Should Gene therapy be allowed to prevent cystic fibrosis?

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“Every week on average five babies are born every week with cystic fibrosis, and 3 sufferers die from it, usually caused by the damage to the lungs”1. CF is a life threatening disease that cannot be cured with the current treatments. The treatments include exercise, dietary programs, inhalation of antibiotics; breathing control techniques and daily physiotherapy.

The initial cause of cystic fibrosis has been agreed by many sources such as  to be the single faulty gene that controls the movement of salt in the body resulting in the growth of internal organ’s becoming clogged with thick sticky mucus.


Although the methods mentioned above increase life span, they only target specific symptoms, rather than curing the defect itself, on the other hand, gene therapy is undergoing research, in both clinical and laboratory to see if it is safe and effective.

“Only 33% of clinical trials have been focussed on ‘single gene’ diseases, and one of them is the common cystic fibrosis.”15 The other 67% is clinical trials of gene therapy in humans have been cancer treatments. So treating gene therapy with cystic fibrosis is something which will have to be taken into consideration, but the technique gene therapy is always going under thorough research.

Here are few reasons why gene therapy will not be able to prevent Cystic Fibrosis:

  • Firstly Gene therapy has a short lived nature, the DNA that is introduced into the target cells must remain functional and the cells containing the DNA must be long-lived and stable. The problem in doing it is integrating the DNA into the genome and the rapidly dividing nature of cells prevents gene therapy from achieving any long term cure. If patients wanted a longer and stable cure, they will have to undergo multiple rounds of gene therapy.
  • There are problems with viral vector; a study has shown potential problems to the patient, such as inflammatory, toxicity and immune responses. Furthermore if the viral vector starts to recover, there is always a possibility that it could cause disease.
  • If the candidate was being treated for a single gene, this would be benefits when it is being treated with gene therapy, whereas more common diseases, like heart disease, arthritis, diabetes and Alzheimer’s disease, are cause by multiple effects on a variety of genes. Theses multigene disorders are quite likely to make treating the gene effectively with gene therapy.
  • Geneticists (those who research genes) are worried that the virus might target the wrong cells. If the DNA is misplaced or integrated in to the wrong genome, if it was inserted into a tumour suppressor gene, it could induce a tumour.

  • Then biggest of all issues is its ethical, legal and religious concerns, many believe if prenatal tests were completed that these could lead to the increase in abortions, furthermore religious groups are considering manipulating an individuals as corrupting God’s work.

All of these are against gene therapy and are good solid reasons why gene therapy should not prevent cystic fibrosis and other disorders too; however it has been approved successful when treating blindness in patients. “Dr .Artur of the University of Pennsylvania said that “the initial improvements were very substantial and occurred in a matter of weeks”8, this is showing how effective and fast the alterations in the gene is, furthermore he quoted “the adult brain seems to be adapting to this vision”8.

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 What is Gene Therapy?

To prevent some symptoms scientists have identified various different medical treatments, this could be anything from inserting genes to orally taking a tablet, but have not found a cure, and are still facing problems in finding a permanent cure. It was not until recently scientist could provide a long term cure with the use of gene therapy.

Although gene therapy has proven many benefits, it is still a hot on-going debate.  The method has been talked my many as “Violating moral codes”3.  The theory is that the “bad”, missing, or damaged genes are replaced or restored ...

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*** A reasonable A level report, which outlines how gene therapy can be used to treat genetic diseases such as cystic fibrosis. A basic explanation of how gene therapy treats cystic fibrosis is included, but the report does not contain sufficient biological detail for an A-level report. Several distinct biological techniques are also incorrectly discussed as 'gene therapy'. To improve: 1)Discuss the technique of gene therapy in A-level detail at the start of the report, explaining its uses 2)Ensure that techniques such as genetic screening and stem cell therapy are not discussed as gene therapy. They are different techniques 3)Include a scientific journal article and reference using standard formatting 4)Evaluate sources based on the data they include, where it comes from, the institution that has published it or the reputation of the scientists who completed it