For genetic metabolic disorders, a potential approach or treatment is gene therapy. In this technique, the absent or faulty gene is replaced with an effective and working gene.
As a result, your body produces the correct enzyme or protein and accordingly eliminates the root cause of the disease.
Gene therapy is an experimental technique that can be effectively used to treat rare diseases.
The future possibilities of this technique enable doctors to treat any disorder by inserting working genes into patients’ body cells instead of drugs and surgery.
Is gene therapy effective?
Studies are in progress to determine how effective it is. There are a few factors that have kept gene therapy from becoming an effective treatment:
Short-lived nature: Before gene therapy can become a permanent cure for diseases, therapeutic DNA will be introduced into target cells. These therapeutic target cells must remain functional, long-lived and stable.
However, the rapidly dividing nature of cells and the difficulties of integrating therapeutic DNA into a genome prevent gene therapy from accomplishing long-term benefits.
Viral vectors: Viruses are used as potential carriers of genes into the patient’s body. These viruses are responsible for a variety of illnesses, such as inflammatory and immune response as well as toxicity on the targeting tissues.
In addition, there is a fear that these specific viral vectors, after entering the patient’s body, may recover their ability to cause disease.
Abnormal immune response: Whenever a foreign object is introduced into our body tissues, it is the nature of our immune system to attack the invader.
This increases the risk of picking up various disorders which are related to an undesired immune response. So, the risk of stimulating immune response certainly reduces the effectiveness of gene therapy.
Multi-gene disorder: If you have any disorders that arise from mutations in a single gene, gene therapy should be very effective. However, disorders that are related to mutations of multiple genes are quite difficult to treat with gene therapy.
Try to find out more about gene therapy from your personal physician and understand how it relates to various health conditions.