Analysis of the Pros and Cons of Gene Therapy - Research Paper Example

Insert Introduction Gene therapy can be defined as a form of disease treatment in which genes of the cells in the body are changed. In gene therapy, a suitable nucleic acid polymer(s) is inserted into the cells of a patient so that the polymers can treat the disease by interfering with protein expression (that causes the disease) or by themselves expressing as proteins or by correcting the mistakes in genes that could be resulting in an ailment. The commonest gene therapy involves substituting a mutated gene (that is causing disease) with a new functional gene obtained from DNA that is deliberately inserted into the cells to encode this new helpful gene. A vector is used in gene therapy as a package for delivering the useful DNA into the cells of the body (Mayo Clinic, 1). In line with the current challenges that continue to plague the field of healthcare such as increased incidences of antibiotic resistance, gene therapy will become a major treatment strategy in the future management and control of ailments. There is bound to be a lot of debate and emotion surrounding whether mankind should or should not embrace this treatment regime as diseases will become untreatable through conventional ways. With advanced knowledge of the subject, however, gene therapy will be useful in the future management of diseases. When gene therapy was initially thought of in 1973, the pioneers of the concept called for utmost caution in the attempts to use this technique on humans. A 1990 United States experiment treated a patient (DeSilva Ashanti) of ADA-SCID in an FDA-allowed procedure. Initially, failures with the technique led to many people writing it off but these assumptions have since changed drastically following several successful trials in the period after 2006 (Kelly, 348). As of 2014 January, over 2,000 trials were actively being pursued. Gene therapy is an important issue in healthcare as it offers immense hope in finally treating diseases that are currently untreatable such as HIV/ Aids and cancer (Kelly, 377). However, there are feared a number of consequences, especially manifesting in future generations, should the application of gene therapy go south (since very little is known about it). Gene therapy should therefore be a concern to every human being in any part of the world as we have a duty to the consequent generations. About Gene Therapy Genes house one’s DNA which is responsible for a number of human functions and characteristics. In this light, therefore, if a gene is mutated or is simply not functioning properly, diseases can result. Basically, knowledge of gene therapy aids in treating diseases by replacing a gene that is retarded/ faulty, adding a gene to an existing gene pool to enhance disease treatment and or promoting the ability of the body to fight off a disease. Gene therapy has been successfully used to treat a number of previously difficult conditions such as X-linked SCID, adenoleukodystrophy, acute lymphocytic leukemia, chronic lymphocytic leukemia, Parkinson’s disease, haemophilia, multiple myeloma, ADA-SCID and Leber’s congenital amourosis, among others. This technique of treatment further holds promise in the future management of presently difficult medical conditions such as cancers, HIV/ Aids, heart diseases, cystic fibrosis, diabetes, and so on (Mayo Clinic, 1). Medical professional are yet to underline when and how gene therapy can be effectively employed to manage diseases with gene therapy only still used in most cases as clinical trials. Researchers are still studying gene therapy in terms of its potential when mutated genes that cause disease are replaced with new functional ones (for example, the gene p53 which suppresses the growth of cancer cells when mutated results in cancer. Therefore, finding a means of replacing the mutated p53 gene could be game changing and perhaps final in the search for the cure of cancer), fixing mutated gene (by inactivating faulty ones that result in disease and/ or activating healthy ones that inhibit disease) and by making disease cells more recognizable by the immune system which then fights off the disease (Carmichael, 345). Gene therapy can be divided into two groups; somatic gene therapy and germ-line gene therapy. In somatic gene therapy, the selected treatment genes are introduced into body cells (non-sex cells) of the patient. In this case, genetic alterations are only in the treated person and are not inherited down the next generations/ offspring. This treatment is individual and for this reason, it is the most commonly pursued and studied form of gene therapy. Diseases caused by single gene problems such as cystic fibrosis, immunodeficiency, and so on, are best treated through this method of gene therapy with a plethora of clinical trials worldwide currently in advanced steps. As for the germ-line gene therapy, germ cells (eggs and sperms) are altered when new genes are introduced and integrated into their genomes. Germ cells reproduce and form the cells of the body and thus in germ-line gene therapy, all the organisms’ cells attain this genetic variation. This means that in this case, modifications are passed into offspring and other consequent generations. Because of this, though the method offers more efficiency that somatic gene therapy, the process has been widely condemned on ethical and technical arguments and also because of its greater risk and the little knowledge about these potential risks