1. Is it possible to treat a genetic disease?
Many genetic diseases result from gene changes which are present in essentially all the cells in the body. Due to this, these diseases often affect several body systems. Most of these diseases cannot be cured. However, there are treatments available to treat or manage the symptoms associated with the diseases.
2. Are the treatments for all the genetic diseases the same?
Treatment and management strategies for genetic conditions are designed to improve signs and symptoms related to the disease. Bone marrow transplant is the only truly available treatment for a range of genetic diseases. Gene therapy, where new genes are implanted in a person's body, is different for every genetic disease.
3. Is there a specially qualified healthcare professional to treat genetic diseases?
Health care professionals who are specially trained and licensed to provide diagnostic and therapeutic services to individuals with genetic diseases are called genetic doctors. They conduct biochemical, cytogenetic, and DNA tests on the patients and provide counseling. Surgeons are involved in doing bone marrow transplant and gene therapy, the only know treatments for genetic diseases.
4. Can oral medicines help in treating genetic diseases?
Genetic therapy is as of now available as part of a clinical trial only. This field is in its research stage. There are certain FDA (U.S.Food and Drug Administration)-approved drugs for treating specific genetic disorders. However, these do not correct the underlying mutation of the disease, but simply alleviate the symptoms.
5. What are the side effects associated with treatments of genetic diseases?
Gene therapy, the treatment option for genetic diseases, may cause side effects such as the working gene may produce too much of the missing enzyme or protein. This may lead to other health problems. The immune system may respond to the working gene copy that is inserted by causing inflammation. The working gene may be slotted into a wrong spot.