Gene Therapy Essay

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Gene Therapy

The Human Genome Project began around 1986. The main goal of the project is to locate and sequence all genes found in human DNA. The objectives for this ambitious effort are to learn more about heredity of disease and to discover the genes that would aid in gene therapy. Advances in gene therapy strive to treat hereditary diseases and possibly eliminate disease from the genome.

The definition of gene therapy is the introduction of genes into existing cells to prevent or cure a wide range of diseases (Jaroff, 1996). Gene therapy has two possibilities of disease treatment, somatic gene therapy and germline gene therapy. Somatic gene therapy involves the manipulation of gene expression in cells that will be corrective to
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Possible vectors are retroviruses. Retroviruses are specialized virus containing RNA that has a knack for finding its way into a cell’s genome and making itself at home (Elmer-Dewitt, 1994). This vector is optimal because it can be genetically engineered to not infect the patient with the virus, but it will carry the corrected gene in the RNA. When the retrovirus attacks affected cells, it will commence replication, replacing the affected gene with the corrective gene. The healthy cells are then injected into the patient to continue replication in the body.

An example of this procedure was first demonstrated in 1990. The patient was a four-year-old girl. She had severe immune-deficiency disease caused by a faulty gene that failed to order the production of a vital enzyme. (Jaroff, 1996) Some of the white blood she carried were extracted and introduced to genetically engineered viruses containing healthy copies of the gene. The viruses performed as expected and attacked her white blood cells. The cells were then injected back into her bloodstream. The newly corrected genes began producing the proper enzyme. The problem that arose with this particular procedure was she was not cured, because the altered cells did not reproduce and eventually died. They young girl needed treatment to be administered every one to two months, based on the lifespan of the cells containing the corrected gene. Treatment did make it possible for this child to lead a relatively normal
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