to the generations of the future (Carmichael, 365). New functional genes can be delivered into cells in gene therapy using viral vectors or non-viral techniques. A viral vector with selected gene(s) introduces the gene into the host through replication mechanisms. Some of the viral vectors that have been used thus far include adeno-associated virus, vaccinia virus, herpes simplex virus, lentivirus, adenovirus and retrovirus. Although non-viral techniques have low levels of gene expression and transfection, they are known to have low immunogenicity in the host and can be essential when large scale production is needed. Examples of non-viral techniques include the use of lipoplexes, inorganic nanoparticles, oligonucleotides, and dendrimers, naked DNA injection, gene gun, magnetofection, sonoporation and electroporation. Some of the common technological problems encountered in gene therapy include immune responses lowering the effectiveness of gene therapy (when they sense a foreign object), the nature of gene therapy to be short lasting (as in somatic gene therapy), viral vector complications, disorders of multi-gene nature, restrictions in several world nations, risks of causing cancers and the high costs of treatment through this method (Kelly, 411). Issues and Perspectives in Gene Therapy There are a multitude of opinion and facts that have been advanced regarding the use of gene therapy as a human treatment regimen. Those who support gene therapy assert that through it, mankind has hope of curing various ailments and genetic disorders. In addition, these proponents argue that through gene therapy, diseases can be eternally wiped out of the earth as these new and disease proof genes can be passed down through generations, as in germ-line gene therapy, thus eradicating diseases. To them, gene therapy offers mankind untold and unbelievable potential in terms of health care (Kelly, 469). Moreover, breakthroughs in trials have been witnessed all around the world that supports these optimisms. According to the Science Daily (2015, p.1) health researchers, in a trial of 9 boys (7 successful), noted that the technique is able to redeem the immune levels of children with SCID-X1 mutations. In another trial, by the University Of Pennsylvania (School Of Medicine), gene therapy was effectively used to improve the eye sights of several people (including 5 children) with Leber’s congenital amourosis (Penn Medicine, 1). On the other hand, there are equally several arguments concerning gene therapy from those who oppose its pursuit as a treatment mechanism for mankind’s diseases. For one, this vehement opposition stems from the fact that there is very little known about gene therapy which does not warrant a robust and unrestrained use of the technology. It is widely understood, even amongst researchers, that more comprehension and research is prudent in matters gene therapy before it is accepted as an integral medical tool and a way of life. To further aggravate issues, gene therapy trials have sometimes been hugely ineffective (Carmichael, 400). Many more people fear that gene therapy could be employed to permanently alter human capabilities for destructive purposes such as in building of indestructible armies of genetically engineered soldiers. In the very wavelength, many equally express their reservation arguing that it will permanently alter/ damage the human gene pool and with gene therapy healthcare, as expensive as it is forecasted to be, becoming a remedy only for the rich. Furthermore, secondary and equally serious medical complications can arise from the use of viral vectors during gene therapy in which case, viral vectors can target the wrong/ additional cells thus damaging these healthy cells and causing other conditions like cancer. In addition, a viral vector can re-attain its virulence once inside the host patient and infect the host. It can also cause a tumor if inserted in the wrong part of DNA (as has been seen in certain trials) and can equally elicit unexpected immune responses which can have fatal effects. Gene therapy is not fully safe and has issues in finding precise ways of introducing genes into cells, aiming at the right cells and minimizing the side effects of therapy. Conclusion There is quite a number of overwhelming issues (and challenges) concerning gene therapy. While some people (especially religious groups) assert that gene therapy is an attempt to play God and alter his creation (genetic design) which shall be inevitably punished, other maintain that it is a major advancement for the human race that should be grasped with both hands. A thorough analysis of the pros and cons of gene therapy still leaves one hesitant and unsure to claim that the advantages outweigh the disadvantages and vice-versa, and with more advancement in the field, the subject of gene therapy is set to spark greater divisions of thought (Carmichael, 418). Works Cited Carmichael, L. E. Gene Therapy. Minneapolis, MN: ABDO, 2014. Print; 345, 365, 400, 418 Science Daily, "Gene Therapy Shows Promise for Severe Combined Immunodeficiency." Science Daily Web, 23 Mar. 2015. Kelly, Evelyn B. Gene Therapy. Westport, Conn.: Greenwood, 2007. Print; 348, 377, 411, 469 Mayo Clinic, "Gene Therapy." Definition. Web, 23 Mar. 2015. Penn Medicine, "Penn Medicine News: One Shot of Gene Therapy and Children with Congenital Blindness Can Now See." Penn Medicine News: One Shot of Gene Therapy and Children with Congenital Blindness Can Now See. Web, 23 Mar. 2015. Read More