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Abstract

Cancer is the product of a multistep process involving an accumulation of genetic alterations in somatic cells [268]. Advances in the understanding of the molecular basis of neoplasia and host—tumor relationships have resulted in new cancer therapy strategies with translation into clinical trials. Gene therapy has emerged as a treatment strategy which relies on the introduction of genetic material to cure, slow the progression, or possibly even prevent a variety of diseases including genetic, infectious (HIV in particular), cardiovascular, and arthritic diseases [12, 83]. Gene therapy has shown potential for the treatment of cancer. A number of cancer gene therapy approaches have been developed based on direct correction of genetic lesions. These ‘mutation compensation’ approaches, designed to correct the molecular lesions underlying neoplastic transformation and progression, have demonstrated efficacy in the context of in-vitro and preclinical model systems. Delivery of therapeutic genes to every cancer cell is beyond the capabilities of current vectors. The efficiency and accuracy of gene delivery remain the most significant barriers to the success of cancer gene therapy [262]. However, preclinical studies have demonstrated approaches that may have promise in the clinical setting. The approaches that have been explored include genetic immunopotentiation with cytokine gene transfer and tumor cell vaccination, molecular chemotherapy with prodrug activation by suicide genes or an increase in tumor cell sensitivity to chemotherapy or radiation therapy, protection of bone marrow cells from the toxic effects of chemotherapeutic drugs, inhibition of activated oncogenes by antisense treatment, transfer of tumor suppressor genes, and pro-apoptotic gene therapy. Each of these approaches have been translated into clinical cancer therapy trials [43, 83].

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Buchsbaum, D.J., Miller, C.R., Mahasreshti, P.J., Curiel, D.T. (2003). Cancer gene therapy. In: Oldham, R.K. (eds) Principles of Cancer Biotherapy. Springer, Dordrecht. https://doi.org/10.1007/978-94-017-2757-0_19

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