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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 investigated as a non-viral vector because it has several advantages such as biocompatibility, biodegradability and low toxicity with high cationic potential. However, low specificity and low transfection efficiency of chitosan need to be overcome prior to clinical trial. In this review, we focused on the specific ligand modification of chitosan for enhancement of cell specificity and transfection efficiency via receptor-mediated endocytosis in vitro and in vivo.
机译:开发有效的靶向基因递送系统进入细胞是基因治疗发展的重要策略。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。并且这也提供了将治疗性质粒选择性递送至特定组织的可能性。壳聚糖已被研究为非病毒载体,因为它具有多种优势,例如生物相容性,生物降解性和具有高阳离子电位的低毒性。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。在这篇综述中,我们集中于壳聚糖的特异性配体修饰,通过体外和体内受体介导的内吞作用增强细胞特异性和转染效率。

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