Introduction
To understand the meaning of gene therapy, it is necessary to begin by having an overview of what a gene is. The human body is made up of trillions of cells. Cells are the basic units that make up all living things. Every cell has the nucleus, which acts as the command center. The nucleus contains chromosomes, which are consequently made up of DNA, a material that is usually inherited and transferred from one offspring to another. As Cotrim and Baum (2008) point out, genes are DNA segments and that each person every individual has two copies of every gene; one from every parent. Misra (2013) states that every human being has approximately 20, 000 genes, most of them being the same in all people. Nevertheless, every individual has a small portion of genes which vary slightly. It is because of these small variations that people have different features. According to Cotrim and Baum (2008) gene therapy is the process where a functioning gene is inserted into cells to rectify a dysfunction or to cause a different cellular function. Using the example of diseases such as muscular dystrophy, cystic fibrosis, and hemophilia, Carmichael (2014) says that gene therapy can be used to rectify the defective genes to eliminate the conditions.
History of Gene Therapy
Gene therapy is not a new concept because the process has been used for a considerable length of time now. Daniel (2013) says that although it is difficult to tell when gene therapy began, he says that 1967 can be regarded as the year when the subject of gene therapy began to be discussed. One of the prominent people who triggered the discussion was the 1968 Nobel Prize winner known as Marshall Nirenberg (Carmichael, 2014). Nirenberg was a Nobel Prize in physiology. In one of his papers, Nirenberg wrote and speculated about the programming of synthetic messages. That means that Nirenberg saw the possibility of cells being modified to different specifications. Although Nirenberg wrote about the advantages that would accrue, he also pointed out some of the dangers and pitfalls that would ensue if specialists do not exercise caution.
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The initial attempts to try gene therapy were not successful. For instance, between 1970 and 1973, Stanfield Rogers, an American scientist, joined efforts with a German physician to develop treatment for hyperargininemia. The two scientists attempted to treat two sisters that were suffering from the condition (Daniel, 2013). They experimented with a virus known as Shope papilloma virus (SPV). Rogers had the conviction that the virus would result into the expression of the gene that controlled the manufacture of arginine. However, scientists would later render Rogers’ assumption as being false.
Despite Rogers and his counterpart’s experiment not yielding any significant results, other scientists and bodies saw the possibility of gene therapy. The National Institutes of Health (NIH) is always credited for the foresight when it took the lead in 1974 to direct research on recombinant DNA. The NIH even created an oversight authority which was known as the Recombinant DNA Advisory Committee (RAC). Initially, the body was only made up of experts that had knowledge in recombinant DNA technology. Nevertheless, the membership expanded to allow individuals that had knowledge in diverse fields such as science, medicine, ethics, as well as, lay communities. The RAC’s main role was to give approval to individuals who wanted to carry out research on gene therapy especially in the laboratories that were funded by NIH in the US. The RAC also began to review the protocols of gene therapy with the collaboration of the US Food and Drug Administration (FDA) (Cotrim & Baum, 2008). The RAC checked the merit and soundness of how the recombinant DNA technology had been applied scientifically. On the contrary, the FDA investigated the safety of the modified products, as well as, their process of manufacturing.
The establishment of the various regulatory bodies means that individuals were not allowed to carry out experiments on gene therapy the way they wished. Perhaps the various experts and government agencies were following the recommendations stipulated by Nirenberg in one of his papers. Anyone who attempted to carry out research on gene therapy without following obtaining approval or following the regulations faced the consequences. One of such individuals who found himself in conflict with the authorities was Martin Cline. In 1980, Dr. Cline attempted applying human gene therapy at the University of California, Los Angeles (UCLA) but the process was marred by controversy (Misra, 2013). Cline did not seek approval from the UCLA regulatory body, which made several critics to raise questions. In an attempt to play gene therapy, Dr. Cline conducted a transfer of recombinant DNA on two patients in Italy and Israel by inserting the beta-thalassemia gene into the cells of their bone marrow. Cline held the tests secretly but were later exposed by the Los Angeles Times. Upon exposure, Cline suffered severe consequences. He was told to resign from the chairman position he held in his department at UCLA on top of losing some grants.
It is important to note that the progress of gene therapy has been hindered mostly by ethics personnel and by critics with religious views. The critics with religious views argued that scientists would be undermining the role of God if they were allowed to carry on with the gene therapy unabated. Even up to now, scientists are still bound by regulations regarding the extent they can go with the application of gene therapy (Misra, 2013). Most of the time, gene therapy is used in the growth of food and inducing desirable genes in animals, mainly for commercial purposes. In human beings, significant control measures exist. Gene therapy is mostly used in the treatment of disorders. Whenever scientists try to apply the process on enhancing normal people’s features, the situation becomes a subject of controversy around the world. However, some experts perform the procedure in secrecy though they receive a lot of criticism should their actions be discovered. However, despite various protests from different people, gene therapy can also be used in human beings too since it has been used successfully in plants and other animals.
How Gene Therapy Can Be Used Today
Before establishing some of the ways through which gene therapy can be used, it is vital to understand the types of gene therapy, as well as, the methods which are used to transfer genes into humans. There are two types of gene therapy namely the germ line therapy and the somatic gene therapy. To start with the germ line therapy, germ cells (egg or sperm) are modified through the introduction of improved genes. That means that the induced changes are inheritable hence can be transferred from one generation to another. The advantage of this genetic therapy is that it is effective at curbing genetic diseases and other disorders that are inherited. However, there are technical challenges at the moment on top of ethical considerations. As mentioned before many critics that hold strong religious views do not support this type of gene therapy and it remains unknown whether germ line gene therapy will be attempted on human beings in the near future. The second type of gene therapy is known as somatic gene therapy (Daniel, 2013). In this process, therapeutic genes are inserted into a patient’s somatic cells. The difference here is that unlike in the germ line gene therapy, the effects of the modification are limited to the individual patient and cannot be transferred from one generation to another.
