Gene Therapy

Essay by C-DawgHigh School, 10th gradeF, January 1996

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Gene therapy is one of the many new scientific frontiers that has been started up as a result of the continuing research of the human genome project. Gene therapy is a very new field of science, and so the full advantages and disadvantages are unknown. However, it is presumed that this will be the next frontier in medical science.

Gene therapy works in three different ways. The basic idea is that new genes will some how "Correct" the mutant genes that are causing harm. It may involve one or several of the following techniques, all of which are used. Gene Replacement Therapy involves the mutant gene being replaced by the normal form. This type of therapy is mainly used when the functioning point of a chromosome is very specific, because the other methods work in a general area. Gene Augmentation is the second method. It is used when a specific gene has been damaged or deleted.

During Gene Augmentation, the normal form of a gene is placed in one of the chromosomes. The third method is Gene Inactivation. This works in the opposite way as the others. A gene produces a protein that reduces or neutralizes the effect of a defective gene. It may also alter the gene so that it reproduces fewer copies of itself, or copies which are not damaged.

A story recently ran in the Washington Post Health section. It was about a 15 year old girl who has brain cancer, name Becca Lilly. She underwent gene therapy six months ago to try and destroy the tumor. No other patient had ever received gene therapy for a brain tumor before. The doctors knew that the odds of her surviving were not very good, but they went ahead with the procedure because if they didn't,