Volpers Christoph, Kochanek Stefan
Center for Molecular Medicine (ZMMK) and Institute for Genetics, University of Cologne, Kerpener Str. 34, 50931 Cologne, Germany.
J Gene Med. 2004 Feb;6 Suppl 1:S164-71. doi: 10.1002/jgm.496.
Due to the very efficient nuclear entry mechanism of adenovirus and its low pathogenicity for humans, adenovirus-based vectors have become gene delivery vehicles that are widely used for transduction of different cell types, especially for quiescent, differentiated cells, in basic research, in gene therapy applications, and in vaccine development. As an important basis for their use as gene medicine, adenoviral vectors can be produced in high titers, they can transduce cells in vivo with transgenes of more than 30 kb, and they do not integrate into the host cell genome. Recent advances in the development of adenoviral vectors have brought considerable progress on issues like target cell specificity and tropism modification, long-term expression of the transgene, as well as immunogenicity and toxicity in vivo, and have suggested that the different generations of non-replicative and replicative vectors available today will each suit best for certain applications.
由于腺病毒高效的核进入机制及其对人类的低致病性,基于腺病毒的载体已成为基因传递工具,广泛用于不同细胞类型的转导,特别是在基础研究、基因治疗应用和疫苗开发中用于静止、分化细胞的转导。作为其用作基因药物的重要基础,腺病毒载体可高滴度生产,能将超过30kb的转基因在体内转导至细胞,且不会整合到宿主细胞基因组中。腺病毒载体开发的最新进展在诸如靶细胞特异性和嗜性修饰、转基因的长期表达以及体内免疫原性和毒性等问题上取得了相当大的进展,并表明当今可用的不同代非复制型和复制型载体各自最适合某些应用。