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Essay on Gene Therapy
Introduction
The transcription and translation processes involved in reading the genes that are a natural part of a cell's own DNA are exactly the same processes used to "read" foreign genes introduced into a cell in the treatment known as gene therapy. The detailed knowledge gained over the past four decades about how these processes work in a living cell is the foundation upon which molecular medicine is built.
The purpose of gene therapy in most cases is to correct the consequences of having inherited mutant copies of a given gene. The mutation to be corrected did not arise in the individual with the genetic disease; it arose in one or more of his or her forebears. To correct the problem caused by this hereditary accident, it is necessary to identify the gene involved, to isolate normal copies of the gene, and to deliver them to cells of the affected individual.
History
The possibilities for applying the emerging techniques of molecular biology to human gene therapy were appreciated by a handful of people as early as the late 1960s, although at that time no one had the slightest idea of how human genes might be isolated. The first human gene would not even be cloned for another dozen or so years. But there were tantalizing clues of what might lie ahead. In the 1960s, a physician-scientist named Stanfield Rogers at the Oak Ridge National Laboratories made an extraordinary observation with the common wart-causing Shope papilloma virus. One of the genes carried by this virus encodes an enzyme called arginase, which degrades excess amounts of the amino acid arginine. In addition to its role as a building block of protein, arginine also plays a role of its own as an intermediate in the processing of nitrogenous wastes in the body. Rogers noted that in rabbits infected with the Shope virus, the levels of arginine in the blood were unusually low.....
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