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.
Viruses tend to be a bit riskier and more difficult to produce, so scientists also focus their efforts on non-viral gene therapy. For example, you can use a process such as magnetofection to add nucleic acid into a specific cell. Another option would be injecting a patient with naked DNA. Either option has its advantages over viral methods in that non-viral gene therapy is easy to produce and has low immunogenicity.
Because gene therapy basically attacks a biological problem right at its source, this type of science holds a great deal of hope for the future. Many laboratories and universities around the world currently are working on creating viable medical treatments using gene therapy, in the hopes of finding a way to treat or cure these debilitating neurodegenerating diseases.
Armand Zeiders enjoys blogging about biomedical research. For more information about protein sequencing service, please check out the Primm Biotech site today.