Genetic inherited diseases are very rare among the human population. However, they are not to be taken for granted because genetic inherited diseases are either fatal or it greatly affects the life of an individual even at birth. Currently, there are no treatments for these diseases. However, there are several studies that are being made to come up with different treatments.
With the development of genetic engineering, scientists have discovered that altering a gene structure would create great results. This method was done in genetically engineered plants wherein the plants are able to become resistant to a certain disease through altering their genetic information adding a resistance to the disease. This resulted to greater crop yields and lesser cost for pesticides.
Scientists have also applied the process of genetic engineering in discovering treatment for genetic inherited diseases. Such specific process is called gene therapy. Gene therapy involves the use of a DNA to treat disease. An introduction of a therapeutic DNA to the cells would enable the cells to produce a new set of functional proteins that would replace a mutated gene which is common in people with genetic inherited diseases. This approach is more direct because it targets directly the causes of genetic inherited diseases since such diseases involve the DNA structure of a human being. This is also why there are no surgeries or medications which can really treat such diseases.
Pharmacological Gene Therapy
Another application of genetic engineering is applied in pharmacological gene therapy. It is a combination of both pharmacological therapy and gene therapy. Its mode of action in treating genetic inherited diseases is to introduce a DNA or RNA fragment to either prevent the defective gene to produce more protein or to increase the production or concentration of more normal protein in the body to lessen the effect of the diseases or eventually curing the disease.
Antisense therapy is also an application of genetic engineering. It is also a form of treatment for genetic inherited diseases or even some other diseases as well such as cancer and HIV or AIDS. In Antisense therapy, the main goal is to make the defective gene inactive so that it would no longer be functional. It involves introduction of a synthesized nucleic acid strand to the defective gene’s mRNA or messenger RNA causing it to be deactivated.
All of these treatments for genetic inherited diseases are still under clinical trials, meaning that these treatments are not yet offered to the public. Unlike plants, humans have a more complex system which makes it hard for scientist to fully understand how they would be able to apply such genetic engineering processes to be able to come up with a cure. Aside from that, there are still some ethical and social issues involved which would be a problem in case these treatments are going to be successful. Hopefully though, in the near future, with the constant improvement of technology and also with the change in people’s culture, these treatments will become available to the public.
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