PDF(Pubmed)
Abstract：
Gene delivery is a complex process with several challenges when attempting to incorporate genetic material efficiently and safely into target cells. Some of the key challenges include not only efficient cellular uptake and endosomal escape to ensure that the genetic material can exert its effect but also minimizing the toxicity of the delivery system, which is vital for safe gene delivery. Of importance, if gene delivery systems are intended for biomedical applications or clinical use, they must be scalable and easy and affordable to manufacture to meet the demand. Here, we show an efficient gene delivery method using a combination of carbon dots coated by PEI through electrostatic binding to easily generate cationic carbon dots. We show a biofunctional approach to generate optimal cationic carbon dots (CCDs) that can be scaled up to meet specific transfection demands. CCDs improve cell viability and increase transfection efficiency four times over the standard of PEI polyplexes. Generated CCDs enabled the challenging transfection protocol to produce retroviral vectors via cell cotransfection of three different plasmids into packing cells, showing not only high efficiency but also functionality of the gene delivery, tested as the capacity to produce infective retroviral particles.
摘要：
基因递送是一个复杂的过程，当试图将遗传物质有效和安全地整合到靶细胞中时，有几个挑战。一些关键挑战不仅包括有效的细胞摄取和内体逃逸，以确保遗传物质能够发挥其作用，而且还将递送系统的毒性降至最低。这对于安全的基因传递至关重要。重要的是,如果基因递送系统旨在用于生物医学应用或临床用途，它们必须是可扩展的，易于制造和负担得起的，以满足需求。这里,我们展示了一种有效的基因递送方法，该方法使用通过静电结合由PEI涂覆的碳点的组合来容易地产生阳离子碳点。我们展示了一种产生最佳阳离子碳点（CCD）的生物功能方法，该方法可以扩大规模以满足特定的转染需求。与PEI复合物的标准相比，CCD提高了细胞活力并将转染效率提高了四倍。产生的CCD使具有挑战性的转染方案能够通过三种不同质粒的细胞共转染到包装细胞中来产生逆转录病毒载体。不仅显示出高效的基因传递功能，作为产生感染性逆转录病毒颗粒的能力进行测试。
