Skin gene therapy for acquired and inherited disorders
M. Carretero1, M.J. Escámez1, F. Prada1, I. Mirones1, M. García1, A. Holguín1, B. Duarte1, O. Podhajcer2, J.L. Jorcano1, F. Larcher1 and M. Del Río1
1Regenerative Medicine Unit and Cutaneous Diseases Modeling Unit, Epithelial Biomedicine Division, Basic Research Department, CIEMAT, Madrid, Spain and 2Leloir Institute, University of Buenos Aires-Consejo Nacional de Investigaciones Científicas y Técnicas, Buenos Aires, Argentina
Offprint requests to: Dr. Marcela del Rio, Regenerative Medicine Unit, Epithelial Biomedicine Division, Basic Research Department, CIEMAT, Av. Complutense 22 edificio 7, 28040 Madrid, Spain. e-mail: marcela.delrio@ciemat.es
Summary. 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). Histol Histopathol 21, 1233-1247 (2006)
Key words: Skin, Gene therapy, Tissue engineeering
DOI: 10.14670/HH-21.1233