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gene therapy guide

GeneTherapy
By CY
Gene therapy is the process of correcting defective genes that cause diseases. This type of therapy is still in the early stages. No gene therapy products have been approved by the Food and Drug Administration (FDA) for legal sale. Gene therapy trials continue, but the success rate has not been very high, which is most of the reason why the FDA will not yet approve it.
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The first gene therapy trial took place in 1990, depending on volunteers to take parting the trial. In 1999, the therapy was thrust into the negative spotlight when a 18 year old died of organ failure just 4 days after starting treatment. His death was the result of a severe immune system reaction to the trial drug. Trials continued until 2003, when the FSA banned the use of retroviral vectors in blood stem cells. This decision was made on the basis of at least 2 people in the trial developing a condition similar to leukemia. While both individuals were successfully treated for it, trials on humans have not occurred since.
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There are several ways gene therapy is done. A



normal gene can be inserted into a location in the body and replace a specific gene. This is the most commonly used method of gene therapy. The next option is replacing a damaged gene through a process called homologous recombination. This process involves knocking out one gene by disrupting its function, and then replace it with one that works properly and has the correct genetic coding.
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Abnormal genes can be repaired by selective reverse mutation. This involves selecting particular genes and processing them back to their original form. The last option is simply to keep a gene, but alter it to meet the specifications.
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Ethical issues regarding gene therapy continue to fuel. The biggest question is what a disability is, what is normal, and who gets to decide the criteria. Cost is another big issue. Such therapy is very expensive and it is debated who will pay for it. As the technology in the area of genes continues, gene therapy may or may not become a reality for those with disabilities and illnesses linked to ineffective genes.

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