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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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.
There have been four somewhat recent successful gene therapy treatments. The four deal with correcting hemophilia, bone marrow transplants, skin cancer, and vessel growth. In the success with the bone marrow transplants, French researchers collected bone marrow cells from patients, used gene therapy to correct the bone marrow, and then returned the bone marrow to the patient. This was 80% successful as reports 16 months after the transplants showed. Squamous cell carcinoma, skin cancer of the head and neck, was treated using gene therapy as well. The fourth trial was where DNA was used to carry a substance that stimulates blood vessel growth to damaged heart tissue and in this trial there was much success noted.
... 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.
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.
To conclude, although gene therapy can cure a wide variety of diseases which cannot be cured by traditional medicine, and patients can get permanent cure without rejections, it can be high-risk and immoral. The negative effects of gene therapy lead to the shrink of the number of volunteers, and many trials have been forced to cease. The Gene therapy's potential to revolutionize medicine in the future is exciting, and hopes are high for its role in curing and preventing childhood diseases.
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.
Gene therapy is the therapeutic delivery of nucleic acid into a patient’s cells as a drug to treat diseases such as Severe Combined Immunodeficiency (SCID) and Adenosine Deaminase Deficiency (ADA) and possibly cancer as well. It was first researched in 1985
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.
Human genetic engineering can provide humanity with the capability to construct “designer babies” as well as cure multiple hereditary diseases. This can be accomplished by changing a human’s genotype to produce a desired phenotype. The outcome could cure both birth defects and hereditary diseases such as cancer and AIDS. Human genetic engineering can also allow mankind to permanently remove a mutated gene through embryo screening, as well as allow parents to choose the desired traits for their children. Negative outcomes of this technology may include the transmission of harmful diseases and the production of genetic mutations.
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.
In the end, gene therapy in humans needs to come a long way before it will be widely accepted, but there is great potential in the technology and it needs to be pursued. Bibliography Anderson, W. F. (1992). The Species of the World. Human Gene Therapy -. Science, 256 (5058), 808-813.
The procedures that will be the future of modern medicine currently fall into the realms of taboo and fictional. These procedures encompass every aspect of medical science, from exploration of the human body, curing diseases, to improving a person’s quality of life. Many of these procedures are not very well known, while a few have been in the spotlight. These procedures include cloning, nano-robotics, retro-viruses, and genetic manipulation via gene-specific medications. For any serious breakthroughs in modern medical science, we must embrace these new forms of treatment instead of shying away from them. Second, I’ll attempt to explain how these methods and procedures could benefit mankind.