Anti-oncogene Ribozymes for Cancer Gene Therapy

Publisher Summary This chapter focuses on a general overview of ribozymes and their applications for cancer gene therapy. Ribozymes are small RNA molecules that possess specific catalytic activities and are being actively investigated for their therapeutic applications in the field of gene therapy. Ribozymes have the ability to modulate specific gene expression because of their site-specific cleavage activity. Ribozymes can be designed for any disease in which a specific protein has been linked to its etiology, and may offer some advantages over antisense oligonucleotide strategies. Since the discovery of the protooncogene, cancer has been defined as a genetic disease. Therefore, gene therapy could be a rational and promising strategy for the treatment of specific cancers. So, oncogenes are obvious targets for the therapeutic application of anti-oncogene ribozymes. One of the problems for successful gene therapy is to define the role of specific oncogenes in specific tumors. Ribozyme technology can be used to help define and delineate the role of oncogenes in cancer and can be used as a therapeutic agent as well. Extensive studies have investigated the efficacy of antioncogene ribozymes, and have shown successful alteration of the human malignant phenotype in vivo . The appearance of gene therapy as an alternative treatment for cancer and other diseases has led researchers toward the development of efficient delivery systems. Among the first methods developed for gene delivery, viral systems have been potentially the most promising. Although viral transfer shows huge potential for clinical settings, nonviral delivery systems are receiving increasing attention. Thus, effective delivery systems with minimal toxicity may advance ribozymes as important therapeutic modalities in the clinical field. Ribozymes could have an important impact on the field of gene therapy in the near future.