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History and Advancements of Gene Therapy

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Gene therapy is an advanced and evolving technology that is being researched and developed extensively. It is an experimental technique that simply, introduces a gene into cells, in the place of a defective or missing gene to correct a genetic disorder. Gene therapy is very interesting and it is very important for scientists to grow the knowledge of gene therapy which can be expanded and applied to help correct different genetic disorders.

In 1966, a paper was published by Edward Tatum, proposing the effectiveness of viruses to be used in gene therapy. A couple of years after the paper was published, a demonstration proved Edward's concept true.

The term 'gene therapy' arose during the 1960's and 1970's. The first successful patient to be treated with gene therapy was in 1990, a four-year-old girl who had ADA (Adenosine deaminase deficiency), a disease that severally affects the immune system and its ability to fight infections. The therapy consisted of her white blood cells being taken from her and being inserted with the correct genes for making ADA, which was then reinjected into her. Along with her, another nine-year-old was infused with their own corrected cells over a two-year period.

Gene therapy has multiple uses, the main being used to fix defective genes or to replace missing genes. Researchers and scientists have been working on gene therapy for decades and the future is very encouraging. Gene therapy can be used to correct a mutated gene. Gene therapy has successfully cured multiple diseases such as immune deficiencies like Severe Combines Immune Deficiency, Adenosine deaminase deficiency, different types of inherited blindness, haemophilia, blood diseases, fat metabolism disorder and gene therapy treatments are under development for cancer. A promising discovery of an effective treatment for melanoma has provided hope for many cancer sufferers. Although gene therapy holds a promise for treating a range of diseases such as cancer, heart disease, diabetes, haemophilia, cystic fibrosis, it is only available as part of a clinical trial.

Gene therapy is the future of curing diseases. Through the process of gene therapy, genes are able to be introduced into existing cells to cure a wide range of diseases. Some examples of gene therapy successes include:

  • Parkinson's disease is a disease that targets the brain, causing the patient to lose cells. As the disease advances, the sufferers lose the ability to control their movements.
  • After a small group of patients were treated by the introduction of new normal cells, they all had improved muscle control.
  • Beta-Thalassemia is a disease where patients don't have enough red blood cells to carry oxygen throughout the body. Sufferers depend on constant blood transfusions for survival.
  • In 2007, a patient with a severe blood disease received gene therapy to treat the blood stem cells. After the treatment, the patient was able to raise the level of healthy red blood cells. The patient is now living without blood transfusions.
  • Immune deficiencies are among the first genetic disorders to be treated successfully by gene therapy.

An example would be Severe Combined Immune Deficiency. SCID was one of the first to be treated by gene therapy. The cure for SCID has been modified since it was first discovered because the old cure could potentially trigger leukemia. The cure now uses safer vectors which are much less likely to cause cancer.

Still in research, gene therapy is far from being perfected. Gene therapy has many risks because of this. Some risks may include inflammation because of how your immune system responded to the working gene copy, the working gene might be slotted in the wrong spot, the working gene might produce too much of the missing protein or enzyme it was targeted to do, causing complicated health problems and other genes may be delivered to the cell instead of the correct gene and the deactivated virus that was intended to target a cell might target another cell. Because gene therapy changes how your body functions, some people may be against that idea because they believe that it is ethically wrong to do. For example, some people might believe that God made us how we are and it is not up to us to change our genes and how they function to fix our imperfections.

Gene therapy is still in development and conducting gene therapy clinical trials with genetically modified organisms presents potential safety and infection issues. All human clinical trials that are undertaken in Australia must be conducted under either the Clinical Trial Notification (CTN) or Clinical Trial Exemption schemes (CTX). In addition to the compliance to the CTN or CTX, any human clinical trials involving gene therapy must also require approval by the Gene Technology regulator under the Gene Technology Act of 2000. The intention of the Gene technology act is "to protect the health and safety of people, and to protect the environment, by identifying risks posed by or as a result of gene technology."

I believe that gene therapy is a big advancement in science. Gene therapy has the potential to revolutionize the way we can cure diseases. Gene therapy, if perfected, has an enormous potential to do good such as curing genetic disorders, but things such as genetic engineering are being researched more. There are already some foods such as rice, that has been genetically altered to have more minerals and vitamins, including vitamin A. This sort of genetic modification isn't bad because it can help increase vitamin levels in some populations of the world that do not have access to more nutritious foods. The concerning side to genetic engineering would be that scientists could find ways to manipulate anything wee need such as our height, build, gender, eye sight ability, potentially developing 'super-humans'.


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