Genes (composed of deoxyribonucleic acid, also known as DNA) function as the biological determinants of heredity that exhibit traits, such as eye color and they are located on chromosomes inside cells.
Gene therapy
Gene therapy is an experimental treatment that involves introducing genetic material (DNA or RNA) into a person’s cells to fight or prevent disease. It facilitates our body by providing the genes in needs, or correct copies of defective genes that may potentially cause genetic disorders. So far, researchers are studying gene therapy in clinical trials to suit different types of cancer and other various diseases, such as hemophilia B, Parkinson’s disease, and HIV.
Several approaches are being attempted in order to cure diseases via gene therapy. First of all, mutated genes that cause diseases are replaced with healthy copies of the genes. Also, mutated genes that are functioning improperly are inactivated. Finally, new genes are introduced into the body to help fighting against diseases.
Generally, a gene cannot be directly inserted into a person’s cell. So, a vector, which is used to deliver a gene to a person’s cell, is used. Virus is the most commonly used vector in gene therapy. Although the viruses are genetically altered to be safe and carry normal human DNA, some risks still exist with gene therapy.
Gene therapy was not available outside a clinical trial until recently. Since 1990, hundreds of clinical trials have been conducted and most have failed as it was difficult to deliver the genes and keep them activated for a long time. Also, the incident of a teenager’s death during a clinical trial at the University of Pennsylvania in 1999 brought the safety concerns on the surface. However, researchers have been slowly overcoming the obstacles and in the last few years there have been reports of successes in attempts to treat cancer, hemophilia B, certain immune diseases and a condition that causes blindness.
After all, gene therapy was first officially approved in Europe. The European Medicines Agency has recommended the application of a gene therapy to treat rare genetic diseases. This was actually the very first regulatory approval of a gene therapy drug in the Western world. Also, a gene therapy to treat cancer won an approval in China in 2003.
As illustrated, a gene therapy has been a controversial topic and it was successful in establishing realistic procedure in the world. Still it is not such a common method that we encounter in the medical field. As it was proven to be beneficial and effective in treating number of symptoms, our deeper attention and acknowledgement regarding this content would be an advantageous step in discovering a new tool to cure diseases.
Here is a video more detailed descriptions about the gene therapy.
KiWon Min
Online reference
http://www.genetherapynet.com/
http://www.sciencedaily.com/news/health_medicine/gene_therapy/
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