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Receptor-Mediated Gene Delivery Using Chitosan Derivatives In Vitro and In Vivo

机译:使用壳聚糖衍生物进行体内和体外受体介导的基因传递

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The development of an efficient targeted gene delivery system into cells is an important strategy for the advancement of gene therapy. The targeted gene delivery system is especially important in non-viral gene transfer which shows the relative low transfection efficiency. And it also opens the possibility of selective delivery of therapeutic plasmids to specific tissues. Chitosan has been considered to be a good candidate for gene delivery system, since it is already known as a biocompatible, biodegradable, and low toxic material with high cationic potential. However, low specificity and low transfection efficiency of chitosan need to be overcome prior to clinical trial. In this study, we focused on the chemical modification of chitosan for enhancement of cell specificity and transfection efficiency.
机译:开发有效的靶向基因递送系统进入细胞是促进基因治疗的重要策略。靶向的基因递送系统在非病毒基因转移中表现出相对较低的转染效率,尤其重要。并且也为选择性地将治疗性质粒递送至特定组织提供了可能性。壳聚糖被认为是基因传递系统的良好候选者,因为它已经被公认为具有高阳离子潜力的生物相容性,可生物降解性和低毒性材料。然而,在临床试验之前需要克服壳聚糖的低特异性和低转染效率。在这项研究中,我们专注于壳聚糖的化学修饰,以增强细胞特异性和转染效率。

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