The Potential of Gene Therapy to Cure Diabetes
Abstract
Gene therapy treats and prevents a disease by introducing a vector of genetic material into certain cells to alter the function or ability of a gene. The promise of gene therapy as a cure for diabetes has been considered ever since this new technology emerged into the clinical and research sphere. Although such methods have yet to undergo human clinical tests, gene therapy holds much potential to bring a radical new way of treating autoimmune diseases such as diabetes. By targeting certain genes that control the insulin and ?-cell production in the pancreas, gene therapy will someday fulfill its potential to cure the disease that is the number one cause of heart disease in the United States. This paper will explore the potential protocols and products that can be used to treat Type I Diabetes.
Diabetes is an autoimmune disease that has affected more than 140 million people in the world. This disease, results from the attack of the killer T-cells of the immune system upon the ?-cells in the pancreas that produces insulin. (Lin et al., 2001). Until recently, this disease could only be treated with daily insulin injections and adherence to a strict, low glucose diet. With more than ninety percent of diabetics at risk for future complications like heart disease, blindness, and renal failure, diabetes has developed into more than just a medical issue. Diabetes is also becoming largely an emotional and economic issue. Victims of this disease have no choice but to adjust their lives around the only object that could change their lives?a daily injection that may cost 50% of the annual income in developing countries and up to 600% in non-developed countries. New technology th...
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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.
Despite being magical of gene therapy, it is high-risk. Few people got benefits from it, and it has a low rate of success. Prior to the human trial, Batshaw and Wilson had done experiment on animals to ensure the safety. Over 20 experiments have been done on mice but only 12 of them survived at last (Sophia, M. and Kolehmainen, J.D., 2000). More seriously, complicating diseases, which can be more dangerous than genetic diseases, might set in during the treatment period. In December 200...
The use for stem cells in type one diabetes involves the insertions of the beta cell. The beta cells are located in the pancreas, which secretes insulin. Insulin is a protein that ge...
Genetic engineering has revolutionized over the years and it is being used to improve food, to discover new medicines, to remove environmental contaminants, to recycle waste, and to provide permanent cures for inherited diseases (Le Vine, 1999). The purpose of genetic engineering in the medical field has been to produce mass-produce insulin, human growth hormones, human albumin, monoclonal antibodies, vaccines, and many other drugs (Applications of Genetic Engineering,
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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.
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Diabetes mellitus affects 18 million people in the U.S. alone (8.7% of the population) and more than 190 million worldwide. The prevalence of diabetes has increased alarmingly in the past three decades and, corresponding to global dietary and lifestyle trends, is projected to nearly double in the next ten years (1). Although diabetes can be treated, serious complications from improperly managed diabetes are common and can lead to death. Recent reports suggest that one of the most promising potential treatments may come from the use of stem cells, undifferentiated cells that can be coaxed into becoming insulin-producing islet-like cells that reduce diabetes symptoms in mice (2).
There are two different types of diabetes, type one and type two diabetes. Diabetes is the disease where the pancreas does not produce insulin which is a necessary hormone needed for the body to function. Insulin is a hormone that the body requires to convert sugars, starches and other foods into energy to be used in everyday life. After a meal has been consumed, the body breaks down the foods consumed into glucose and nutrients that the body needs and they are then absorbed into the bloodstream through the gastrointestinal tract. After the glucose has been absorbed into the bloodstream, the body then sends a signal to the pancreas for it to begin producing insulin for the body. In people with diabetes, their bodies may stop producing insulin or their bodies may no longer respond to the hormone. Without insulin, glucose cannot gain the key of access into the cells to produce energy for the body to function. If the glucose does not make its way into the cells, it stays in the bloodstream which will result in elevated glucose levels which can lead to other health problems. The overall disease of diabetes is the similar but there are some differences between type one and type two. In type one diabetes, the body’s own immune system attacks the cells of the pancreas and destroys them so that they cannot produce insulin anymore causing them to become diabetic. Scientists believe that the cause of type one diabetes is more often passed through the genes of the parents, however, in recent times being overweight is starting to be more of the cause of developi...
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
Safdar, M. (2010) Gene Therapy: Advantages and Disadvantages [Online] Available at: http://www.biotecharticles.com/Genetics-Article/Gene-Therapy-Advantages-and-Disadvantages-271.html [Accessed July 17 2011]
Genetic engineering was originally designated for medical purposes to correct the mistakes in DNA that condition certain diseases, and therefore prevent them. The first approved trial of gene therapy was conducted in 1990 by Anderson and colleagues on a disease called adenosine deaminase deficiency, in which the loss of a