Gene Therapy for Cystic Fibrosis

Table of Contents


The gene is one of the most widely studied units in the hereditary system. It is generally described as the physical and functional unit of hereditary. If the gene is altered, it cannot perform its functions well and this will result in what we call a gene disorder. As usual, any disorder in the body will lead to the development of a disease and if not corrected it may eventually lead to death. One of the techniques for correcting defective genes is called gene therapy. (Robert 5-6)


Gene therapy is a way of correcting gene disorders or abnormalities through techniques tried in experiments. There are ways in which scientists are trying to approach the gene therapy issue. Some of these ways include; inserting a gene that has no abnormality anywhere within the cells to replace the faulty genes, inactivating or “knocking out” a mutated gene that is not functioning properly, and also introducing a new gene into the body to help fight diseases. (Pamela, Davis 289)

The rationale of gene therapy is usually to introduce genes into the cells to be used in place of the abnormal gene. How does this happen? Ways of inserting a normal gene in the system of the original faulty system are determined. However, care must be taken to ensure that the gene is not inserted directly. It is important to understand that some viruses have been found to be able to combine and give their genes to human cells if infected with the target cells in the human body. These viruses act as carriers and are called vectors. They must be free from the natural constraints of evolution and should be able to change from the disease-causing virus to a useful virus for use in gene therapy. The researchers are supposed to modify the carrier viruses so that they can’t cause diseases when used in people. (Robert 5-6)

Gene therapy is faced with several challenges in its bid to become an effective treatment for genetic diseases. Gene therapy may not be a save heaven. Some gene therapy may lead to poisoning of the body system; it may also lead to the development of malignant tissues which cause cancer. The immune system of the body may treat any foreigner (the vectors) as a disease-causing organism. Through repeated invasions by foreign viruses and other organisms, the white blood cells and the immune system, in general, may not welcome invaders it has seen before. This means that repetition of the gene therapy process once a problem arises again is impossible. Another problem is brought about by the virus itself. Once it enters the body, it may regain its ability to cause disease and become a threat to the patient. Lastly, some genetic disorders are complex, for example, multigene disorders, and may not be treated using gene therapy. In other words, gene therapy is well suited for disorders that arise from a single gene mutation. Scientists are still researching possible ways of reducing these challenges. (Robert 5-6)

Gene therapy can be used to treat cystic fibrosis which is a disease that causes among others chronic lung disease. An abnormality in the gene called cystic fibrosis transmembrane conductance regulator (CFTR) is the cause of the disease. These abnormalities include a short CFTR gene than the normal one or a protein that has a different shape from the normal one, when this gene is abnormal, certain things that bring about abnormality may happen; the movement of chloride ions is affected. This leads to excessive secretions in the organs affected mostly the lungs and the person with such an abnormality becomes prone to infections. Other effects are that an abnormal gene is degenerated easily leading to health problems and also the formation of fewer proteins than expected. When these mutations occur in the cystic fibrosis transmembrane regulator, one is said to have cystic fibrosis. This research and understanding of the cystic fibrosis gene are essential in the correction of the CF gene disorder. There are several methods of managing this abnormality. We talk of managing because there is no permanent cure for the same. In a real sense, they treat the symptoms rather than treating the cause. A good diet is required for a person diagnosed with this abnormality. The lungs may be treated through different medical therapies and transplantation. However, the most effective way of treatment is through gene therapy which is the main focus here. It treats the cause. (Pamela, Davis 219-224)

In the introduction, I explained that one of the ways of doing gene therapy is through inserting a normal gene to replace the mutated gene. In one of the efforts to treat the abnormality, the CFTR gene was combined with lipid and this mixture was given to the patients in the form of an aerosol. 25 percent of the patients who received the mixture regained their normal chloride transport. This is one of the ways of doing gene therapy to control cystic fibrosis and research is still underway to increase its effectiveness. (Robert, 2009)

In another bid to treat cystic fibrosis, researchers used the method of infecting a virus to the patients and it worked very well. As discussed earlier this method involves injecting a virus that is a carrier of the gene. When these scientists infected the viruses in the patients, they were completely cured of cystic fibrosis. To minimize some of the challenges like the one posed by the immune system, they created a virus that avoided the immunity system. Due to this property of the virus, it was able to stay in the body for a long time and therefore solving the problem of unnecessary repetition of the process. (Robin 23-24)

It should be noted that the virus needs to remain in the body for a long time, without being attacked by the white blood cells to ensure that it gives a gene to its intended target, in this case, the lungs. For gene therapy to be effective, the virus should also be able to penetrate to the intended organ, the lungs, without inhibitions. A lot of mutations were done on the virus, and at the end of the day, the viruses that had the desired characteristics were selected and then infected with the lung cells of the patients. As far as the research on the treatment of cystic fibrosis was concerned, the viruses on trial underwent several changes and mutations and were able to connect to different receptor cells on the surface of the lungs. It also developed an ability to move straight inside the cell through so many obstacles. The method worked very well and this gives hope to so many people in the whole world who are suffering from this condition. (Robert 5-6)


In conclusion gene therapy- a way of replacing faulty genes with normal ones-is a method of managing cystic fibrosis and other many genetic disorders. The research is promising good results and though it’s in an infant stage, hope is still there. Scientists have started to see light at the end of the tunnel and amid the challenges like the ones discussed above, there is no doubt that cystic fibrosis will be a thing of the past.

Work cited

Robert, S., 2009. ‘Evolved’ virus may improve gene therapy for cystic fibrosis. UC BERKELEY. p. 5a.

Pamela, Davis. “Cystic fibrosis. Vol 64 of lung biology in health and disease.” Informa Health Care, (1993): 248-382

Robin, M., 2009. GENE THERAPY TO TREAT CYSTIC FIBROSIS The guardian.12. Web.

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