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Gene Therapy
Gene therapy offers a new treatment paradigm for curing human disease. Rather than altering the disease phenotype by using agents who interact with gene products, or are themselves gene products, gene therapy can theoretically modify specific genes resulting in disease cure following a single administration. Initially gene therapy was envisioned for the treatment of genetic disorders, but is currently being studied in a wide range of diseases, including cancer, peripheral vascular disease, arthritis, neurodegenerative disorders and other acquired diseases. Even though the range of gene therapy strategies is quite diverse, certain key elements are required for a successful gene therapy strategy (i.e. cloning). The most elementary of these is that the relevant gene must be identified and cloned. Upon completion of the Human Genome Project, gene availability will be unlimited, but until then the starting point for any gene therapy strategy remains gene identification and cloning for relevant genes related to the disease. Once the gene has been identified and cloned, the next consideration must be expression. Questions pertaining to the efficiency of gene transfer and gene expression remain at the forefront of gene therapy research. Currently many debates in the field of gene therapy revolves around the transfer of desired genes to appropriate cells, and then obtaining sufficient levels of expression for disease treatment. Hopefully, future research on gene transfer and tissue-specific gene expression will resolve these issues in the majority of gene therapy protocols. Other important considerations for a gene therapy strategy include a sufficient understanding of the pathogenesis of the targeted disorder, potential side effects of the gene therapy treatment, and understanding of the target cells to receive the gene therapy. Gene transfer vector is the mechanism by which the gene is transferred into a cell. Currently there are at least 150 clinical gene therapy protocols worldwide. Since the approval process for these protocols is not as public outside the U.S., it is difficult to obtain an exact number of worldwide protocols. Of the publicized protocols, 125 are approved in the United States, 48 in Europe and at least 1 each in China and Japan. As of 31 December 1995, 1024 patients had been treated in either a gene transfer or gene therapy protocol. Much controversy exists regarding how many of these have benefited from their gene therapy, and no one has yet been cured. Public controversy in the field of human gene therapy is driven by several factors. Ordinary citizens as well as scientists easily understand the enormous potential of gene therapy, but the former may not appreciate all the pitfalls and uncertainly that lies in the immediate future. The financial interests of biotechnology firms and, some have asserted, the career interests of some gene therapists have encouraged extravagant, or at least overly optimistic, public statements about contemporary gene therapy. In spite of the proliferation of protocols, the actual number of patients treated remains small, and only one genuinely controlled study of human gene therapy has been published as of this date. References: Anderson WF. Human Gene Therapy. 1995; 6:1505-1506. Lyon J and Gorner P. Altered fates: Gene therapy and the retooling of human life. (New York: W.W. Norton) 1995; 1-569. Mulligan RC. The basic science of gene therapy. Science 1993; 260: 926-932. Wolff J A and Lederberg J. A history of gene transfer and therapy. Chapter 1 in Wolff JA, editor, Gene therapeutics: Methods and applications of direct gene transfer. (Basel: Birkhaeuser) 1994; 3-25. Report and Recommendations of the Panel to Assess the NIH Investment in Research on Gene Therapy Available at: http:/www.nih.gov/news/panelrep.html

 



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