Publication: Skin gene therapy for acquired and inherited disorders
Authors
Carretero, M. ; Escámez, M.J. ; Prada, F. A. ; Mirones, I. ; García, M. ; Holguín, A. ; Duarte, B. ; Podhajcer, O. ; Jorcano, J.L. ; Larcher, F. ; Del Río, M.
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Publisher
Murcia : F. Hernández
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DOI
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info:eu-repo/semantics/article
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).
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