Gene therapy is the treatment of hereditary, non-hereditary, multifactorial diseases, which is carried out by introducing other genes into the patient’s cells. The goal of therapy is to eliminate gene defects or give cells new functions. It is much easier to introduce a healthy, fully functioning gene into a cell than to correct defects in an existing one.
Gene therapy is limited to studies in somatic tissues. This is due to the fact that any intervention in germ and germ cells can give a completely unpredictable result.
The currently used technique is effective in the treatment of both monogenic and multifactorial diseases (malignant tumors, some types of severe cardiovascular, viral diseases).
About 80% of all gene therapy projects relate to HIV infection and cancer. Currently, research is underway on hereditary diseases such as hemophilia B, Gaucher disease, cystic fibrosis, hypercholesterolemia.
Treatment of genetic diseases involves:
· Isolation and reproduction of individual types of patient cells;
· The introduction of foreign genes;
· Selection of cells in which the foreign gene has "taken root";
· Implantation of the patient (for example, through blood transfusion).
Gene therapy is based on the introduction of cloned DNA into the patient’s tissue. In this case, injection and aerosol vaccines are considered the most effective methods.
Gene therapy works in two ways:
1. Treatment of monogenic diseases. These include disorders in the functioning of the brain, which are associated with any damage to the cells that are produced by neurotransmitters.
2. Treatment of hereditary diseases. The main approaches used in this area:
· Genetic improvement of immune cells;
· Increased tumor immunoreactivity;
· Oncogen expression block;
· Protection of healthy cells from chemotherapy;
· The introduction of tumor suppressor genes;
· Production of antitumor substances by healthy cells;
· Production of antitumor vaccines;
· Local reproduction of normal tissues using antioxidants.
The use of gene therapy has many advantages and in some cases is the only chance for a normal life for sick people. However, this field of science is not fully understood. There is an international ban on germ and pre-implantation germ cell tests. This is to prevent unwanted gene constructs and mutations.
Some conditions have been developed and universally accepted under which clinical trials are allowed:
The gene transferred to the target cells must be active for a long time.
In a foreign environment, the gene must remain effective.
Gene transfer should not cause negative reactions in the body.
There are a number of issues that today remain relevant for many scientists around the world:
Will scientists working in the field of gene therapy be able to develop a complete gene correction that will not pose a threat to offspring?
Will the need and usefulness of the gene therapy procedure for an individual couple exceed the risk of this intervention for the future of humanity?
Are such procedures justified, given the overpopulation of the planet in the future?
How will such procedures on a person relate to questions of homeostasis of the biosphere and society?
In conclusion, it can be noted that genetic therapy at the present stage offers mankind ways of treating the most serious diseases, which more recently were considered incurable and fatal. However, at the same time, the development of this science presents scientists with new problems that need to be addressed today.