What is Gene Therapy and what is it used for?
Gene therapy (otherwise known as somatic gene therapy or therapeutic gene therapy) is a process that is used to correct defective genes responsible for development of various diseases. (www.accessexcellence.org/RC/AB/BA/Gene_Therapy_Overview.php) There are two main types of gene therapy; somatic (body) cell gene therapy and germ line gene therapy. As it suggests, somatic gene therapy is correcting the faulty genes of normal body cells. Germ line therapy is altering genes in gametes. Everyone on earth carries a few defective genes. Most of us are unaware of this fact however, unless we are afflicted with a genetic disease. Approximately one in ten people have, or will develop, a disease that is a direct result of faulty genes. (www.ghr.nlm.nih.gov/handbook/therapy/genetherapy)
The possible future uses for gene therapy are seemingly endless, with current research of gene therapy underway on how it can be used in the treatment of cancer, genetically caused blindness, and sickle cell disease. (www.cancer.gov/cancertopics/factsheet/therapy/gene)
What are the concepts and processes involved in Gene Therapy?
The general process by which scientists correct these genes are by that the absent or faulty gene is replaced by a working gene, so the body can make the correct enzyme or protein and therefore eliminate the cause of the disease. (www.accessexcellence.org/RC/AB/BA/Gene_Therapy_Overview.php)
There are two methods used to insert genetic material into human chromosomes. The first of which, called in vivo technique, a normal gene is inserted into the chromosome to replace the abnormal, disease causing one. A carrier molecule (called a vector) is used to transfer the correct gen...
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...various scientific organizations, such as The Society of Molecular Biology.
www.nature.com is also an internationally recognized website, based on the most trusted and popular scientific journal, Nature.
The books I used, namely, ‘Human Germline therapy’, ‘Gene Therapy Protocols’, and ‘Altered Fates’ all contain controversial material regarding the use of this technology, but I believe it was important to include this information as it gives a more diverse view on the issues of Gene Therapy and the techniques that are required.
‘www.catholicscience.com’ and ‘www.catholic.org’ both go against the scientific method in their reasoning about Gene Therapy as it is important to give perspective of more than just the scientific community. The religious community plays an important role in society so it is important to include their views on this matter.
Germline gene therapy is where the correct "good" gene is inserted into the germline in place of the defective "bad" gene, and when reproduction occurs the gene will be passed on to the progeny. Inserting the "good" gene into the very early embryo sta ges of development allows for both germline and somatic cells to be corrected. Government has limited the research to only somatic cell gene therapy such as performed in Cystic Fibrosis research.
Gene therapy is reinserting certain genes that helps deal with genetic diseases. There are three basic forms of this gene therapy. The first is Gene Inactivation Therapy in which the transferred gene neutralizes the proteins and evens out the amount or rids of the defective proteins. Another type is Gene Augmentation Therapy where the original form of the gene or the normal form of the gene is inserted into one of the cell’s chromosomes. This procedure is used normally when a gene with little activity or a deleted gene is the cause for the genetic disease. The third type of gene therapy is Gene Replacement Therapy. This form is used when the genetic disease involved specific genes that are necessary for proper functioning. The normal gene being put in place of the mutant gene accomplishes this form of gene therapy.
... fight the disease. It is crucial that regulation be a necessary component of gene therapy research and applications. In hopes that the government can regulate and can receive this treatment, not restricting it to people that has serious genetic diseases. Gene therapy will change the field of medicine from what it is today. As scientist discovers more genes and their functions, the potential of this treatment is limitless. Though gene therapy is an auspicious treatment choice for numerous diseases (including inherited disorders, some types of cancer, and certain viral infections), the procedure remains precarious and is still under study to make sure that it will be safe and effective. Thus government regulators and scientist must take a lead role in adopting a practical approach to address these issues and determining the correct procedures for dealing with them.
Human gene therapy is a method used in the medical field that treats diseases at a molecular level, by solving the source of the problem; our genes. Today, diseases and disorders are commonly treated by solving the symptoms, the surface of the problem. Many disorders and diseases are caused by defective proteins and within those defective proteins are damaged and defective genes. These defective genes can be treated through gene therapy. Gene therapy is not new and has been developed and improved by researchers for the past couple years. Being an experimental technique, gene therapy also has its pros and cons, but so far is showing positive and rising success rates.
Just as there are different types of people who look at one glass of water and describe it as half full or half empty, the public has many different views on the future of our society. Gene therapy is also a glass that can be viewed in different angles – different perspectives. Some say it has great potential to shape the ideals of our future, while others believe it signifies intolerance for disabilities, imperfections that supposedly deplete from a person’s interests, opportunities and welfare (quoted by Peter Singer, xviii). This global issue has brought people with different opinions in the open, arguing their views using history, morality and foresight.
