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.

While cures are not available yet for any of these diseases, there are treatments available that can help control symptoms to a degree and in some cases, slightly slow the progression of these diseases. In the future, scientists hope that gene therapy can be used to stop or slow the horrific effects of these terrible medical conditions which affect millions of people throughout the world.

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.

There are several non-viral methods of gene therapy, as well. Injecting the host with naked DNA is one option, as is magnetofection. Magnetofection is process that uses magnetic fields to introduce nucleic acid into a target cell. The advantage of non-viral methods is that these can be easily produced on a large scale, and they typical have low host immunogenicity.

While gene therapy is not yet a mainstream treatment for these neurological diseases, the advantage of gene therapy is that it addresses the problems within the cell directly, thus altering genes and making them function properly. The hope is that we reach a point in scientific advancement when we can eliminate these diseases entirely or at least alleviate the effects so that people can live longer, fuller lives.




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