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Cystic Fibrosis And Gene Therapy. Essay

1337 words - 5 pages

Contents1 Summary2 Cystic Fibrosis3 Gene Therapy4 Gene Therapy and Cystic Fibrosis5 Problems With Gene Therapy6 Recent Developments in Gene Therapy7 Ethical Issues Related to Gene therapy8 ConclusionSummaryGenes, which are carried on chromosomes, are the basic physical and functional units of heredity. The amount of information geneticists have discovered about genes in the last 20 years is staggering. It was only in 1973 that the first recombinant DNA methods were successfully used. Since then development of techniques that allow geneticists to locate the position of genes has led to a greater understanding of the causes of genetic disorders. Now scientists are able to consider treating people with genetic disorders by introducing normal genes into body cells. Gene therapy has been successfully used in a few cases and many more genetic disorders are currently being investigated. The future looks promising.The aim of this report is to examine Cystic Fibrosis as a genetic disorder, how it affects the lives of those born with it and to investigate gene therapies associated with it. This report also investigates the ethical issues and recent developments in gene therapy as well as the factors that have kept it from becoming an effective treatment for genetic disorders.All of the information presented in this report has been obtained from internet websites and various school textbooks.Cystic FibrosisCystic Fibrosis is an incurable hereditary disorder that causes the body to produce an abnormally thick, sticky mucus that clogs the pancreas and the lungs, leading to problems with breathing and digestion, infection, and ultimately, death. The disease is characterised by a defect in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. This results in irregular chloride and sodium ion conductance in epithelial cells of a number of different organ systems. (Oxford University of Gene Medicine, 2000, http://users.ox.ac. uk/~genemed/) This means that it prevents salts from moving through body tissues properly, disrupting digestion and more importantly leading to chronic lung infection.Cystic fibrosis is a recessive genetic disorder and is the most common life-threatening genetic illness in the majority of Caucasian countries. If both parents are carriers of the defective cystic fibrosis gene, their child has a 25 percent chance of inheriting both those genes and developing the disease (see appendix fig 1). If only one abnormal gene is inherited (that is, from only one parent), the child will not have symptoms of the disease but will be capable of passing on the defective gene to the next generation. One in 40 Caucasian people are healthy carriers of the cystic fibrosis gene. Three decades ago most babies born with cystic fibrosis died in early childhood, but advances in diagnosing and treating the disease have significantly improved the chances of survival.Gene TherapyGene therapy is essentially the removing and replacing of a particular gene...

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