Genetic prodrug activation therapy shows promise as a therapeutic opti
on for the treatment of cancer as well as a variety of other diseases.
it involves the insertion of a gene encoding a drug-metabolizing enzy
me into cells and the systemic administration of a prodrug. The prodru
g is converted to a cytotoxic agent by the action of the expressed enz
yme. To ensure that the enzyme is only expressed in the targeted subse
t of cells, the transcriptional apparatus of a gene that is unique to
this subset is used to regulate the gene encoding the drug-metabolizin
g enzyme. As with all types of gene therapy, one of the major obstacle
s to successful clinical treatment is the development of safe and effe
ctive gene delivery systems.