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

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1Biology 100–Section 005November 25, 2013Gene Therapy’s Potential in Curing Genetic DiseaseBased on the articleGene Therapy’s Potential in Curing Genetic Disease, critically analyzetheprogress and challenges of gene therapy in treating genetic diseases. Discuss the potentialbenefits of somatic gene therapy, including its successes and limitations. Additionally, evaluatethe ethical concerns surrounding germ-line gene therapy and its implications for futuregenerations. Your answer should provide a balanced assessment of the scientific, medical, andsocietal considerations involved in gene therapy, and should be between 600-800 words inlength.

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2Gene Therapy’s Potential in Curing Genetic DiseaseThe completion of the Human Genome Project has opened the doors leading toseemingly unlimited scientific possibilities. With a correct understanding of DNA, scientists canidentify specific sections that code for positive (or negative) results; they can enhance orduplicate the positive sections and try to remove the ‘errors’.Genetically modified organisms(GMO) typically refer toanimals or food, but soon it could become commonplace for it to referto humans. Scientists have had great success in using genetics to improve the quality (and oftenthe quantity) of food thathumanseat on a daily basis. Although controversial, many advocateusing this same technology on humans, particularly to cure them of disabling,genetic diseases.Despite the dangers posed by human genetic modification, gene therapy has thepotential to cure the genetic diseases that plague humanity. This report will coverthe history ofhumangene therapy, its potential for curing disease, andthe dangers presented by germ-linegene therapy.Human application of gene therapy was conceptualized in the early 1970s, by TheodoreFriedmann and Richard Roblin(1).They recognized the potential of gene therapy, butcautioned that they did not have enough information to begin human testing. They proposedthat more research be done to make this a safe and effectivetreatment,and alsoadvocated“formulatinga complete set of ethicoscientific criteria to guide the development and clinicalapplication of gene therapy techniques” (1).It was almost twenty years later that the FDA approved the first gene therapy case inthe United States.Dr. William French Anderson oversaw thetreatmentof a four year-old girl,Ashanti DeSilva, with ADA-SCID (Severe Combined Immunodeficiency); this genetic disease

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