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The pathophysiology of diabetes mellitus rests with the hormone insulin. In a unaffected human, complex sugars are broken down into monosaccharides and released into the blood stream. Insulin is released by the pancreas in response to increased blood sugar levels, which then binds to insulin-receptors on tissue cells. This binding of insulin to the cell, increases permeability of the cell membrane for glucose uptake, providing sources of energy to the cells. In the process, blood glucose levels are lowered and completes the negative feedback loop. Type 1 diabetes is caused by an autoimmune response. Antibodies form on the insulin producing β-cells in the pancreas, which signals a cellular and humoral immune response. Cytotoxic T-cells and macrophages target and destroy the β-cells, leading to depletion of insulin producting cells and thus absolute insulin deficiency in the individual. Type 2 diabetes presents from the combination of genetic predisposition, environment and diet l...
Gene therapy began with the Human Genome Project. The Human Genome Project has found gene locations for many diseases. Among the diseases that have been found Huntington’s disease, cystic fibrosis, ADA deficiency, and two genes for breast cancer are just a few examples. After a disease-causing gene is found, correcting it is the next logical step. This can be achieved through various methods. One of the first tested methods was to obtain the faulty gene from the patient, introduce the corrected gene into cells, and finally inject the altered cells back into a blood vessel.
6. Claiborn K, Stoffers D. Toward a cell-based cure for diabetes: advances in production and transplant of beta cells. The Mount Sinai Journal Of Medicine, New York [serial online]. August 2008;75(4):362-371. Available from: MEDLINE with Full Text, Ipswich, MA. Accessed November 19, 2013.
Gene therapy begins with locating the dysfunctional gene. The most complicated part of gene therapy is the transportation of a new gene into another. The new gene data is most commonly inserted into an adeno-associated virus, which is a vector, this virus disallows the potential harm that might be within the body because it does not have the necessary genes to spread an illness in the human body and there are no known causes of this virus infecting humans. The virus, or vector, infects only the targeted cell.
Evolution has made the human immune system fast, strong and diverse, but having an immune system sometimes comes with a heavy price. The immune system is fragile, and if one thing during development of a fetus goes awry then someone can become immunocompromised. One autoimmune disease that is prevalent is diabetes mellitus type 1, with an estimated 30 million people worldwide being affected (Selfvaccine). Diabetes Mellitus, commonly known as diabetes, is categorized into Type 1 and Type 2, and the focus will be on the former. Type 1 diabetes mellitus is a chronic autoimmune disease occurring when insulin beta cells located in the pancreas are destroyed by autoreactive T cells. 1 When the first clinical symptoms of diabetes are apparent in a patient, 80% of the beta cells have been destroyed. (self-antigen) There is no cure for diabetes so someone with this disease would need to monitor their blood glucose levels and manually give themselves insulin to regulate those levels. Since individuals first diagnosed with diabetes still have beta cell insulin production, then there should be an immunological cure that focuses on impeding the antigen presenting of dendritic cells to T cells and the neutralization of T cells to halt the destruction of beta cells and even regenerate beta cells.
Gene Therapy is the insertion of a gene or genes into cells in order to provide a new set of instructions to those cells. Gene insertion can be used to correct an inherited genetic defect which is causing disease, to counter or correct the effects of a genetic mutation, or even to program a cell for an entirely new function or property. Genes are composed of molecules of deoxyribonucleic acid, or DNA (see Nucleic Acids), and are usually located in the nucleus of cells. The instructions that direct the development of an organism are encoded within the genes. Some diseases such as cystic fibrosis are caused by an inherited genetic defect. Other diseases are caused by a miscoding of a gene so that the instructions it contains are disrupted or changed. Genetic miscoding occurs when the DNA in a cell is being duplicated during cell growth and division (somatic mutation) and is common when a cell becomes cancerous. Clinical use of gene therapy began on September 14, 1990, at the National Institute of Health in Bethesda, Maryland, when a four-year-old girl received gene therapy treatment for adenosine deaminase (ADA) deficiency, an ordinarily fatal inherited disease of the immune system. Because of this genetic defect she was susceptible to recurrent life-threatening infections. Gene therapy for this patient involved using a genetically modified virus to carry a normal ADA gene into her immune cells. The inserted ADA gene then programmed the cells to produce the missing ADA enzyme, which led to normal immune function in those cells. This treatment temporarily helped her to develop resistance to infection. Gene therapy may eventually provide effective treatment for many diseases that currently have no cure. These include inherited disorders such as cystic fibrosis, muscular dystrophy, and juvenile diabetes. In addition, gene therapy can also be used to treat many disorders that are not inherited, since gene insertion can also program a cell for an entirely new function. Several new therapies for non-genetic disorders are currently being tested.
Gene therapy is a relatively simple process. First, the mutated gene at fault for the cause of the condition being treated must be identified. Second, the site of the unhealthy cells in the human has to be found. Then, a health...
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.
gene manipulation (Goetz 178). Synthetic insulin is now available for use to treat diabetes. This man made