LINE OF BIOLOGICALLY ACTIVE GENE-THERAPY SUBSTANCES BASED ON TGFBR2 GENE FOR CORRECTION OF PATHOLOGICAL STATES OF CELLS OF ORGANS AND TISSUES AND HUMAN ORGANS AND TISSUES, METHOD OF OBTAINING AND USING

摘要

FIELD: medicine.;SUBSTANCE: group of inventions relates to medicine and concerns a means for correcting the pathological conditions of the cells of organs and tissues and/or human organs and tissues based on the TGFBR2 gene, associated with a quantitative decrease in the TGFBR2 protein, where the organs and tissues cells are selected from fibroblasts, keratocytes and epithelial cells of the eye, chondroblasts; organs and tissues are selected from the skin, cartilage tissue or muscle tissue, which is a set of biologically active gene therapy substances, each of which is a gene-therapy substance, selected from the group of gene therapy substances, each being a genetic construct based on a vector plasmid, including the cDNA of the TGFBR2 gene, with the TGFBR2 protein coding sequence, with deletions of 5' and 3' non-translated regions, in combination with a transport molecule or without it. Group of inventions also relates to a method for producing said agent; a method of using said agent for correcting the pathological conditions of cells of organs and tissues and/or human organs and tissues based on the gene TGFBR2, associated with a quantitative decrease in the protein TGFBR2.;EFFECT: group of inventions provides a high and stable level of the target protein in the cells.;5 cl, 18 ex, 20 dwg