Gene therapy hasprogressed significantly in recent years. However, issues surrounding its useare controversial. Present and evaluate the arguments for and against the useof gene therapy in humans.Nowadays, gene therapy has a significant impact on our life. It is widely used, even if we do not realize it. This field of science is currently growing, so it is worth it is worth to know something about it. Gene therapy is a method of treatment of diseases with a genetic basis. Its aim is to eliminate abnormal cells, introduce changes in the phenotype and physiology of cells, as well as, among other things, introduce into the cell the correct copy of the gene. According to U.S National Library of Medicine (2018), gene therapy is a therapy in which genes are used to treat or prevent the different diseases. Thanks to this technique, it is possible that in the future doctors instead of administering drugs to patients or surgeries, will heal people by inserting appropriate genes into their cells. There are different methods in gene therapy, such as: replacing a mutated gene that causes disease with a healthy copy of the gene; inactivating, or knocking out, a mutated gene that is functioning improperly; introducing a new gene into the body to help fight a disease. Gene therapy is quite new, it is constantly being researched in terms of whether it is safe and what it can have in the future. Although it is not fully researched, it seems to be a very promising technique, it can be a huge opportunity to discover ways to treat various diseases, including hereditary diseases, certain types of cancer and some viral infections). In this work, arguments for and against the use of gene therapy in humans will be discussed. Introducing therapeutic nucleic acids into the cells of the human body, i.e. gene therapy, has the potential to revolutionize modern medicine but recent years have brought this field both many successes and failures.The removal of undesirable effects on the organism causedby erroneous gene mutations by introducing foreign, but correct DNA or RNAsequences into the body cells, is gene therapy. This is a very modern method oftreatment that can be performed by administering a preparation containing acomplete gene or only selected DNA or RNA fragments. We can divide gene therapyinto two categories: germ line gene therapy and somatic gene therapy. Insomatic gene therapy genetic the change is not passed along to the nextgeneration, although the gene material is inserted into a cell. In germ linetherapy gene the change is heritable so gene will be passed on to the nextgeneration. (Wirth, Parker and Ylä-Herttuala, 2013) British law prohibits editing of germ-cells or embryos in a clinicalcontext. At present it is unlikely to this process be allowed in any Europeanjurisdiction. (Wellcome.ac.uk, 2018)There has been considerable success using gene therapy in the treatment of genetic diseases associated with immunodeficiency. Very good results are also obtained in the treatment of hemophilia, where a significant improvement in patients condition is achieved. In the case of Parkinsons disease, the use of therapy contributes to the improvement of motor function, but does not completely relieve the disease symptoms. There is great hope in cancer gene therapy because it does not require a long-lasting process of reading and copying the correct genetic information to the cells. The challenge is to fight congenital genetic diseases, which, as in the case of cancer, require a longer process of reading and copying the therapeutic genetic code, which often causes side effects that threaten the patients life.In recent years, there have been many studies testing gene therapy in otherdisease entities. According to Nathwani et al., (2014) anexample is hemophilia B a genetic disorder that is deficient in bloodcoagulation factor IX. It manifests itself in numerous, frequent bleeding andrequires constant exogenous supply of missing protein to the blood of patients.Monogenic diseases such as hemophilia are an ideal target for gene therapy,which is why scholars sought to heal patients by providing the correct form ofthe gene encoding factor IX using a specially crafted adenoviral vector. Theresults of this study were very encouraging. Stable expression of factor IX atthe level of 3 to 11% of the norm was obtained, which was enough to alleviatethe symptoms of the disease. Over a few to over a dozen months of follow-up, 4of 6 patients did not observe any bleeding, while the other two reduced theneed for frequent prophylactic injections. Side effects were very light. Theymainly concerned the increase in the level of liver enzymes. According to Coune, Schneider and Aebischer (2012)another example of recent advances in gene therapy is the attempt to improvethe status of patients with advanced stage of Parkinsons disease. The recentlycompleted clinical trial using the lentiviral vector attempted to restore theproper production of dopamine in certain brain centers (the deficiency of thisneurotransmitter underlies the disease). The vector contained three genesencoding key enzymes for dopamine synthesis, which, expressed in thecorresponding neurons, were able to repair the biochemical defect present inpatients with Parkinsons syndrome. As a result of the innovative therapy, 15patients experienced significant improvement in motor function with minimalside effects. Although scientists have not observed complete remission of thedisease, this trial provides a solid basis for further research.According to Medicalnewstoday.com, (2018) Cellular Networking, Integrationand Processing is a gene transfer technique, which involves introducingspecific genes into the pancreas using a virus as a vector. The team notesthat beta cells are rejected in patients with type 1 diabetes. With the genetransfer method, the newly introduced genes encourage non-beta cells to