Alzheimer's Disease: A Genetic Analysis

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Alzheimer's is a chronic disease caused by a progressive loss of structure or function of neurons. The most common symptoms of Alzheimer's include memory loss and confusion. As of now, no cures exist but many strategies may help improve symptoms, thus creating a happier life. Alzheimer's is widespread with nearly 44 million people having it (“2015”). Alzheimer's disease is one of the most widespread disorders in the world. Current treatments and therapy can treat symptoms experienced, especially in the early stages, but none can delay or hinder the progression of Alzheimer's. With the need of new treatments to deter Alzheimer's advancement, gene therapy is seen as a great alternative to the current treatments (Nobre). All Alzheimer’s patients …show more content…
This can be seen when neurologists at UC San Diego began experimenting with a new treatment for Alzheimer's. With the use of skin samples from the backs of eight Alzheimer’s patients, the neurologists secluded an enduring type of connective tissue cells. Using a virus, the neurologists genetically modified the cells to attempt to produce and secrete a likely therapeutic protein known as NGF (Nerve growth factor), to try to turn the cells into small biological drug pumps. After this, the neurologists inserted these mutated skin cells straight into each of the eight patient’s basal fore brain. Cells that live in the basal fore brain are usually the first to die from Alzheimer's disorder and the neurologists wanted to test whether NGF could help rescue the withering of the fore brain. After ten years, they checked back on how the patients were doing. The gene therapy seemed to work. The autopsy results show that the mutant skill cells were able to reliably pump out NGF in the patients’ brains, which consequently enhanced the cells’ size and their ability to ramificate new neural fibers. The treatment rescued the susceptible cells from dying off; even the cells that showed …show more content…
Many deficiencies have been treated using gene therapy. The majority of the time, the patients blood stem cells are extracted and retroviruses are used to replace the defective genes with working copies. Then, the patients receive their stem cells back. Because the cells are treated outside the patient's body, the virus infects the genes and transfers them to only the target cells that are in need of them. Severe Combined Immune Deficiency (SCID) was one of the first genetic disorders to be treated successfully using gene therapy, showing neuro-scientists that gene therapy could work and has many future applications. Although the first trial ended when the virus triggered leukemia (a type of blood cancer) in some patients, researchers have begun trials with safer viral vectors that are proven to less likely cause cancer. Adenosine Deaminase Deficiency (ADA Deficiency) is another example of an inherited immune disorder that has been treated successfully with the use of gene therapy. For the most of the patients in these trials, the immune systems function improved to the point that they did not need injections of the ADA enzyme. Also unlike the last experiment, none of the patients developed leukemia (“Gene”).With these experiments consisting of gene therapy, the impact of immune deficiencies were
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