Introduction
Gene therapy is a technique which has the prospect of providing an advantageous method in curing genetic diseases. Although still in development, successful trials have occurred, and conceptually, the idea being tested is ideal in the fight against lethal diseases such as haemophilia, cystic fibrosis and cancer. However, much controversy surrounds the idea, due to its undeveloped nature, potential biological risks and hefty failures in the past. The use and development of gene therapy in the future is a deliberated topic. This investigation aimed to consider potential risks and benefits, giving rise to the question: gene therapy; friend or foe?
Biological Background
Gene therapy is a process used to cure diseases and disorders caused by mutated or defective genes in patients, which alters or inhibits the production of essential proteins used in normal functionality (Hunt, 2008). Fundamentally, the treatment involves the delivery of functioning DNA into cells, which incorporates into the genome and replaces the defective gene, or repairs, alters or deactivates genes causing the disease (Genetic Home Reference, 2014; Hunt, 2008).
Gene therapy employs vectors to deliver functioning genes into cells, a ‘vector’ being a vehicle in which DNA can be transferred. They can be viral or non-viral, and treatment can occur in vitro and ex vivo (inside and outside the body) (ScienceDaily, 2014; University of Utah, 2014). The use of viral vectors mimics the survival techniques of viruses. A virus is a miniscule particle containing genetic material; lacking material to reproduce on its own, it infects a host, injecting its DNA or RNA, and utilises the host’s cellular machinery to reproduce (Freudenrich, 2014). In gene therapy,...
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The beginning of Gene Therapy began in the late 1980’s, which was completely unsuccessful. In the fall of 1999 the death of University of Pennsylvania trial participant, Jesse Gelsinger was followed by much public outcry and legal problems that put an immediate halt on all gene therapy research. The reason Jesse was being treated with Gene therapy was to attempt to cure the teenager’s rare liver disease.
Gene therapy is an experimental technique that allows doctors to insert a gene into a patient’s cell rather than using drugs or surgery. Gene therapy is a process of which defective or undesired genes in the body with “normal” genes. A vector is re-engineered to deliver the gene to a target cell. Then the gene is transferred to the cell’s nucleus and must be activated in order to function. The main focus of gene therapy is to replace a lost or improper gene with a new functional copy into a vector that is inserted into the subject’s genome by way of penetrating its DNA. Gene therapy can be done outside of the body known as ex vivo by way of taking cells form patients bone marrow or blood and then growing them in a laboratory. Thus the corrected copy of the gene is inserted into the cells before being put back into the body. Gene therapy can also be done in vivo which can be done directly to the patient’s body. The word gene therapy really defines the management of genetic information that is encased in the cells, however, in most recent procedures the available technology is closely related in adding new genetic information, and many researchers favor the term gene transfer rather than gene therapy to mirror the reason that the purpose of gene work cannot always be therapeutic.
Gene therapy works by introducing new and functioning genetic material to damaged genes to help it function and to produce beneficial proteins. If a gene is inserted directly into a cell, it usually will not function. So to complete this task, a vector, a modified virus is used to carry and deliver the new gene. There are two different categories of vectors than can be utilized in this process; recomb...
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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.
So Gene Therapy, being a process solely aiming to heal genetic issues in human beings, is a technology which is desirable for our modern society. If a method can be produced, which cause...
Viral vectors use viruses to transport a modified gene into a patient's body. They are right now be...
"The aim is to decrease the fear of a brave new world and to encourage people to be more proactive about their health. It [Gene therapy] will help humans become better physically and even mentally and extend human life. It is the future” (Hulbert). Dr. Hulbert, a genetic engineer, couldn’t be anymore right; more time, money, and research needs to be put into gene therapy and genetic engineering, since it can cure certain illness and diseases that are incurable with modern medicine, has fewer side-effects than conventional drugs or surgery, and allows humans to be stronger physically and mentally at birth. Gene therapy or genetic engineering is the development and application of scientific methods, procedures, and technologies that permit direct manipulation of genetic material in order to alter the hereditary traits of a cell, organism, or population (NIH). It essentially means that we can change DNA to make an organism better. Genetic engineering is used with animals and plants every day; for example with genetic...
