Essay on Gene therapy: Can it live up to its promise?

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Gene therapy: Can it live up to its promise?
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“Cystic fibrosis to be treated by gene therapy technology” GUARDIAN

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Gene therapy definitely sounds like a promising gospel to Peter Elliot, 23, who was diagnosed to have cystic fibrosis (CF) when he was 10 months old.

CF is a severe genetic multi-organ disease, affecting primarily the lung and pancreas. In UK, over 7,000 people have the disease; 1 in 25 Caucasians are carriers, whereas 1 in 2500 babies are born with CF. Due to CF, Peter has difficulties in breathing very quickly during exercise because the thickened mucus in his lung cannot be cleared effectively. The stagnated mucus in his lung is a perfect breeding ground for germs. Thus Peter had repeated chest infections since he was four months old. In addition, the effectiveness of antibiotics has declined with prolonged usage of drugs. His lung function deteriorated as CF progressed but he rejected his doctor’s recommentation of having lung replacement. For health reasons, Peter is dependent on his family and has been taking leave at work for intensive treatments. A normal life without cystic fibrosis is what Peter and his family have longed for, but is this what gene therapy can live up to? No, at least not yet.

Genetic diseases were deemed to be untreatable long ago. In the 60s, the idea of gene therapy offered a beam of hope for humankind. It was not until three decades later, therapeutic studies were first approved. The first successful application of gene therapy was reported in 1990. However, successive failure attempts that caused serious complications to patients led to safety fears afterwards.

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“Analysis: Gene therapy has immense potential” TIMES

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CF has been the prime target for gene therapy since the discovery of the faulty gene in 1989. In 1990, scientists revealed that transferring a healthy gene to cells with the faulty gene could heal CF. Although initial research findings were encouraging, it failed to treat cystic fibrosis so far: Immune response was generated to destroy the virus carrying the healthy gene in our body. It is also hard to deliver the gene to all the problematic cells in our body and traffick genes to lung through the thick mucus. These problems led to the failure of a recent phase two trial that used virus as the transporter to cure CF.
While virus is not doing the job right, other trials involving the use of an alternative, liposome, a fatty substance has started. Liposome is a better carrier than virus because it would not be destroyed by immune response. However, it is less effective as compared to virus. As a result, researchers are still unable to deliver sufficient amount of gene to cause any significant improvements.
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“Breakthrough raises gene therapy hopes” INDEPENDENT

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So far, our technology enables the transportation of healthy genes to sites containing the faulty gene. Yet, scientists need to overcome a number of hurdles to make it a viable therapy e.g. to improve the efficiency of transferring the good gene to faulty sites and to increase the effective duration of the transferred gene. To date, researchers are still looking for the optimal delivery method. Assuming that the therapy is feasible, the time required to overcome the above problems is largely
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