Gene Therapy

Gene Therapy is a treatment aimed at ridding a person of any genetic disease by introducing normal genes into his body using genetic engineering methods, so that they can overcome the effects of defective genes. The most radical approach in this case is the introduction of normal genes at a very early stage of human embryonic development, so that the new gene can connect with the germ cells (egg and sperm) and the person would inherit the characteristics that this gene carries. However, this approach is not absolutely safe and ethically justified, since the consequences of such gene introduction will subsequently affect all the descendants of the person who underwent such an operation; therefore, this method is practically not used in medicine. In somatic cell gene therapy, a healthy gene is introduced into somatic cells (such as bone marrow stem cells), from which other cells develop. All new cells that appear on the basis of these modified cells will be completely normal, and if they are present in the body in sufficient quantities, then the human disease will be eliminated (defective genes, however, will remain in the germ cells, but their influence will be suppressed). Currently, gene therapy is most often performed to treat diseases caused by the presence of a defect in one recessive gene, so that the existing defect can be eliminated by introducing a normal allele (treatment of diseases associated with the presence of defects in dominant genes (for example, Huntington's disease) requires modification or replacement of a defective allele when its effect is expressed in the presence of a normal allele). Examples of such recessive diseases include adenosine deaminase deficiency (ADA deficiency) and fibrocystic degeneration. Enough model experiments related to gene therapy have already been carried out: lymphocyte stem cells are taken from the patient’s body using monoclonal antibodies, which are then grown together with retroviruses genetically programmed so that they contain the normal ADA gene (see Carrier). Thus, this gene connects with stem cells, which, upon returning to the patient’s bone marrow, begin to produce normal lymphocytes there. Clinical experiments involving gene therapy for fibrocystic degeneration involve using liposomes to introduce a normal gene into the lungs of sick people using an inhaler. Research is also being conducted into gene therapy for certain types of cancer (eg, melanoma and breast cancer). IN In this case, genes are introduced into tissue affected by cancer cells that accelerate the formation of substances that destroy tumor cells.

Gene therapy is a treatment aimed at ridding a person of any genetic disease by introducing normal genes into his body using genetic engineering methods. The goal is to overcome the effects of defective genes at the expense of normal ones.

The most radical approach is to introduce normal genes at a very early stage of embryonic development, so that the new gene combines with the germ cells and the person inherits the traits it carries. However, this method is unsafe and unethical, because... will affect all descendants. Therefore, it is practically not used in medicine.

In somatic cell gene therapy, a healthy gene is introduced into somatic cells, such as bone marrow stem cells. All new cells that develop from the modified ones will be normal. If there are enough of them, the disease can be cured, although the defective genes will remain in the germ cells.

Most often, gene therapy is used for diseases due to a defect in one recessive gene, in order to eliminate the defect by introducing a normal allele. To treat diseases caused by defects in dominant genes, replacement of the defective allele itself is required.

Examples of recessive diseases are adenosine deaminase deficiency and cystic fibrosis. Experiments are being conducted to transfer a normal gene into stem cells using genetically modified retroviruses.

In cystic fibrosis, the possibility of introducing the gene into the lungs using liposomes is being explored. Gene therapy is also being studied for some types of cancer by introducing genes that accelerate the destruction of tumor cells.

Gene Therapy: Treatment of the Future

In recent decades, gene therapy has become one of the most innovative and promising areas in medicine. This treatment method aims to eliminate genetic diseases by introducing normal genes into the patient's body. Gene therapy opens up new opportunities to combat hereditary and acquired diseases that were previously considered incurable.

The basic principle of gene therapy is to replace or modify defective genes responsible for a specific disease. There are several approaches to performing gene therapy, but the most common are germ cell therapy and somatic cell therapy.

In germ cell therapy, normal genes are introduced early in embryonic development. This allows the new gene to be integrated into the genome of embryonic cells and transmitted to all descendants of that organism. However, this method is ethically complex and potentially dangerous, since its consequences can affect not only direct descendants, but also subsequent generations.

Somatic cell therapy, on the other hand, aims to introduce normal genes into specific tissues or organs of the patient. In this case, genes are introduced into somatic cells, which then develop into healthy cells in the body. This approach avoids passing on genes to offspring and limits the effect of therapy only on a specific organ or tissue.

Currently, gene therapy is widely used to treat diseases caused by defects in recessive genes. One successful example of gene therapy is the treatment of adenosine deaminase deficiency (ADA deficiency) and fibrocystic degeneration. In ADA deficiency, the normal gene is inserted into the patient's stem cells and then returned to the patient's body. In this way, the stem cells begin to produce normal lymphocytes, which allows the deficiency to be corrected and the immune system to be restored.

Liposomes are used in the treatment of fibrocystic degeneration to deliver normal