Genetic disorders are the health conditions, which are formed as a result of the mutation of the genetic code. It is estimated that there are 10,000 different kinds of diseases which are caused by a single gene mutation. The World Health Organization estimates that 10 in every 1000 individuals or 70 million to 80 million individuals worldwide have one of these single-gene diseases.
Since the majority of genetic diseases have no cure, people with such defects can observe how their symptoms evolve with time and their quality of life decreases. But there is hope of treating and even curing the genetic disorders due to the recent advances in medical science in the area of gene therapy. In this paper we are going to discuss about gene therapy and its promise to the individuals afflicted with genetic diseases.
Genetic Disorder Classes
Although genetic diseases vary thousands of different ways, they can generally be classified in to one of the following three types:
- Single-gene disorders: Such genetic disorders are caused by the mutation of one gene. They are also known as monogenic and examples of such diseases are cystic fibrosis, sickle cell anemia, CMT4 and Tay-Sachs disease.
- Chromosomal disorders: These disorders influence the structure itself in which the genetic material is found and this is the chromosome. Dysmorphism in the number or structure of chromosomes in a cell leads to chromosomal disorders. Some examples of such disorders are Down syndrome, Turner syndrome and Fragile X syndrome.
- Complex disorders: Complex disorders are caused by alteration in several genes as well as environmental or lifestyle choices. Alzheimer, multiple sclerosis, diabetes, and asthma are complexity examples of genetic disorders.
What is Gene Therapy?
Gene therapy is a form of medical treatment which tries to treat the root cause of a medical condition by attacking the faulty genes which cause the medical condition. Mutations in genetic material are normally defective due to hereditary factors or environmental factors like UV or chemical exposures, or radiations. Gene therapies are currently able to:
- Substitute defective genes or disease-causing genes with healthy genes.
- Transfer new genes to the body to assist in curing a disease
- Switch off genes that cause disease or malfunctioning.
Currently, the U.S. Food and Drug Administration (FDA) has approved gene therapy in some genetic diseases, such as hemophilia, spinal muscular atrophy, and sickle cell disease. Nevertheless, to a greater number of individuals afflicted with a genetic disorder, gene therapy remains experimental and is at present, only accessible through a clinical trial.
The Future
Gene therapy may be regarded as a far-fetched dream, but a significant number of those who have worked on the future of genetic therapies believe that gene therapies will be the standard of care to rare genetic diseases prior to the year 2036 and that they will be curable beyond this time frame.
Gene therapy is capable of preventing curing and even eradicating genetic diseases. By going to the very core of the disease, and treating it directly, such a groundbreaking treatment will be able to better the lives of tens of millions of individuals throughout the world and finally change the very way that we perceive healthcare.
