Advantages and Disadvantages of Gene Therapy

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Recommended: The ethics of gene therapy balancing the risks review

Considered one of the more controversial up comings in the world of science, it’s hard to ignore what amazing benefits gene therapy has to offer. With many potential treatments aimed to target some rather serious diseases, much support has been gained for the field. Although gene therapy remains at the forefront of change, much discussion has been created due to several potential drawbacks. These drawbacks, however substantial, must not be overlooked for the risk of lives isn’t a fair price to pay for innovation of healthcare reform.

Gene therapy is used as a way to treat or minimalize defective genes that lead to or cause diseases. Genes are hereditary units which are base sequences that provide the encoding instruction of a protein. Different proteins are made depending on what the specific amino acid sequence had encoded. Proteins can be different in size, shape, and function. Many times in the event of a mutation, genes become transformed so that the protein supposed to be encoded either isn’t produced or is made with inhibiting functionality, thus resulting in a potential genetic disorder. Therapy for this usually involves the implementation of new genes in cells to take the place of non-functioning or absent ones. This process involves a virus that is used to carry the genetic information to the patient’s cells. These viruses have been modified to find and detect specific problem target areas. To do this, scientists many times will use viral vectors, or vehicles that are used as the form of transportation for the virus.

Avoiding toxicity in the target cell is the elemental key in order for gene therapy to be successful. Certain viral vectors have proven to perform better than others with this. Retroviral vectors use ...

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...e in the Twenty-first Century. New York: Oxford University Press, 1999.

Johnston, Josephine, and Françoise Baylis. (2004). "What Happened to Gene Therapy? A Review of Recent Events." Clinical Researcher 4: 11-15.

Leiden, Jeffrey M., (2000). “Human Gene Therapy: The Good, the Bad, and the Ugly.” Circulation Research 86:923-925.

National Reference Center for Bioethics Literature (NRCBL). (2002). "Scope Note 24: Human Gene Therapy." Available from http://www.georgetown.edu/research/nrcb l/publications/scopenotes/.

Orkin, Stuart H., and Arno G. Motulsky. (1995). "Report and Recommendations of the Panel to Assess the NIH Investment in Research on Gene Therapy." National Institutes of Health, 1995. Available from www.nih.gov/news/panelrep.html.

Verma, Inder M., and Nikunj Somia. (1997). “Gene therapy – promises, problems and prospects.” Nature 389: 239-242.

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