Gene therapy

Gene therapy is the treatment of hereditary,non-hereditary, multifactorial diseases, which is carried out by introducing into the patient's cells other genes. 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 the cell, than to correct defects in the existing one.

Gene therapy is limited to studies in somatic tissues. This is due to the fact that any intervention in the sex and germ cells can give a completely unpredictable result.

The current technique is effectivein the treatment of both monogenic and multifactorial diseases (malignant tumors, certain types of severe cardiovascular, viral diseases).

About 80% of all gene therapy projects concernHIV infection and cancer. At present, research is under way on such hereditary diseases as hemophilia B, Gaucher's disease, cystic fibrosis, hypercholesterolemia.

Treatment of genetic diseases implies:

· The isolation and multiplication of individual cell types of the patient;

Introduction of foreign genes;

· Selection of cells in which the foreign gene "took root";

· Implantation of them to the patient (for example, through blood transfusion).

Gene therapy is based on the introduction of cloned DNA into the patient's tissue. The most effective methods are injecting and aerosol vaccines.

Gene therapy works in two ways:

1. Treatment of monogenic diseases. These include disorders in the brain that are associated with any damage to cells that neurotransmitters produce.

2. Treatment of hereditary diseases. The main approaches used in this area are:

· Genetic improvement of immune cells;

· Increase of tumor immunoreactivity;

· Block of expression of oncogenes;

Protection of healthy cells from chemotherapy;

· Insertion of tumor suppressor genes;

· Production of antitumor substances by healthy cells;

· Production of antitumor vaccines;

· Local reproduction of normal tissues with the help of antioxidants.

The use of gene therapy has many advantages andin some cases is the only chance for a normal life for sick people. Nevertheless, this area of ​​science is not fully understood. There is an international ban on tests on genital and pre-implantation germ cells. This is done to prevent unwanted gene constructs and mutations.

A number of conditions have been developed and generally accepted, under which clinical trials are allowed:

1. The gene transferred to the target cells should be active for a long time.

2. In a foreign environment, the gene must remain effective.

3. The transfer of the gene should not cause negative reactions in the body.

There are a number of issues that still remain relevant for many scientists around the world:

• Will scientists working in the field of gene therapy develop a full genocorrection that will not pose a threat to the offspring?

• Will the need and utility of the gene therapy procedure for a particular married couple exceed the risk of this intervention for the future of mankind?

• Are such procedures justified, given the overpopulation of the planet in the future?

• How will such procedures relate to the person with questions of homeostasis of the biosphere and society?

In conclusion, it can be noted that the genetictherapy at the present stage offers mankind the ways of treating the most serious diseases that were recently considered incurable and fatal. However, at the same time, the development of this science poses new problems for scientists, which must be solved today.

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