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Gene therapy - Essay Example

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Gene Therapy, Safety Concerns and Improving Safety Methods 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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Download file to see previous pages Currently, research is going on for gene therapy by various approaches in different parts of the world for various diseases like combined immuno-deficiencies, hemophilia, Parkinson's disease, cancer and even HIV (Verma and Weitzman, 2005). Though gene therapy has been under research for the past 2 decades, no single gene therapy has been approve for clinical use. The main reason for this is safety concerns pertaining to gene therapy trials and treatment. In this essay, various strategies to increase the safety of gene therapy will be discussed through review of suitable literature. 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, 2004). The cells used thus 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. After this, the cells are injected into the patient’s body by vein. In in vivo therapy, gene delivery is done in the cells that are still in the body. The simplest method of introducing therapeutic genetic material into the cells is direct introduction into target cells. However, this method is not practical because it can be used only with certain tissues and requires large amounts of DNA. Other methods are there wherein the genes are delivered into cells by vectors like viruses or bacteria, by electroporation or tiny synthetic "envelopes" of fat molecules (Hecht, 2004). Of these, the most commonly used vector is virus. Vector is the carrier of the gene. Viruses are used as vectors to deliver genetic material to the nucleus of the cell that contains its DNA. The natural ability of a virus to enter a cell is used for this purpose. The viruses used for gene therapy are retrovirus, adenovirus, adeno-associated virus and herpes simplex virus (Genetics Home Reference, 2008). While preparing the vectors for gene therapy, the DNA coding for a part or whole of the normal genes of the virus to be used as a carrier is removed and replaced with the treatment gene. The carriers are engineered in such a way that there ability to enter the cells is not lost but they cannot reproduce. Genes delivered by tiny synthetic "envelopes" of fat molecules enter the cell by cell membrane which has very high concentration of fat molecules. In electroporation, the genes are delivered into the cells by creating tiny openings in the cell membrane. This is done by using a bionic chip. The chip contains a single living cell embedded in a tiny silicon circuit (Hecht, 2004). Another method of gene therapy is where the therapeutic gene gets inside the target cell by chemically linking the DNA to a molecule that will bind to special cell receptors. After binding to these receptors, the DNA is engulfed by the cell membrane and passed into the interior of the target cell. However, this is less effective than the other methods (Genetics Home R ...Download file to see next pagesRead More
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