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Biology 100 � Section 005 Gene Therapy�s Potential in Curing Genetic Disease

Exploration of gene therapy's role in treating genetic disorders.

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    1 Biology 100 – Section 005 November 25, 2013 Gene Therapy’s Potential in Curing Genetic Disease Based on the article Gene Therapy’s Potential in Curing Genetic Disease , critically analyze the progress and challenges of gene therapy in treating genetic diseases. Discuss the potential benefits of somatic gene therapy, including its successes and limitations. Additionally, evaluate the ethical concerns surrounding germ - line gene therapy and its implications for future generations. Your answer should provide a balanced assessment of the scientific, medical, and societal considerations involved in gene therapy, and should be between 600 - 800 words in length.

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    2 Gene Therapy’s Potential in Curing Genetic Disease The completion of the Human Genome Project has opened the doors leading to seemingly unlimited scientific possibilities. With a correct understanding of DNA, scientists can identify specific sections that code for positive (or negative) results; they can enhance or duplicate the positive sections and try to remove the ‘errors’. Genetically modified organisms (GMO) typically refer to animals or food , but soon it could become commonplace for it to refer to humans. Scientists have had great success in using genetics to improve the quality (and often the quantity) of food that humans eat on a daily basis. Although controversial, many advocate using this same technology on humans, particularly to cure them of disabling, genetic diseases. Despite the dangers posed by human genetic modification, gene therapy has the potential to cure the genetic diseases that plague human ity. This report will cover the history of human gene therapy , its poten tial for curing disease, and the dangers presented by germ - line gene therapy. Human application of gene therapy was conceptualized in the early 1970s, by Theodore Friedmann and Richard Roblin (1) . They recognized the potential of gene therapy, but cautioned that they did not have enough information to begin human testing. They proposed that more research be done to make this a safe and effective treatment, and also advocated “ formulating a complete set of ethicoscientific criteria to guide the development and clinical application of gene therapy techniques” (1). It was almost twenty years later that the FDA approved the first gene therapy case in the United States. Dr. William French Anderson oversaw the treatment of a four year - old girl, Ashanti DeSilva, with ADA - SCID (Severe Combined Immunodeficiency); this genetic disease

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