Michael S I, Curiel D T
Department of Biochemistry and Molecular Genetics, University of Alabama at Birmingham 35294, USA.
Gene Ther. 1994 Jul;1(4):223-32.
Gene transfer to eukaryotic cells may be accomplished by capitalizing on endogenous cellular pathways of macromolecular transport. In this regard, molecular conjugate vectors have been developed which deliver DNA via the receptor-mediated endocytosis pathway. An attractive feature of this vector system is the potential to achieve targeted gene delivery based upon flexible incorporation of a targeting ligand. In this review we describe steps that have been taken to optimize this vector system. Specific strategies include the incorporation of mechanisms to achieve conjugate escape from the endosome and the derivation of methods to eliminate sources of nonspecificity. These developments have demonstrated the potential to construct a vector system in which multiple independent components may function in a concerted manner to accomplish targeted high efficiency gene delivery. In their present state of development, molecular conjugate vectors may have many potential applications for in vitro use.
基因转移至真核细胞可通过利用内源性细胞大分子转运途径来实现。在这方面，已经开发出分子偶联载体，其通过受体介导的内吞作用途径递送DNA。该载体系统的一个吸引人的特点是，基于靶向配体的灵活掺入，有实现靶向基因递送的潜力。在本综述中，我们描述了为优化该载体系统所采取的步骤。具体策略包括引入使偶联物从内体逃逸的机制以及衍生出消除非特异性来源的方法。这些进展证明了构建一种载体系统的潜力，其中多个独立组件可以协同发挥作用，以实现靶向高效基因递送。就其目前的发展状态而言，分子偶联载体可能在体外使用中有许多潜在应用。
