Ethics of human gene therapy


Gene therapy remains a controversial topic in the society even though the experts have proven the effectiveness. The society keep asking "what gene therapy gives us" and "should it be legalized" although there are some countries permits this method. This research paper will introduce the reason why gene therapy should be legalized in treating the disease on the wide concept associated with the pros and cons. Discussion will focus on the effectiveness of gene therapy compared to conventional methods and the ethics of it in the human norm. The argument on cost effective of gene therapy might provide an irrefutable reason to the world in accepting this method of treatment. Besides this paper will also answer the religious party's statement saying that gene therapy is interrupting the God's work although it actually not. At the end of this paper, the society should once again think about what gene therapy can gives them and would be able to influence the others in accepting gene therapy. With the support from the society, the government and organization involved would consider to legalize gene therapy as future treatment in this challenging field.


"Prevention is better than cure."DesideriusErasmus

Back in the 1970, human genes can already be sequenced and cloned result from the advances in molecular biology. Scientists are looking for a technique to produce useful proteins, such as the protein lacking in diabetics; insulin, by introducing human genes to bacterial DNA. The custom-made bacterium which contains human genes then produce the subsequent protein, which then will be harvested and injected into patient who cannot produce it naturally. Scientists took the coherent step by trying to introduce the useful genes straight into human cells, focusing on diseases origin by solitary-gene defects, such ascysticfibrosis, hemophilia, muscular dystrophy and sickle cell anemia. However, this has been much intricate than altering simple bacteria, primarily because of the problems involved in carrying large fragments of DNA and delivering it to the exact site on the genome.

Nevertheless not even one of the human gene therapy products has the permission from The Food and Drug Administration (FDA) to be marketed. This is because not all of gene therapy experiment have hundred percent successful in clinical trials. Furthermore since the first gene therapy experiment began in 1990, there is only slight improvement has been made. To make it worst, when 18- year-old boy named Jesse Gelsinger died in 1999, gene therapy development eventually retard. The case started when he participated in gene therapy experiment for Ornithine Transcarboxylase Deficiency (OTCD). From the post mortem, he was died because of the various organ failures few days after the treatment. The reason why he died is because of the wrong stimulation of human immune system that is origin from the adenovirus carrier. (Human Genome Project Information, (2009)).

The researchers working at UCL Institute of Ophthalmology and Moorefield Eye Hospital NIHR Biomedical Research Centre have declared results from the first experimental trial in the world to test a ground-breaking gene therapy treatment for a type of hereditary blindness. The result proves that the experimental treatment is harmless and can recover the human sight. Although the scientist has yielded the excellent experiments of gene therapy in human kind, people still asking the controversial question, that is, why should gene therapy be legalized?

Many human diseases are results from the absence or improper occurrence of a protein. The scientists are trying to extract and construct these normal proteins through the recombinant technology and genetic engineering. The protein could then be administered to patients in order to compensate for its absence and actually cure the disease. Viewing from the positive angle, we will realize that gene therapy to treat diseases should be legalized because of its advantages. In fact, gene therapy is body-friendly, can reduce the cost for long term treatment and improve the quality of life without interrupting the human nature.

This report will show strong evidence of why gene therapy should be legalized to treat patient. Basically the research findings are confined throughout the world because it would provide the people better understanding on gene therapy and give them sufficient evidence to put their support on the topic.

Body of report

Genes are the basic physical and vital units of heredity, which are carried on chromosomes. Genes are specific sequences and arrangement of DNA that encode information on how to manufacture proteins. Without genes, the protein can't be produced by the body. Although genes seems to be the main point in making the substances, it's the proteins that perform mainly life functions and even build up the most components of cellular structures. In treating the disease, genes are modified in gene therapy so that the encoded proteins are able to carry out their ordinary functions and eventually eliminate the disease. (Goodenough. J., (2005)

Gene therapy is body-friendly

Gene therapy include the injection of a new gene which totally foreign from the body system. The nature of body system is, when subjected with a new foreign object, it will encounter the object by producing specific antibodies. So when a new gene is introduce into the body, the cell of the immune system will be stimulated to produce the antibodies against it. Some response might actually cause death to the person. It has been proven that a man died from multiple organ failures 4 days after starting the treatment. The scientist post-mortem shows that he died because his body has been triggered by severe immune response which leads to organs failure. ("Human Genome Project Information" (2009) ). Besides that, in human body, when foreign substances came in, the stimulated cell will proliferate to produce memory cells. Memory cell stores information about the substance. Once the system is stimulated, there is no turning back. The memory cells will response rapidly upon subsequent exposure to the same substance. This technique can't be repeated to the same patient because the new gene will be eliminated from the body thus this will reduce the effectiveness of gene therapy.

