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Gene Therapy - Essay Example

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Gene therapy can be defined as the use of DNA as a drug to treat disease by delivering therapeutic DNA into a patient’s cells/ it is a form of treatment involving the alteration of genes inside the somatic cells that halt disease/ an experimental technique that uses genes to…
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Gene Therapy
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Download file to see previous pages Gene therapy uses a vector which functions to deliver DNA inside body cells after packaging. The DNA, once in the body through the vector goes into the bloodstream then into cells and finally is incorporated into a chromosome. However, naked DNA approaches have been considered too more so in the field of vaccine development. Once embedded in the patient’s system, the DNA is expressed by the cell machinery, leading to the production of therapeutic protein which corrects the patient’s condition. Emphasis lies on administering a gene that will cause a protein to be expressed and that the patient specifically needs. In addition, with the advances in knowledge of nuclease functions in humans, there have begun explorations into ways of incorporating genes that encode nucleases into chromosomes. The expressed nucleases then disrupt the genes causing the disease by ‘editing’ the chromosome (Giacca, 2010).
The concept of gene therapy was first thought possible in 1972 but caution was implored especially concerning its application/ experimentation on humans. In 1990, however, Ashanti DeSilva became the first recipient of gene therapy treatment in the United States for ADA-SCID. Early skepticisms arose with several initial clinical failures with many regarding gene therapy as an over-rated procedure but successes since 2006 have seen many regain their faith in this new form of treatment. Over 2,000 recorded clinical trials have so far been performed on humans. These include successful treatment of diseases such as multiple myeloma, Parkinson’s disease, Leber’s congenital amaurosis, adrenoleukodystrophy, hemophilia, ADA-SCID, chronic lymphocytic leukemia and acute lymphocytic leukemia. With such successes, many governments and companies (especially research institutions) have continued to invest even more on gene therapy. Recently, Glybera became the first gene therapy procedure to be embraced in Europe and the ...Download file to see next pagesRead More
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