produceinsulin, without any side effects.However, many failures put a shadow on successful gene therapy. According to Wong, Hawthorne and Manolios (2010) gene therapy can be very helpful in the fight against diabetes but also carries a lot of risks. Gene therapy must be refined, because uncontrolled distribution of genes and cells in the body can be very dangerous. It could lead to a situation in which all cells would start to produce insulin, and then our body would be flooded with it. Only pancreatic cells are now created for the production of insulin. A well-functioning pancreas controls the level of this hormone. Too high a level of insulin would lead to hypoglycemic shock, which is life-threatening. They are especially frequent when adenoviral vectors are used because a significant part of the population has antibodies directed against them (adenoviruses are known human pathogens, they cause colds, among others).According to (Sibbald, 2018) and NATURE International weekly journal of science (2016) inseptember 1999, 18-year-old Jesse Gelsinger took part in a clinical trialtesting gene therapy in a genetic condition called ornithinecarbamyltransferase deficiency. This enzyme is indispensable for a man toproperly metabolize and excrete nitrogen from the breakdown of proteins. Theaccumulating toxic ammonia causes progressive damage to the central nervoussystem ending in a coma. The case of Jesse was so light that the symptoms ofthe disease could be controlled by diet and medications. As part of the study,he received the highest dose of the viral vector. The next morning, Jesseshowed symptoms of severe liver damage and disseminated intravascular coagulation.In the fourth day after receiving the new drug, despite the professionalmedical care, the young patient died. His death was the direct result of amassive immune response to an adenoviral vector that circulated through mostinternal organs.In conclusion, gene therapy in recent years has made ahuge step forward thanks to the continuous improvement of techniques forintroducing genetic material. Gene editing is becoming a tool that isincreasingly used to develop new therapies for onerous diseases. Unfortunately,the reaction of the immune system to the introduction of modified genes isstill unknown. Gene therapy methods may in the future become a basic tool inthe fight and treatment of genetic diseases. At the moment, the greatest hopeis associated with the treatment of cancer and lifestyle diseases of thecirculatory system. In addition, better methods for delivering therapeuticgenes to cells are also needed because it is difficult to control and eliminatethe integration of viral genes into host cells over widely used viruses. Thedevelopment of genetic engineering, research on new nucleic acid vectors,improving their efficiency and safety allow us to believe that perhaps in thecoming years gene therapy will become a widely available, safe and effectiveform of treatment for at least some diseases, complementing conventionalmethods. Gene therapy also raises ethical problems research is conducted oncell cultures, then on mice and then results from human experiments arenecessary. Such persons must be informed about possible risks, which are oftendifficult to assess. However, the potential of research on gene therapy isconsiderable and this field may become widely available and effective. Then itwill be a complement to surgical, radiological and conventional methods.Gene therapy is a very modern and promising treatment technique that canbring many positive solutions to medicine. However, attention should be paid tothe fact that genetic therapy is still under development, clinical trials arebeing carried out and it is not yet widely used.ReferencesCoune, P., Schneider, B. and Aebischer, P. (2012). Parkinsons Disease: Gene Therapies.Dalgleish, A. (1997). Why: Gene Therapy?. Gene Therapy, 4(7), pp.629-630.Fda.gov. (2018). U S Food and Drug Administration Home Page. [online] Available at: https://www.fda.gov/ [Accessed 9 May 2018].Fischer, A. (2016). Gene therapy: Myth or reality?. Comptes Rendus Biologies, 339(7-8), pp.314-318.Mavilio, F. and Ferrari, G. (2008). Genetic modification of somatic stem cells. The progress, problems and prospects of a new therapeutic technology. EMBO reports, 9, pp.S64-S69.Medicalnewstoday.com. (2018). GDPR consent required. [online] Available at: https://www.medicalnewstoday.com/articles/317343.php [Accessed 28 May 2018].Nathwani, A., Reiss, U., Tuddenham, E., Rosales, C., Chowdary, P., McIntosh, J., Della Peruta, M., Lheriteau, E., Patel, N., Raj, D., Riddell, A., Pie, J., Rangarajan, S., Bevan, D., Recht, M., Shen, Y., Halka, K., Basner-Tschakarjan, E., Mingozzi, F., High, K., Allay, J., Kay, M., Ng, C., Zhou, J., Cancio, M., Morton, C., Gray, J., Srivastava, D., Nienhuis, A. and Davidoff, A. (2014). Long-Term Safety and Efficacy of Factor IX. The New England Journal of Medicine.NATURE International weekly journal of science. (2016). Gene-therapy trials must proceed with caution. [online] Available at: https://www.nature.com/news/gene-therapy-trials-must-proceed-with-caution-1.20186 [Accessed 28 May 2018].U.S National Library of Medicine (2018). What is gene therapy?. [online] Genetics Home Reference. Available at: https://ghr.nlm.nih.gov/primer/therapy/genetherapy [Accessed 28 May 2018].Wellcome.ac.uk. (2018). UK legislation and regulation governing the use of gene editing | Wellcome. [online] Available at: https://wellcome.ac.uk/what-we-do/our-work/uk-legislation-and-regulation-governing-use-gene-editing [Accessed 9 May 2018].Wirth, T., Parker, N. and Ylä-Herttuala, S. (2013). History of gene therapy. Elsevier.Wong, M., Hawthorne, W. and Manolios, N. (2010). Gene therapy in diabetes. pp.165175.Get Help With Your AssignmentIf you need assistance with writing your assignment, our professional assignment writing service is here to help!Find out more
