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Cancer treatment has been the major goal of the gene therapy studies over the decades. Although there is no cancer gene therapy drug in the market yet, substantial progress has been made in defining potential targets and in developing viral and nonviral gene delivery systems recently. Numerous genes have been studied as the targets for cancer gene therapy so far. Various gene therapy strategies, including suicide gene therapy, oncolytic viral therapies, antiangiogenesis, and gene therapy vaccines have been developed. The combination of gene therapy with conventional methods, such as chemotherapy, radiotherapy, and immunotherapy, has further improved the therapeutic efficacy. Although the preclinical and experimental studies have yielded highly encouraging results, there are still few gene therapy agents at phase III trials. In the current chapter, we will review gene transfer systems, targets, gene targeting strategies, and cancer gene therapy in the clinic.

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