However, viewing from the positive angle, the advantages of gene therapy overshadow the disadvantages. Gene therapy is the technique for correcting the fault gene responsible for the disease progression. One of the ways is the insertion of new gene to the target cell so that the cell can produce the correct proteins for the diseases and replace the fault gene. In this process the production of protein is somehow natural compared to conventional methods. The cell produces the protein after translating the genetic code in the new gene. This treatment had surprised many people. As an example, there were three young adult suffering from an unusual genetic form of severely impaired eyesight called Leber's congenital amaurosis, in which a mutated gene prevents the retina from manufacturing a nutrient essential to eye health. These peoples actually legally blind with a small visual field. After undergone gene therapy by a group of doctor from University of Pennsylvania School of Medicine, those patients show a positive result. They can see much clearly from day to day and totally recovered after several weeks. ("Gene Therapy to Reverse Blindness "(n.d.

Furthermore in conventional method, synthetic or artificial drugs are widely used to treat the disease. Physically the disease seems to be treated. The truth is these drugs don't cure a patient of the disease but their just limit the progression and symptom. So by using gene therapy which totally natural to treat the disease, it would be less unnatural substances working in the body. Moreover patient who chose to use drugs to treat their disease have to take those unnatural subtances continuously throughout their life. From the study, most drugs have side effects to the consumer. Cystic fibrosis for example is an inherited diseasethat causes the body to make mucus that's extremely thick and sticky. This thick, sticky mucus actually works like a lubricant and helps defend the body's tissues. But when excessive mucus is produced, it blocks passages in most of the body's organs and causes infection to set in. Usually to treat this disease, the patient would have to take drugs called Triamcinolone. This drug has some side effect to the consumer after taking for a long term. Study shows that this drug cause severe allergic reactions such as rashes, difficulty in breathing, tightness of chest muscle and also psychotic behaviors. ("Drugs Side Effects" (2000)). However if gene therapy is use to treat this disease, the situation will not happen to the patient. If things work out precisely, this equates into far fewer side effects. Therefore using gene therapy to treat some diseases is extremely much better than using conventional methods as it is somehow natural and has fewer side effects. For no doubt, gene therapy should be legalized.

Gene therapy is cost effective for long term treatment

Besides, peoples who oppose the gene therapy may claim that the cost is too high compared to the conventional methods. Gene therapy is a new biotech revolution that requires the expertise to make it work. There are two things that really need huge sum of funding in gene therapy. The first thing is the production of certain drugs or gene of interest. Early in 2004 the government of Britain had invested an extra 100 million in the gene therapy development in order to fasten the result. Based on one of the scientist in University of Sussex, Dr. Paul Nighthgale claim that the outcomes of the research is actually not impressive at all then the rhetoric suggest. For the treatment of diseases, most patients claim that the gene therapy is a very costly procedure especially for private hospital facilities. Furthermore gene therapy is not available in all country throughout the world. There are only some country in the world that have the facilities with full of expertise. This is because the cost to build facilities is extremely high and there are countries which prefer not to build it and use the money for other prior purposes. So by including the direct and non- direct cost of the treatment it is assume that gene therapy may cost up to $100,000 per individual and is said to be much expensive treatment compared to conventional methods. (Lane (n.d.)).

However some patient who has undergone gene therapy may definitely agreed that gene therapy is actually cost effective rather than conventional methods. Why is that so? This is because gene therapy does not need several times of treatment like others. In gene therapy treatment, patient will be treated once in a life time. It is really worth to the patient with the genetic disorder because the cost is acceptable. Compared with conventional treatment, it may seems cheaper than gene therapy but when viewing for a long term treatment, it is actually much expensive than gene therapy. This is because conventional methods usually the drugs is use to treat the disorder. Surprisingly these drugs actually do not cure the disease but only limit the progression. So the patient with the disorder needs to continuously buy the drugs when it finished. Compared to conventional methods, gene therapy could provide a cost effective treatment alternative to the repeated treatment for the patient with the drugs and at the same time prevent the complication of using synthetic drugs.

