Over the years genetic disorders and gene-related illness have been responsible for high mortality rates and reduced quality of life. Genetic disorders can be due to misalignment, missing genes or excess of a gene. Genetic therapy works to replace the defective genes with new ones. Gene therapy is the insertion of genes into an individual's cells and tissues to treat a disease, and hereditary diseases in which a defective mutant allele is replaced with a functional one. (Medlineplus 2020)
Gene therapy was first tested in the 1990 to an individual suffering from a disease known as adenosine deaminase (ADA) deficiency which affects the immune system. Statistics state that, ‘They are more than 6000 diagnosed disorders in the UK’ with more disorders being recognised daily. On average, ‘30000 infants and children are diagnosed each year leaving more than 2.4 million people suffering from a genetic disorder’ Having a genetic disorder can not only have a huge impact on the individual who is affected, but can also have a huge impact on their quality life and their loved one who may have to look after them and provide care for them. (news medical life sciences 2020)
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Some may agree on fact that gene therapy is successful in curing genetic disorders as it has drastically changed many lives. This is due to the fact that gene therapy replaces a faulty gene or adds a new gene in an attempt to cure disease or improve your body's ability to fight disease. Gene therapy holds promise for treating a wide range of diseases, such as cancer, cystic fibrosis, heart disease, diabetes, haemophilia and AIDS. This means that the patient has the opportunity to be diagnosed and have the reassurance of being treated. In addition, the effects of therapy are long lasting and timeless. By replacing a defective gene with a functional gene in a disease like cystic fibrosis, there are limited chances of remission, and this is usually a one-off treatment that will limit the symptoms. Also, gene therapy is not just a remedy to the individual suffering from a condition, but it has an impact the entire genetic offspring. When you remove a gene predisposing one to breast cancer, they won’t transfer the defective genes to their offspring but the new functional gene.
There are also many more reasons why someone may believe that gene therapy is successful in curing genetic disorders. For example, it is stated that, “Severe Combined Immune Deficiency (SCID) was one of the first genetic disorders to be treated successfully with gene therapy, proving that the approach could work,” (Biolyse 2020) emphasising that gene therapy can eradicate disease and improve the quality of life. Patients with Parkinson's disease gradually lose cells in the brain that produce the signalling molecule dopamine. As the disease advances, patients lose the ability to control their movements. Research shows that a small group of patients with advanced Parkinson's disease were treated with a retroviral vector to introduce three genes into cells in a small area of the brain. These genes gave cells that don't normally make dopamine the ability to do so. After treatment, all of the patients in the trial had improved muscle control. This indicates that gene therapy is a reliable source of treatment for individuals with genetic disorders.
On the other hand, some people may disagree with this statement because although gene therapy is a promising treatment option for a number of diseases including inherited disorders, some types of cancer, and certain viral infections, the technique remains risky and is still under study to make sure that it will be safe and effective. For example, there is no guarantee that this is reliable and something could go wrong during the process which could have a huge impact on an individual’s life. Gene therapy can fail to work thus limiting an individual's capabilities or even worser the condition. Incompatibility issues and immune response can also lead to failure of the procedure. It is stated that, “Many people have found that the benefits of their gene therapy treatments began to wear off as soon as they were no longer taking their medicine or visiting their doctor for treatments.”
This means it can be a lifelong course that someone must follow to reduce or eliminate the genetic issue that affects their health. This also could have an influence on the individuals loved one is as they would be worried about their health and wellbeing, which could cause ongoing stress to not only the individual but also the family, Moreover, some people, may disagree that gene therapy is successful in curing genetic disorders because gene therapy has some potential risks. For instance, stem cells grown in the lab may be contaminated with a virus which could be passed on to the patient and so make them sicker. Also, some people are against stem cell research because they feel that human embryos should not be used for experiments since each one is a potential human life. The body’s immune system may see the newly introduced viruses as intruders and attack them. This may cause inflammation and, in severe cases, organ failure. Additionally, because viruses can affect more than one type of cells, it's possible that the altered viruses may infect additional cells, not just the targeted cells containing mutated genes. If this happens, healthy cells may be damaged, causing other illness or diseases, such as cancer. Finally, gene therapy is a costly treatment option, such a fee may not be affordable to many, and this will create socioeconomic segregation as the rich will be disease-free while the poor remain to bear the burden of such illness.
In conclusion, both arguments are strong and justifiable however, overall I believe that the positives outweigh the negatives and that gene therapy is a successful way to treat genetic disorders as clinical trials of gene therapy in people have already shown some success in treating certain diseases, such as Severe combined immune deficiency and Haemophilia which shows that it is a reliable source of treatment.
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