The Success Of Gene Therapy In Curing Genetic Disorders
Gene therapy is the process by which genes can be used to treat or prevent future disease. Human gene therapy began approximately three decades ago, in the 1970’s genetic engineering first became possible through the use of recombinant DNA technology; however, difficulties were noted during early trials due to difficulties in controlling the insertion sites for the new genes into the genome. Advances in Gene editing in recent history have allowed researchers to more accurately control where in the genome changes occur therefore ‘turning the gene on and off’ by adding or subtracting DNA from particular locations. The purpose of this paper will therefore be to discuss the success of gene therapy in curing genetic disorders through the use of research articles and recent studies to provide a balanced argument and factual conclusion.
Sickle cell disease (SCD) is an inherited disease which is one of the most predominant of disorders. In the United States alone, there is around 90,000 people that suffer from sickle cell disease and over 275,000 born with the disease on a yearly basis. Patients have extremely painful vaso-occlusive crises, which is a complication of sickle cell anemia, the effects are irreversible organ damage, poor quality of life and reduced life expectancy. It is stated that ‘’Allogenic hematopoietic stem-cell transplantation currently offers the only curative option for patients with severe sick cell disease’’, therefore it will amount in many people’s lives being affected by the disease if they do not get this treatment. There is disadvantages to gene therapy treating sickle cell disease, such as the shortage of sibling donors that match and the expenses are high, therefore only a limited about of people can get the treatment.
People with Parkinson’s disease lose their ability to control movement over time due to the loss of cells in the brain. 15 patients with advanced Parkinson’s disease were treated using gene therapy instead of oral dopamine replacement therapies. The trial was named ProSavin, it was aimed at patients that had the disease for 5 years or more, age 48-65 years old and over half the motor response to oral dopaminergic therapy. The aims of the 1/2 study was the number and state of condition after treatment from ProSavin using GT compared to the motor response that were assessed from oral dopamine replacements. After 12 months the results showed that all patients had improved motor behavior, without any harmful effects that were caused by the gene therapy. Therefore, gene therapy was successful in treating Parkinson’s disease.
In 2013, a gene therapy trial was postponed due to two young people being diagnosed with leukaemia. They were born with severe combined immunodeficiency (SCID). This is where they are missing the genes that make up the immune system. This genetic disorder was also known as the bubble baby disease. The gene therapy treatment involved inserting the gene that their bodies were missing. 8/10 people were treated from the disease with no side effects, however 2/10 were cured but then developed cancer and had to get chemotherapy to be further treated, (Dave, U.P 2003). This study shows that even though many of the patient were cured, it still wasn’t 100% efficient as it went on to cause leukaemia. Although there are many cases where GT has been successful in treating genetic disorders, there Is still many problems associated with it. The advantages of gene therapy are that it has the ability to replace defective cells, curing many of genetic diseases, that without GT could have had a much shorter life expectancy. Research has also shown that it can be used to treat a wide range of genetic disorders, including cystic fibrosis, Parkinson's disease and many more.
The disadvantages of GT is that is it very expensive as it treats conditions that are wide spread, meaning that it takes a lot of time assessing and treating an individual. Also, many genetic disorders can be very rare, therefore research and tests on the genetic disorders must be carried out before they can be successfully treated, which requires extensive funding. There is also a risk of the immune response when having gene therapy treatment, where the body will fight against anything that is considered foreign and ultimately causing harm to themselves. Scientists must use vectors that reduce the risk of the body’s immune response from reacting to the new genes.
Overall, GT has many negative effects and risks that can potentially cause more harm. If the new gene that is being inserted in the body happened to collide with another gene, it could potentially have a damaging effect, causing cancer from uncontrollable cell division.
From both studies I have researched I have noted that GT has a potential in curing many genetic disorders and does so successfully with few. However, I believe there are many risk factors in GT that could result in serious after effects from the treatment, causing more harm and in some cases resulting in death. I believe that with extra funding and testing, there could be a greater development in GT. With the expenses of treatment and testing of rare genetic disorders it will continue to be unavailable to those who cannot afford the treatment.
