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Gene Therapy - Research Paper Example

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Instructor Institution Date Gene Therapy Introduction Gene therapy is the therapeutic use of genes as medication. Gene therapy involves the transfer of a working copy or therapeutic genes into specific cells of a person with an aim of repairing a gen copy that is faulty (Fackelmann 239)…
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Download file to see previous pages During this period, there was the discovery of cell lines which were genetically marked. Additionally, the cell transformation and how it occurred was clarified during this period as well. Developments in gene therapy were motivated by the later arrival of the recombinant DNA techniques. Clone genes were also developed which were valuable in the demonstration of how foreign genes can be used to male corrections to various defects in genes. This also revealed the importance of DNA in correcting the disease phenotypes which emanated from defective cells. Gene transfer methods and efficient retroviral vectors have shown that in vivo and in vitro phenotype correction is an effective gene therapy approaches (Dolly and Hunt 28). This is the reason why gene therapy has been justified and accepted to be applicable in clinical genetic studies which involve human subjects. Gene therapy techniques are likely to have positive impact in future scientific discoveries. This is because of its postulated future use in the replacement of faulty genes and the introduction of new genes for treatment or modification of the clinical direction of a specific medical condition. Technical Aspects on how Gene Therapy Works Genetically altered material is delivered via three major methods. The first method involves retrotransposons or retroviruses (Murray 15). Retroviruses are commonly used due to their ability to transfer their own DNA or genetic information. Additionally retroviruses are able to alter the human gene genetically. Cell DNA has parts which are able to make copies of themselves into other sections within the cell genome (Rudolph and Aslam 44). These parts which are exemplified by the yeast transposon can be used in gene therapy. However the application of transposons in alteration of genes is still under research (Judson 40). Secondly, gene therapy is made possible through blasting DNA or genes by the use of pressurized guns which are filled with helium (Bryant, Duker and Reichel 90). The helium gun is used to fire gold bullets of very small size that are coated with genes which are genetically altered (Garber 60). This technique was performed on rats with tumors in gene therapy experiments. The results revealed that the tumor cells which successfully received the inoculated altered cells demonstrated activation of coding for cytokines which played a role of activation immune cells. The third aspect of gene therapy includes liposomes (Hogan 10). This method is still being investigated where hollow molecules of fat within a solution are used in gene therapy. This methodology is used in attempts to genetically treat cystic fibrosis (Fackelmann 239). Cystic fibrosis is a respiratory tract disease which results from the buildup of chloride ions. In the gene therapy for this disease, liposomes which are coated with genes that are genetically altered are inhaled. The genetically altered genes are designed to prevent chloride ion buildup. The experiments of gene therapy have revealed that the ion levels within the respiratory tract decreased significantly with the inoculation of the genetically modified genes. The advantage of this method is that it poses less potential risk as compared to retroviruses. Positive Aspects of Gene therapy Gene therapy is advantageous because it can be used to treat individuals who have congenital genetic disease. This can be achieved through the replacement of the ...Download file to see next pagesRead More
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