The challenge, however, sets in in the gene delivery process. Gene delivery refers to the process of getting the new gene to a patient’s target cells. Carmichael (2014) points out that a carrier molecule known as vector ought to be used to deliver the replacement gene. The gene delivery vector also needs to be precise and have the capacity to the replacement gene of the required size. The vector also needs to ensure that once the new or replacement gene is induced into the patient, it ought not to cause any allergic reaction or inflammation. Two methods are used to deliver vectors into the cells of patients; ex-vivo and in-vivo. In ex-vivo, extracted cells from the patient are used. Normal genes are first cloned into the vector. Secondly, the defective cells are removed and mixed with the vector that is genetically engineered. Lastly, the transfected cells are inserted into the patient to create protein which is required to fight the disease. In the in-vivo technique, cells from the patient’s body are not used rather; vectors that have the normal gene are induced into the blood stream of the patient to bind with the target cell.
It is also vital to understand that there are two types of vectors; the viral and non-viral vectors. The viral vectors are harmless viruses that are used to deliver new genes into patients. One of the ways scientists use viral vectors is by using harmless viruses to penetrate the cell membrane of cells under the disguise of a protein molecule (Cotrim & Baum, 2008). When the vector is lodged in the right location of the cell, it begins to issue instructions required to manufacture the protein that was missed or altered previously. In the non-viral vectors, direct DNA is injected in patients. Clinical trials are still ongoing to explore ways through which replacement genes can be injected directly into patients. Non-viral vectors are also the easiest way of applying gene therapy.
Regarding application of gene therapy, the process is mainly being used to treat disorders either inherited or developed. Although not much success has been achieved, experts are carrying out studies on how gene therapy can be used to treat cancer. Carmichael (2014) points out that the most possible way of getting vectors into cancer cells is through the use of viral methods. One of the challenges is that some of the viruses may cause diseases such as flu. But the most popular application of gene therapy is in the treatment of disorders. One of the disorders that have been treated through gene therapy is Sjogren’s syndrome (SS) which is regarded as the second most prevalent autoimmune disorder in the US. The disorder is characterized by the existence of a focal lymphoid infiltration of the cells of the salivary and lacrimal glands. The disorder is treated by the insertion of immunomodulatory genes into the salivary glands to increase the production of saliva.
How Athletes Can Use Genetic Alteration to a Physical Advantage
Athletes participating in highly competitive sports are usually tempted to use underhand methods to emerge winners. Already, some athletes have been diagnosed with symptoms of using drugs aimed at enhancing their performance. Experts argue that if athletes can use drugs to improve their performance, then it will not be long before they resolve to use gene therapy to their physical advantage (Wells, 2008). It is relatively easy to investigate whether an athlete has used drugs but it will be a challenge to establish whether an athlete has used gene therapy unless the act is exposed.
Some of the athletes, especially the sprinters, have demonstrated to the world about what is needed to win some races. Although practice and nutrition contributes significantly to winning, some traits also contribute considerably to the athletes’ victory. Still on the example of sprinters, tall and masculine athletes are likely to win races. Although the current less masculine athletes may not be able to change their genetic traits, they may consider having their children induced with modified genes. That would mean that they may use the germ line therapy (Loria, 2016). Although this type of gene therapy is still highly regulated, it may be practiced in the black market because of the profitability involved.
Gene doping may take different forms. First, people may want their children to develop certain abilities which they may not have themselves. They will then find it necessary to be induced with replacement cells so that they bear children with desirable qualities to compete in the various sports. Some athletes may also consult the services of genome experts to alter their muscle formation (Loria, 2016). Most of the gene therapy will involve direct injections with the desired genes. As a result, athletes may achieve new records that are not humanly possible. The integrity and validity of different sports will consequently be questioned.
In summary, gene therapy allows for the correction of certain deformations in cells that is not possible through the use of conventional means of treatment. The process is beneficial particularly in people with certain disorders that affects their wellbeing. However, the advance of gene therapy has variously been criticized by ethics people and religious individuals. One of the protest given is that gene therapy, especially that involving germ line therapy, is a way of undermining God’s creation. However, researchers have established that gene therapy can be used to treat severe diseases such as cancer and research is still being conducted to find out whether the process can be used to eliminate cancer. Nevertheless, gene therapy may be used by athletes in the future to gain physical advantage over their competitors. Athletes may use the black market to infuse genes with desirable qualities to compete favorably in sports. It is important that experts find out ways of checking signs of gene doping so that sporting competitions are not compromised.
References
Carmichael, L. E. (2014). Gene therapy . Minneapolis, MN: ABDO Publishing Company.
Cotrim, A. P., & Baum, B. J. (2008). Gene therapy: some history, applications, problems, and prospects. Toxicologic pathology , 36 (1), 97-103.
Daniel, S. (Ed.). (2013). Advanced Textbook on Gene Transfer, Gene Therapy And Genetic Pharmacology: Principles, Delivery And Pharmacological And Biomedical Applications Of Nucleotide-based Therapies (Vol. 1). World Scientific.
Loria, K. (2016). Humans of the future could be much faster than Usain Bolt or Michael Phelps . Business Insider . Retrieved 13 April 2018, from http://www.businessinsider.com/genetic-modification-athletes-improve-athletic-performance-2016-8?IR=T
Misra, S. (2013). Human gene therapy: a brief overview of the genetic revolution. J Assoc Physicians India , 61 (2), 127-33.
Wells, D. (2008). Genetic engineering in athletes. BMJ: British Medical Journal , 337 (7661), 63.
