gene therapy (rus. генная терапия) — treatment of inherited and acquired diseases by introducing genetic elements into the patient's somatic cells to recover or suppress certain gene functions and ensure some specific features of the cells.


Broadly defined, gene therapy includes:

1) insertion of a gene into an individual’s cells to restore an insufficient function of their own gene which caused a disease;

2) suppression of an individual’s gene expression which causes a disease using molecular genetic technology;

3) insertion of genes that enhance immunogenicity and cause death of diseased cells, or stimulate the immune response.

Currently, genetic manipulation of human germ cells is not considered part of gene therapy.

In addition to the genes encoding proteins, gene therapy uses genes encoding RNA, complementary to the mRNA of target gene (antisense RNA), RNA precursors of short interfering RNA (siRNA), or short synthetic nucleotide sequences (antisense oligonucleotides and siRNA). These genetic elements selectively inhibit gene expression by suppressing corresponding mRNA translation or promoting its enzymatic cleavage. In medicine approaches using such genetic structures are called antisense therapy and RNA interference (RNAi).

Depending on specific tasks, genetic elements can be integrated into host cell genome for long-lasting expression, or function separately from chromosomal DNA for temporal expression. The insertion of genes into the cells can be carried out outside of an organism. For this purpose target cells are obtained from the patient and injected back into the patient after the introduction of the required gene. Alternatively, the genes are introduced directly into the host organism. Special targeted delivery systems (see the articles on vectors, gene delivery) are used for the effective introduction of genetic elements into the cells.


  • Shirinsky Vladimir P.


  1. Levonen A. L., Vahakangas E., Koponen J. K., Yla-Hertualla S. Antioxidant gene therapy for cardiovascular disease. Current status and future perspectives // Circulation. 2008. V. 117. P. 2142–2150.

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