科学进展已促成了多项临床试验，从而使基因疗法得以获批用于人类治疗。然而，由于一些挑战，如细胞摄取效率低下、转导效率（TE）低、靶向性有限、肝脏毒性和免疫反应，成功的疗法仍然有限。为了解决这些问题并增加可用疗法的数量，迄今为止已应用了来自多种材料（如聚合物、肽、脂质、纳米颗粒和小分子）的添加剂。本综述的范围是从材料角度突出这些选定的递送系统。
