In a world perplexed by untreatable genetic diseases, gene therapy has the potential to cure and prevent diseases and disabilities by using a method in which scientists alter the DNA in humans or animals. This new discovery in still in its infancy, but through continued research and clinical trials, gene therapy has the ability to save millions of lives.
Gene therapy is a method of correcting defective genes by replacing them with a fixed copy (Dedicated). Biochemist and physician Stanfield Roger first introduced this concept in 1970 when he suggested the use of healthy DNA to replace faulty DNA with the goal of curing inherited diseases (History). Following this, Dr. Theodore Friedmann and the scientific director of the Council of Bioethics
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As of today, over 1800 clinical trials have been completed and multiple more have been approved or will take place in the near future (Result). The first patient to be treated through gene therapy was four-year-old Ashanti DeSilva in 1990 when she was suffering from adenosine deaminase, or ADA, deficiency, which affects the immune system and makes one more susceptible to infections and disease (Four). Dr. W. French Anserson removed DeSilva’s white blood cells and injected them with genes that make ADA, he then inserted the white cells back into DeSilva. This treatment proved affective and prevented DeSilva’s likely premature death. However, DeSilva requires periodic gene therapy, as the treatment did not produce healthy cells. (Hope) There is no doubt gene therapy saved DeSilva’s life, but she currently takes a dose of enzymes and this makes it difficult to analyze the extent to which gene therapy had in DeSilva’s situation (Four). Nonetheless, this case exemplifies the future of gene therapy with continued research and experimentation. For example, one study used gene therapy to lead 5 adults and 19 out of 22 children with one-type leukemia into remission