Gene therapy improve human life

The ethics of using gene therapy is a controversial issues that had been hotly debate over a few decades. It started in 1960s when the recombinant DNA research began. Long time before the technology of gene therapy became available, the bioethics emergent discipline was focused only on the gene tic engineering. From times to times the technolgy of genetic engineering keep increasing corresponding to the advancement and succession of the research. Most of ethical issues were raised by the religious party. They claimed that man had neither power nor right to create or modify the God's work. So they fervently opposed the human gene therapy by saying that man is trying to tamper or corrupting God's work by interfering with the genetic code of an individual. (Bose, D. (2009)). Besides, gene therapy also claimed to be the invader of human privacy. This is because the scientist also may maneuver the gene of interest into genetically control traits in human offspring that is far from the basic purposes which is for health. For example, the insurance company will be able to know the genetic disease of the customer. The customer may be discriminate to pay higher insurance premiums to get the insurance or maybe will be denied from applying the insurance. This proved that gene therapy really cause the invasion of individual privacy.

Viewing from the positive angle gene therapy actually gives more benefit rather than disadvantages. From the past research by the expert, gene therapy obviously have greater success rate compared to conventional methods and least risk. (Parmar. H. (2006)).The doctor that can practice the procedure is the chosen one which has highly expertise on gene therapy. With the expertise and the suitable condition, mutation or any other failure won't occur. There is no doubt because before any procedure in gene therapy starts, the expert will first determine the pros and cons to the patient. Human gene therapy is an intricate process that really needs a lot of technical skills and scientific knowledge. People who opposed the gene therapy by saying that man is interfering God's work cannot be acceptable as it is aimed to eliminate and prevent hereditary disease. This will increase the quality of life of the patient. Moreover the patient is giving the freedom to choose whether they want the information of their gene to be exposed or not. (Miller, H I., (1998)). So the issue of privacy invasion is not the matter as it totally depends on patient permission. Regardless of all the criticisms, the world can't deny the fact that human gene therapy can eternally eliminate the diseases that keep hunting the human civilization for centuries.


The society claimed that there are still little firm evidences that can be used to convince them about what gene therapy actually can do and how safe it is. Although current results of gene therapy is very successful if we compared to the last few decades but its still not enough to show the miracle of gene therapy. The best ways to convince them is by doing more experiment to both animal and human and reveal the result to the public. It would provide sufficient evidences to manipulate their mind in accepting gene therapy.

Viewing from ethical issues, gene therapy does not interrupting God's work and human privacy. There are ways to explain this to the society so that they will change their negative view on gene therapy. One of the ways is by making an ethical guidance to the medical practitioner in the treatment of genetic disorders. This guidance will guide the doctor and also the patient in making decision on the treatment so that there is no misunderstanding and misused of knowledge. Through this way also, one body of counselor should be formed. The purpose of this body is to provide an advice to the doctor about the procedure and the patient about the pros and cons.

Besides that, gene therapy is for sure a very profitable field in the future. For now, the cost of treatment using gene therapy is said to be much expensive than conventional methods. In overcoming this problem, the government should encourage private organization to invest and sponsor in this field so that the cost will be equally distributed to both government and the organization. There is still huge space for gene therapy to improve and this of course need the support from all people. If all the people works together on the right track, gene therapy will become a major way in treating the disease especially genetic disorder.


Nowadays it is crystal clear that many exhilarating innovation of gene therapy are rising. Although these entire products have the prospective for incredible patient benefit, yet these products still have the unknown risks. So to ensure the patient get the safest treatment, the scientist must accept all the responsibility of the procedure so that irrational risks can be prevented. In a nutshell, gene therapy is a very vital method for treating patient with disease especially genetic disorder. From this research, gene therapy can be seen as the new revolutionary technique advancing in the genetic engineering to treat the diseases. Current treatment so far has given excellent results from all standpoints. Upcoming endeavors should be focusing on how to improve the efficiencies of gene therapy so that the society will not argue the importance of this method in treating the patient. Even though human gene therapy treatment raises many controversial, the outcome of this technique is symbolized as a very high level of expertise and should be accepted. Viewing from the positive angle, the advantages of gene therapy overshadow the disadvantages. Gene therapy should be legalized because it is body-friendly, cost effective and improve the quality of life.


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  • Kotz, D., (2009) "Gene Therapy to Reverse Near-Blindness" U.S. News and World Report, viewed on 28 February 2010 <>
  • Lane, A., (n.d.) "Healing the wound that never heal" Markula Centre for Applied Ethics, viewed on 20 March 2010 <http: //>
  • Matthews, R, (2004) "Gene therapy is just an expensive myth, claim scientists" Centre for genetics and society, viewed on 22 March 2010 <>
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  • Parmar, H, (2006)"Gene Therapy Makes a Comeback" Clinical Discovery, viewed on 2 March 2010 <>

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