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Dadun > Depósito Académico > CIMA (Centro de Investigación Médica Aplicada) > Área de Terapia génica y Hepatología > Inmunología experimental > DA - CIMA - Terapia génica y Hepatología - Inmunología experimental - Artículos de revista >

Cells as vehicles for therapeutic genes to treat liver diseases
Authors: Prieto, J. (Jesús)
Fernandez-Ruiz, V. (Verónica)
Kawa, M.P. (MP)
Sarobe, P. (Pablo)
Qian, C. (Cheng)
Keywords: Cancer
Liver diseases
Progenitor cells;
EPC
Issue Date: 2008
Publisher: Nature Publishing Group
Publisher version: http://www.nature.com/gt/journal/v15/n10/full/gt200844a.html
ISSN: 1476-5462
Citation: Prieto J, Fernandez-Ruiz V, Kawa MP, Sarobe P, Qian C. Cells as vehicles for therapeutic genes to treat liver diseases. Gene Ther 2008 May;15(10):765-771.
Abstract
Gene therapy involves the transfer of genetic sequences to tissues to obtain a curative effect. Effective gene transfer can be achieved by introducing the therapeutic gene into virus-like particles that facilitate the penetration of the transgene into the cells. However, direct injection of viral vectors may activate innate immunity leading to toxic effects. On the other hand, viral vectors frequently induce neutralizing antibodies, which limit the efficacy of repeated vector administration. Moreover, targeting of the transgene to the desired tissue is a goal that not always can be attained with current vectors. The use of cells as vehicles for therapeutic genes may offer solutions for these issues. Ex vivo transduction of specific cells with vectors encoding therapeutic genes followed by injection of the engineered cells to the patient will reduce the inherent toxicity of the vector while preventing the development of neutralizing antibodies. At the same time, this therapeutic approach can take advantage of the homing properties of the transduced cells to target transgene expression to the sites of interest. Thus, it has been shown that administration of dendritic cells engineered ex vivo with vectors encoding selected antigenic determinants or immunostimulatory molecules is an efficient means to elicit protective immune responses. Similarly, since endothelial progenitor cells (EPC) move to inflammed, ischemic or neoplastic tissues, the injection of EPC transduced ex vivo with appropriate therapeutic genes is an effective method to direct transgene expression to the lesions to be treated. Promising data in animal models of disease point to a future clinical application of this therapeutic strategy.
Permanent link: http://hdl.handle.net/10171/21670
Appears in Collections:DA - Medicina - Medicina Interna - Artículos de revista
DA - CIMA - Terapia génica y Hepatología - Inmunología experimental - Artículos de revista

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