Gene Therapy :: essays papers

Gene TherapyGene therapy offers a new treatment paradigm for curing human disease.Rather than altering the disease phenotype by victimization agents who interactwith agent products, or are themselves constituent products, gene therapy cantheoretically modify specific genes resulting in disease cure followinga single administration. Initially gene therapy was envisioned for thetreatment of genetic disorders, but is currently being studied in awide range of diseases, including cancer, fringy 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 therelevant gene must be identified and cloned. Upon completion of theHuman Genome Project, gene availability will be unlimited, but until then(prenominal) the starting point for any gene therapy strategy remains geneidentif ication and cloning for relevant genes related to the disease.Once the gene has been identified and cloned, the following considerationmust be expression. Questions pertaining to the efficiency of gene designate and gene expression remain at the forefront of gene therapyresearch. Currently many debates in the field of gene therapy revolvesaround the transfer of desired genes to appropriate cells, and thenobtaining sufficient levels of expression for disease treatment.Hopefully, future research on gene transfer and tissue-specific geneexpression will resolve these issues in the majority of gene therapyprotocols. Other important considerations for a gene therapy strategyinclude a sufficient understanding of the pathogenesis of the targeteddisorder, potential side effects of the gene therapy treatment, andunderstanding of the target cells to receive the gene therapy.Gene transfer vector is the mechanism by which the gene is transferredinto a cell. Currently there are at least 150 cli nical gene therapyprotocols worldwide. Since the approval process for these protocols isnot as public orthogonal the U.S., it is difficult to obtain an exactnumber of worldwide protocols. Of the publicized protocols, 125 areapproved in the United States, 48 in Europe and at least 1 each inChina and Japan. As of 31 December 1995, 1024 patients had been treatedin either a gene transfer or gene therapy protocol. Much controversyexists regarding how many of these have benefited from their genetherapy, and no one has yet been cured.Public controversy in the field of human gene therapy is driven byseveral factors. Ordinary citizens as well as scientists easilyunderstand the enormous potential of gene therapy, but the former maynot appreciate all the pitfalls and uncertainly that lies in theimmediate future. The financial interests of biotechnology firms and,