CONICET | Buscador de Institutos y Recursos Humanos

Book- VIRAL GENOME Chapter: "GENE DELIVERY SYSTEMS". Pereyra Andrea MD. and Herenu Claudia PhD. Institute for Biochemical Research-Histology B-Pathology B, Faculty of Medicine, National University of La Plata, La Plata, Argentina. Send correspondence to: Claudia Herenu INIBIOLP. Faculty of Medicine, UNLP CC 455 1900 La Plata Argentina tel: (54-221) 425-6735 fax: (54-221) 425-8988 The present chapter will be focused on different gene delivery systems for Gene therapy approaches with the purpose of the insertion of genetic materials into an individual?s cells and/or tissues to treat diseases. By correcting genetic defects causing diseases via genome manipulation, gene therapy can truly revolutionize medical intervention for treating monogenetic inherited diseases, acquired diseases or polygenetic conditions. The foreign genetic material can be administered in vivo, ex vivo or in vitro depending on the nature of a disease. A successful gene therapy system must perform several functions. In all cases, the therapeutic gene must first be delivered across the cell membrane, which is a significant barrier. Once delivered inside the cells, the therapeutic gene may exist episomally or be integrated into the host chromosome depending on the nature of the gene transfer vector. Moreover, as gene delivery system, is an important issue the replication and segregation of the therapeutic gene during cell division to maintain long-lasting therapeutic gene expression. These specifications will be discussed for each gene delivery system along the chapter. Current viral vector systems will be detailed in this chapter: adenovirus, retrovirus, adeno-associate and herpes virus. Non-viral vector systems will be included, such as naked DNA, plasmids, DNA transposons and the novel technology of magnetic nano-particles which have been actively developed as additional tools for crossing the cell membrane or a combination of these for gene delivery.