Gene Therapy: The New Vaccine
ABSTRACT
This research paper details what gene therapy is and what it is (and potentially will be) used for. It also describes the gene therapy techniques and delivery methods that have been clinically tested and/or verified by scientists and the ideas that stimulate activity in the field in the race to perfect gene therapy methods and their application, as well as telling about the beginnings of its clinical testing and where this budding technology is headed. Finally, it discusses one last question: Is gene therapy the vaccination of the future?
RESEARCH
Gene therapy is a biotechnological technique that has recently made significant leaps of progress in the world of scientific research. The theories behind its use have created many new goals and ideas in scientists’ minds, and there is much opportunity for discovery in the field.
There are two types of genetic technology that are currently being researched for application in clinical testing and for the cure of certain genetic diseases in humans: somatic cell gene therapy, and germ-line therapy. Somatic cell gene therapy is a development that could potentially eliminate a hereditary disease’s effects in a patient through the injection of genetic material that would fill in for a nonfunctional gene, alter an abnormal one in the patient’s chromosomes, or exchange the defective gene for a new, fully-functioning one (www.ornl.org). Germ-line therapy would be used similarly in embryos’ germ cells, but would have the additional effect of the faulty gene’s permanent eradication so that it could not be passed on to future descendants.
There are also multiple types of somatic cell gene therapy. In vivo gene therapy, the most common in clinical testin...
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...entists will make many revolutionary discoveries. With this new technology being researched, there’s no telling when the technology will be perfected. Who knows? Someday soon, gene therapy could even be something as commonplace as vaccination.
Bibliography:
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Friedmann, The Development of Human Gene Therapy
http://www.ornl.gov/sci/techresources/Human_Genome/medicine/genetherapy.shtml
http://www.accessexcellence.org/RC/AB/BA/Gene_Therapy_Overview.html
http://asgt.org/news_releases/basics.html
http://www.fda.gov/fdac/features/2000/500_gene.html
http://www.cancer.gov/cancertopics/factsheet/Therapy/gene (G.T. for Cancer)
http://content.nejm.org/cgi/content/short/346/16/1185 (Ex Vivo for X-SCID)
http://asgt.org/news_releases/06022005a.html
http://genome.gov/13014325
Reilly, Abraham Lincoln’s DNA and Other Adventures in Genetics
Turksen, Adult Stem Cells
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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.
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.
In the modern world humans have been able to design and create nearly anything, most to aid us in our daily lives and improve our standard of living. It is only inevitable that eventually humans would take our superior knowledge and skill to manipulate life itself and change our genome to produce a healthier and even more superior human standard of life. In recent years discussion about gene therapy has changed into a promising possibility to treat many of our common human diseases and disorders. Although gene therapy might be the answer to many problems, it has been met with a number of logistical and ethical hardships. With the prospect of being a treatment for inherited genetic disorders, cancers, and viral infections, gene therapy seems like the logical fix-it-all bandage that many people would benefit from.
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.
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.
Rubanyi, G. (2001). The Future of Human Gene Therapy. Molecular Aspects of Medicine. 22,3, pp. 113-142.
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...
In future times, gene therapy will become more popular and companies will be competing with each other for the development of the cheaper, more trust worthy and more efficient gene therapy technologies. Future technology that performs the gene therapy treatment will most likely become gradually cheaper overtime so that the average middle class person will be able to afford gene therapy. Insurance companies may also start allowing their services to cover for the cost of gene therapy (Issues and Impacts, 2014).
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 therapies necessity, stems from the fact that it can cure, life threatening gene based diseases, with minimal after effects. Although there are some ethical and physical issues that can occur through this technology, when comparing it to the other genetic technologies, it’s quite reliable. Although it is in its infancy, the technology has still been present for several years, with the first case of gene therapy being done in 1990. Sure, there have been some complications, but its still a growing technology with a strong future in front of
Gene therapy has been a very controversial issue since its origin in 1990 when A four-year old girl became the first gene therapy patient. She has adenosine deaminase (ADA) deficiency, a genetic disease which leaves her defenseless against infections. Gene therapy involves substituting normal genes for the genes associated with a disease or disorder otherwise altering a person’s genetic makeup. Each gene has a specific code for what it is supposed to do. So, on top of our genome, we have a complex epigenome. These epigenomes consist of all kinds of different markings. The genes themselves are not altered when epigenetic effects occur; however, their effect is altered. Gene therapy can be categorized in to two different types—somatic cell therapy and reproductive cell also referred to as germline therapy. Somatic cells involves targeting
Gene therapy gives patients who born with diseases that are incurable to traditional medicine a permanent cure. If patients received germ-line therapy, which involves replacing disordered genes in sperms or eggs, their offspring would also have correct gene orders. The positive effect would influence the whole family.
...ring deadly diseases and preventing abortions. In order for gene therapy to one day become effective much more research needs to be done to discover the consequences of altering specific genes. Also the technology of gene therapy needs to be cost effective so people who need help are able to get help. In the end gene therapy in humans needs to come a long ways before it will be widely accepted but there is great potential in the technology and it needs to be pursued.