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 TGFB1 gene, associated with a quantitative decrease in the protein of the transforming growth factor beta-1, where the cells of organs and tissues are selected from fibroblasts, keratocytes and epithelial cells of the eye, chondroblasts; organs and tissues are selected from 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 gen therapy substances, each being a genetic construct based on a vector plasmid, including the cDNA of the TGFB1 gene, with the beta-1 transforming growth factor protein coding sequence, with deletions of 5' and 3' non-translated regions, and also containing regulatory elements that ensure transcription of this sequence in eukaryotic cells of human organs and tissues, 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 TGFB1 gene, associated with a quantitative decrease in the protein of the transforming growth factor beta-1.
EFFECT: group of inventions provides a high and stable level of the target protein in the cells.
5 cl, 20 dwg, 18 ex
Authors
Dates
2018-04-25—Published
2016-01-20—Filed