Gene therapy is a technique which has developed in the wake of recombinant DNA (rDNA) technology. It is a process which results in the correction of a genetic disorder by the addition of a piece or fragment of DNA into the genetic material of a living, functioning cell. A mere thirty years ago this concept belonged to the realm of the human imagination made manifest in the works of science fiction. Today it belongs to the realm of the human imagination made manifest in the works of science, period. It is mind boggling to try to comprehend the far reaching effects of gene therapy. How is it affecting society? Who will benefit from its use? Should it be used at all? Should research continue? How do we answer all of these questions? The answers are not readily available, nor are they black and white, but an attempt at finding some solutions must be made. Before exploring this line of thought further, a basic understanding of the technical aspects of gene therapy is essential.
Gene therapy works in three ways; it works to replace a missing or defective gene with a normal one, replace a faulty gene so that it will function properly and it works to activate and deactivate a gene, allowing it to “switch” on and off. Gene therapy is done by the deliverance of a gene to a cell via a carrier, or vector, such as a virus. Scientists lean more towards using a virus because they can seek out particular cells and transfer pieces of deoxyribonucleic acid into them. Scientists also take advantage by deactivating their harmful characterizes and modifying them to carry particular gene into designated cells. After gene therapy is done, the genes can then stimulate the production needed for standard functioning, allowing that gene to return to its previous normal state. Therefore, if a patient were to be in the beginning stages of cancer, gene therapy would seek out the cancerous gene and replace it with a healthy one and minimizes the disease from
In September 14, 1990, an operation, which is called gene therapy, was performed successfully at the National Institutes of Health in the United States. The operation was only a temporary success because many problems have emerged since then. Gene therapy is a remedy that introduces genes to target cells and replaces defective genes in order to cure the diseases which cannot be cured by traditional medicines. Although gene therapy gives someone who is born with a genetic disease or who suffers cancer a permanent chance of being cured, it is high-risk and sometimes unethical because the failure rate is extremely high and issues like how “good” and “bad” uses of gene therapy can be distinguished still haven’t been answered satisfactorily.
In this paper, I will argue that genetic therapies should be allowed for diseases and disabilities that cause individuals pain, shorter life spans, and noticeable disadvantages in life. I believe this because everyone deserves to have the most even starting place in life as possible. That is no being should be limited in their life due to diseases and disabilities that can be cured with genetic therapies. I will be basing my argument off the article by “Gene Therapies and the Pursuit of a Better Human” by Sara Goering. One objection to genetic therapies is that removing disabilities and diseases might cause humans to lose sympathy towards others and their fragility (332). However, I do not believe this because there are many other events and conditions in society that spark human compassion and sympathy towards others.
Although humans have altered the genomes of species for thousands of years through artificial selection and other non-scientific means, the field of genetic engineering as we now know it did not begin until 1944 when DNA was first identified as the carrier of genetic information by Oswald Avery Colin McLeod and Maclyn McCarty (Stem Cell Research). In the following decades two more important discoveries occurred, first the 1953 discovery of the structure of DNA, by Watson and Crick, and next the 1973 discovery by Cohen and Boyer of a recombinant DNA technique which allowed the successful transfer of DNA into another organism. A year later Rudolf Jaenisch created the world’s first transgenic animal by introducing foreign DNA into a mouse embryo, an experiment that would set the stage for modern genetic engineering (Stem Cell Research). The commercialization of genetic engineering began largely in 1976 wh...
[7] Stock, G., and Campbell, J.. "Engineering the Human Germline: an Exploration of the Science and Ethics of Altering the Genes We Pass to Our Children, New York; Oxford University Press, 2000. back
Since its inception, gene therapy has captured the attention of the public and ethics disciplines as a therapeutic application of human genetic engineering. The latter, in particular, has lead to concerns about germline modification and questions about the distinction between therapy and enhancement. The development of the gene therapy field and its progress to the clinic has not been without controversy. Although initially considered as a promising approach for treating the genetic of disease, the field has attracted disappointment for failing to fulfil its potential. With the resolution of many of the barriers that restricted the progress of gene therapy and increasing reports of clinical success, it is now generally recognised that earlier expectations may have been premature.
Bergeson, E. (1997) The Ethics of Gene Therapy [Online] Available at: http://www.ndsu.edu/pubweb/~mcclean/plsc431/students/bergeson.htm [Accessed 14 July 2011]
Due to the fact that the field of biotechnology is very serious and potentially dangerous, rules must be set down in order to keep the research in check. The high risk research of genetic therapy needs guidelines that have to be followed in order to keep the study just. The articles that are discussed in this essay focus on ethical issues and ideas that should be followed in the field in order to keep research safe and valid.
The topic of gene therapy in humans is one that is highly debated due to the ethical implications connected to the science. Both sides of the debate have various reasons for their position, but the main factors come down to the ethics of changing someone’s genome and the consequences that accompany the altercations. The two types of gene therapy, somatic and germ-line, are seen in different light. There is more debate over germ-line therapy because the alterations have more consequences than somatic gene therapy. There are many moral and ethical decisions that need to be considered before gene therapy can be widely accepted.