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Gene
therapy is an
approach to treating potentially fatal or disabling diseases by modifying the
expression of an individual's genes toward a therapeutic goal. The premise of
gene therapy is based on correcting disease at the DNA level and compensating
for the abnormal genes.
Replacement gene therapy
replaces a mutated or missing gene, most often a tumor suppressor gene, with a
normal copy of that gene which serves to keep cell growth and division under
control. The p53 gene, the most common gene mutated in cancer has become a
prime target for gene replacement, and has met with some success in inhibiting
cell growth, inhibiting angiogenesis (the development of a tumor's blood
supply), and inducing apoptosis (cell death).
Knockout gene therapy
targets the products of oncogenes (a gene that can induce tumor formation) in
an effort to render them inactive and reduce cell growth.
With constantly expanding knowledge of the genes associated with cancer, their
functions, and the delivery systems used in administering these genes, gene
therapy has a promising future.
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