Gene Therapy - Research Paper Example

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Gene Therapy: Problems and Solutions to Problems Gene therapy is a method of treating various acquired and genetic diseases with genes. This is an experimental technique and is done by inserting genetic material into the patient’s cells. In this form of treatment, the diseases are either treated or prevented by replacing abnormal gene, or by introducing the absent gene or by supplementing the defective gene (Genetics Home Reference, 2008)…
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Gene Therapy
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p.). Currently, research is going on for gene therapy by various approaches in different parts of the world for various diseases like combined immune deficiencies, hemophilia, Parkinson's disease, cancer and even HIV (Verma and Weitzman, n. p.). Though gene therapy has been under research for the past 2 decades, no single gene therapy has been approved for clinical use. The main reason for this is safety concerns pertaining to gene therapy trials and treatment. In this essay, problems associated with gene therapy and strategies to prevent those problems will be reviewed. Safety issues related to gene therapy are mainly related to the methods employed in gene therapy. There are basically 2 methods of gene therapy and they are ex-vivo therapy and in-vivo therapy. In ex-vivo therapy, gene delivery is done in cells after being removed from the body (Hecht, n. p.). The cells used are basically grown in the laboratory. The cells are than modified outside the body and then transplanted back into the body. In some research trials, cells from blood or born marrow are taken out and cultured in a laboratory. Thereafter, the cells are exposed to the virus with the desired gene. The virus infects the cells and transfers the therapeutic genetic material into the nucleus of the cells. ...
In hemophilia and cancer treatment, the gene is directly injected into the tumor (Hecht, n. p.). Viral vectors can introduce toxicity, immune and inflammatory responses. Also, though the viruses are engineered to be innocuous, there is always a fear that they may cause disease (Hecht, n. p.). Immunogenicity is an important factor with regard to safety of the patient subjected to gene therapy trial. The immunogenicity is that of the vector and it is derived from the proteins of the virus and the capability of the viruses to infect macrophages and dendrite cells. Such an immune response can result in elimination of the transduced cells and also the vector (Hecht, n. p.). The potency and dose are very important aspects for good outcomes in gene therapy and these are basically linked to the production process and also the purification process (Raty et al., n. p.). These processes maximize the infective virus and decrease the number of defective viruses or empty viral particles. Using administration methods to increase the concentration of the virus locally helps in decreasing the side effects and also aids in lowering the amount of total dose that is administered into the patient. In the ex-vivo approach, cells of the patient that are outside the body like skin can be used for transduction for augmentation of the grafting of the tissue and also for providing therapeutic protein secretion. These methods help in limiting the exposure to the patient to vectors of gene delivery, thus increasing safety aspects. These methods are however, laborious. Another method of decreasing the amount of dose administered to the person is by combining lipoproteins of low density with avidin (Raty et al., n. p.). In this method, substances that Read More
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Excellent paper! Used it to finish an assignment for a biology course. It was easy as ABC, for the first time in my life.
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