Abstract

GENE THERAPY FOR CANCER

*Chate Satyabhama Vitthal (B. Pharm) and Mr. L. D. Hingane (PhD Scholar)

Abstract

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 Gene therapy can be broadly defined as the transfer of genetic material to cure a disease or at least to improve the clinical status of a patient. One of the basic concepts of gene therapy is to transform viruses into genetic shuttles, which will deliver the gene of interest into the target cells. Safe methods have been devised to do this, using several viral and non-viral vectors. Two main approaches emerged: in vivo modification and ex vivo modification. Retrovirus, adenovirus, adenoassociated virus are suitable for gene therapeutic approaches which are based on permanent expression of the therapeutic gene. Non-viral vectors are far less efficient than viral vectors, but they have advantages due to their low immunogenicity and their large capacity for therapeutic DNA. To improve the function of non-viral vectors, the addition of viral functions such as receptor mediated uptake and nuclear translocation of DNA may finally lead to the development of an artificial virus. Gene transfer protocols have been approved for human use in inherited diseases, cancers and acquired disorders. Although the available vector systems are able to deliver genes in vivo into cells, the ideal delivery vehicle has not been found. Thus, the present viral vectors should be used only with great caution in human beings and further progress in vector development is necessary.

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