Monday, September 30, 2013

Neurodegeneration: An Overview The Uses Of Gene Therapy

By Armand Zeiders


There are many diseases affecting human beings that cause the death of neurons in various parts of the body, such as the brain or central nervous system. These diseases include everything from highly common disorders such as Parkinson's disease and Alzheimer's disease to less common, but still highly debilitating diseases, such as Huntington's disease and Amyotrophic Lateral Sclerosis.

Unfortunately, there are no cures for any these diseases, although there are few different treatments that can help patients better manage discomfort and control the various symptoms. A few treatments have even slowed down the progression of a disease. Scientists, however, are looking to advancements in gene therapy as a possible way to deal with the diseases or even provide cures.

In layman's terms, gene therapy is the introduction of a specific gene into the body which then goes to work to either reverse the effects of a disease or to slow the progression of a disease. A scientist might replace a gene that shows mutation and replace it with a gene that is healthy. The scientist might also introduce a completely new gene into the host, which will fight a disease or deactivate the mutated gene.

Gene therapy does not take the form of a pill, but rather must be delivered into the body's cells using either a non-viral or viral transport. The viruses that are used are altered so that they are safe for the host, and rather than cause a disease, they deliver genetic material to the cell that will help to fight the disease. We often think of viruses as something that brings disease, but in the form of gene therapy, the virus creates changes that impact the body in a positive way.

In addition to using viruses as a transmission method, there is also non-viral gene therapy which includes using naked DNA and magnetofection to provide new genetic material. Both of these methods, as do other non-viral therapy methods, are easier and less costly to produce. Non-viral gene therapy tends to have less of a negative effect on the host in terms of immunogenicity.

Scientists around the world are studying the potential of gene therapy, and there is great hope that in the future that this type of therapy can ward off these diseases. Unlike a medication which might simply alleviate effects and make life a bit easier, gene therapy completely alters the structure of cells, hitting the problem directly at the source. In the years ahead, we should see amazing strides in the field of gene research and gene therapy.




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