Every year, the rate of mortality increasing because most diseases may lead to death if not treated early. One of the methods that can be used to cure some diseases is by using the treatment known as gene therapy. Based on Pruitt’s (2008) study, numbers of inherited and acquired diseases were reduced since gene therapy has the ability to provide new treatments to cure them. According to Shi and Zou (2008), gene therapy is defined as expression of protein or interrupts the synthesis of protein in cell by transferring the genetic material into a host in order to treat or prevent a disease. Besides that, Kelly (2007) stated that an “abnormal” hereditary disease-causing gene in an individual’s cells and tissues is treated and used gene therapy by to replace them with a “normal” gene. Around 1970’s, idea to use “genes” as “drugs” for human therapy was originally from United States (Giacca, 2010). Moreover, there are some objectives in using the gene therapy as a treatment. First, gene therapy is used to cure or slow the progression of disease by introducing the genetic material into target cells and next objective is to aim at the direct correction of endogenous genetic defects by delivered some additional copies of a gene (Pruitt, 2008; Giacca, 2010). Furthermore, Yadav and Tyagi (2008) found that there are two types of gene therapy which are germline gene therapy and somatic cell therapy. As stated by Shi and Zou (2008), therapy that involved modification of any cells in a patient’s body is called as somatic cell gene therapy while germ line gene therapy is therapy that involved modifying of human eggs or sperms that pass genes on to future generations. Other than that, animal tissue culture is used to test the effective...
Gene therapy is a method used by doctors that ‘uses genes as medicine’. It repairs faulty or damaged gene copies by transferring a therapeutic or working gene into specific parts of faulty gene copy. This means that gene therapy can be used to replace a faulty gene or to introduce a new gene whose function is to modify the clinical course of a condition or to cure a problem in the gene (Refer to image 2). Image 1 shows how gene therapy is completed using an adenovirus vector while image 2 explains the method of gene therapy and shows how cells are removed, altered and injected back into the patient. Gene therapy has potential to treat and cure many medical conditions such as various types of cancer, degenerative diseases and viral infections such as AIDS. Research into gene therapy still needs to be done before this approach to the treatment of certain conditions will realise its full potential. Trials are being conducted in the United States and Europe and a modest number initiated in other countries, including Australia. The majority of these trials are focussing on treating acquired conditions such as cancer (European Society of Gene & Cell therapy, 2011).
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.
Gene therapy has become an exciting and controversial issue on the scientific and medical horizon. Science offers new technologies that, in the future, will be able to treat and cure common genetically passed diseases. However, as it is an extremely broad subject, some time must be dedicated to its interpretation and explanation. First, a general definition of gene therapy is required. Genethics, the Clash between the New Genetics and Human Values, by David T. Suzuki and Peter Knudtson, defines gene therapy as "the medical replacement or repair of defective or faulty genes in living human cells." It is not really so elementary as the definition would imply. Within gene therapy there lie certain aspects, some more controversial than others, some more achievable and probable than others. The ethical question must be addressed at each turn. However, all of this will be discussed at greater length subsequently.
Gene therapy enables patients to survive incurable diseases. In the field of genetic diseases, ADA-SCID, CGD and hemophilia are three main ones. ADA-SCID is known as the bubble boy disease. CGD is related to immune system that would lead to fungal infections which are fatal. Patients with Hemophilia are not able to induce bold bleeding (Gene therapy for diseases, 2011). Gene therapy also has good effects on cancer treatment and neurodegenerative diseases, which include Parkinson’s disease and Huntington’s disease. Viral infections, including influenza, HIV and hepatitis can also be treats by it (Gene therapy for diseases, 2011). According to the Science Daily in 2011, gene therapy now can apply to heart failures and neurologic diseases as well.
One of the biggest concerns involved in gene therapy in humans is the lack of knowledge and the possibility for consequences later on or i...
Gene therapy poses many risks, but may prove the ideal solution for countless diseases. As seen throughout the past few decades, gene therapy can cure genetically inherited diseases by introducing therapeutic genes into the body. Critics may argue that there are moral and ethical problems associated with this novel technique, but for the most part scientists realize the importance this advancement will have. Gene therapy may be the key to curing dozens of diseases, and has endless possibilities, but more research is needed before its safe or accepted as common practice.