Skin gene therapy for acquired and inherited disorders

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Show simple item record Carretero Trillo, Marta Escámez Toledano, María José Prada, F. Mirones, I. García Díez, Marta Holguin Fernandez, Almudena Duarte Gonzalez, Blanca Jorcano Noval, José Luis Podhajcer, O. Larcher Laguzzi, Fernando Río Nechaevsky, Marcela del 2021-11-10T10:13:28Z 2021-11-10T10:13:28Z 2006-11
dc.identifier.bibliographicCitation Carretero, M., Escámez, M.J., Prada, F., Mirones, I., García, M., Holguín, A., Duarte, B., Podhajcer, O., Jorcano, J. L., Larcher, F. & Del Río, M. (2006). Skin gene therapy for acquired and inherited disorders. Histology and Histopathology, 21(11), 1233-1247.
dc.identifier.issn 0213-3911
dc.description.abstract The rapid advances associated with the Human Genome Project combined with the development of proteomics technology set the bases to face the challenge of human gene therapy. Different strategies must be evaluated based on the genetic defect to be corrected. Therefore, the re-expression of the normal counterpart should be sufficient to reverse phenotype in single-gene inherited disorders. A growing number of candidate diseases are being evaluated since the ADA deficiency was selected for the first approved human gene therapy trial (Blaese et al., 1995). To cite some of them: sickle cell anemia, hemophilia, inherited immune deficiencies, hyper-cholesterolemia and cystic fibrosis. The approach does not seem to be so straightforward when a polygenic disorder is going to be treated. Many human traits like diabetes, hypertension, inflammatory diseases and cancer, appear to be due to the combined action of several genes and environment. For instance, several wizard gene therapy strategies have recently been proposed for cancer treatment, including the stimulation of the immune system of the patient (Xue et al., 2005), the targeting of particular signalling pathways to selectively kill cancer cells (Westphal and Melchner, 2002) and the modulation of the interactions with the stroma and the vasculature (Liotta, 2001; Liotta and Kohn, 2001).
dc.description.sponsorship Our work is supported by grants SAF-2004-07717 from Ministerio de Ciencia y Tecnología (Spain) and LSHG-512073 from UE to M. Del Rio, LSHG-503447 from UE to J.L. Jorcano and LSHG-512102 from UE to F. Larcher. We express our gratitude to Dr. Y. Gache, Dr. F. Spirito and Dr. G. Meneguzzi for providing EM pictures to illustrate this work.
dc.format.extent 15
dc.language.iso eng
dc.publisher Universidad de Murcia
dc.rights © The authors, 2006.
dc.rights Atribución 3.0 España
dc.subject.other Skin
dc.subject.other Gene therapy
dc.subject.other Tissue engineeering
dc.title Skin gene therapy for acquired and inherited disorders
dc.type article
dc.subject.eciencia Biología y Biomedicina
dc.rights.accessRights openAccess
dc.type.version publishedVersion
dc.identifier.publicationfirstpage 1233
dc.identifier.publicationissue 11
dc.identifier.publicationlastpage 1247
dc.identifier.publicationtitle Histology and Histopathology
dc.identifier.publicationvolume 21
dc.identifier.uxxi AR/0000012939
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