Gene Therapy Essay

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The Human Gene Therapy Genes, which are carried on chromosomes, are the basic physical and functional units of heredity. Genes are specific sequences of bases that encode instructions on how to make proteins. When genes are altered so that the encoded proteins are unable to carry out their normal functions, genetic disorders can result. A therapy used to treat the genetic disorders in human beings is called gene therapy. Gene therapy has the potential to bring changes in treatment of various diseases and disorders. The therapy relies on gene replacement as the solution to treat the disorders. The genes that are responsible for the disease are removed or replaced with a 'healthy' or working gene. Thus, the enzyme or protein requirement of the body is fulfilled. It results into the elimination of the disease. Gene therapy is a technique for correcting defective genes responsible for disease development. Therefore, the aim is to add a functional copy of the lost gene back into the genome and express it at sufficient levels to replace the missing protein. It is only suitable if the pathogenic effects of the disease are reversible. The earliest mentions and references that specifically addressed a scientific approach to carry out human gene therapy were in 1966, by Edward Tatum and Joshua Lederberg. However, it was as early as 1963 when Lederberg mentioned first in an article called “Biological Future of Man", the first reference to the concept of gene therapy. “We might anticipate the in vitro culture of germ cells and such manipulations as the interchange of chromosomes and segments. The ultimate application of molecular biology would be the direct control of nucleotide sequences in human chromosomes, coupled with recognition, selection and integration of the desired genes…”1 On May 26, 1966, a symposium took place at Columbia University College of

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