Human Gene Therapy: A Revolution In Progress

1044 words - 4 pages - by xebecrural ✓ Expert Reviewed

Introduction
Genetic disorders usually occur as a result of the defectiveness of a mutated gene. We have not yet discovered a way to change the mutated genes to healthy genes, but we do have treatments for the symptoms, one being gene therapy. Human gene therapy, “holds great potential for treating the relatively small number of disorders traceable to a single defective gene” (Reece et al., 2011, p. 418). Basically, gene therapy seeks to fix what is wrong with abnormal genes as solutions to genetic diseases. Gene Therapy’s process of inserting a normal allele of the defective gene into the body cells of the tissue (that have been affected by the infections), has been successful very few times because we do not much about the interactions between the genes and the rest of the body. Not knowing these types of information about gene therapy raised many scientific and ethical issues. Gene therapy does have disadvantages; however, its development is a major contribution to our society as it helps give more life time for ill people.
The process of gene therapy
Gene therapy treats deficiencies in the body by depending on a good delivery system to take correct genes to the affected cells. If a defective gene causes a protein to not function, then gene therapy will produce a normal copy of the gene and take it to the proteins (to restore their function) (“How does gene therapy work?” 2013). If a gene is introduced to a particular cell, then the cell will not be able to function. Therefore, scientists use vectors (transports the new gene); usually they use certain viruses as vectors so that the gene will enter by infecting the cell. Infecting the cell will give it the cell a sudden “push” to take in the gene. This new delivered gene will make a functioning protein if the treatment was successful (“How does gene therapy work?” 2013). If the treatment was not successful, then the consequences could be life-threatening. Gene therapy was first tested on a 4-year-old girl on September 14, 1990. She was diagnosed with adenosine deaminase, a disease where her body does not try to fight against infections. Dr. W. French Anderson used her white blood cells as the vector. He injected genes for making adenosine deaminase into the blood cells. Dr. Andersen sent the vector to the adenosine deaminase, so the normal gene could enter it (“Gene Therapy,” n.d.). It was a successful experiment.
The advantages and disadvantages of gene therapy
Some of the many human gene therapy experiments have successfully treated genetic diseases. Therefore, it is considered to be a significant breakthrough in medicine. For example, scientists have used gene therapy on leukemia patients, and the end result was success. Basically, experiments were performed on three men with advanced cases of leukemia. For many years, scientists worked to increase the strength of the immune system, so fighting cancer could become easier. Dr. Carl June and his colleagues used a novel carrier (vector) to...

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