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Gene therapy has been used to treat a wide range of diseases, including cystic fibrosis. Cystic fibrosis is a common and painful genetic disease that can lead to infections in the lungs, pancreas and digestive system. According to the National Genome Research Institute, around 30,000 people in the United States have this condition. Gene therapy for cystic fibrosis started in 1990 when scientists were able to successfully correct a defective cystic fibrosis transmembrane conductance regulator gene. Since that time, techniques and breakthroughs in gene therapy have occurred but this treatment has both benefits and consequences.
The most notable benefit of gene therapy for cystic fibrosis is that it has the potential to restore health to many who are suffering with the disease. Currently, gene therapy can be used to treat lung problems caused by cystic fibrosis, and scientists hope that in the future technologies in gene therapy will treat other organs affected by the disease. It’s not just cystic fibrosis that has the potential to be cured in this way — gene therapy has the potential to permanently eradicate many diseases. Researchers are seeking to eventually eliminate cystic fibrosis at the root.
One disadvantage of gene therapy is the controversy surrounding the medical treatment. Some question the ethics of gene therapy, believing that scientists shouldn’t interfere with the genetic makeup of an individual.
Another potential drawback the relative newness of the technology. Researchers are still investigating potential side effects and many clinical trials are still underway. For this reason, medical mishaps can occur.
In addition to gene therapy, those who suffer from cystic fibrosis can consider other treatment alternatives, such as proper nutrition. Those with the disease should follow a specially tailored dietary program that involves getting the extra calories that they need to fight infections. Additionally, increasing antioxidants can be helpful in treating the condition.
Inhaled medications, such as mucolytics and antibiotics, can be used to fight bacteria and infections associated with cystic fibrosis. One common antibiotic, azithromycin, has been shown to have special benefits for people with this illness, and it may help maintain and improve lung function.
A major concern is how gene therapy could set the table for genetic engineering in the future. While that might be used to correct abnormalities in the womb, genetic engineering could be used to create the "ideal human" at some point. The slippery slope argument is what a lot of people opposed to gene therapy appear to use -- once a little genetic tampering is deemed normal, what's to prevent people from exploiting the technology to its full potential?