The ability to introduce genetic material into target cells has tremendous potential as a novel means for disease treatment and correction. While gene therapy is still in clinical trials, the past 20 years has seen the development of a large armamentarium of plasmid and viral vectors for human use. Initial trials focused on the correction of genetic diseases, but vectors are now available to deliver pharmacologic agents, reengineer cells, or express ribozyme and inhibitory RNAs that downregulate gene expression. Currently the majority of clinical trials have been in cancer. Approaches directed at malignancies include reengineering of autologous T cells to recognize tumor antigens and eliminate cancer cells, introduction of DNA repair genes into hematopoietic cells to protect them from chemotherapy and foster dose escalation, and introduction of immune stimulatory molecules to foster cancer cell recognition by the immune system. This chapter provides an introduction to gene therapy by illustrating the technology and discussing current clinical applications.
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