LINE OF BIOLOGICALLY ACTIVE GENE THERAPY SUBSTANCES BASED ON COL1A1 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 relates to a means for correcting the pathological conditions of cells of organs and tissues and/or human organs and tissues based on the COL1A1 gene, where the cells of organs and tissues are selected from cells of fibroblasts, keratocytes and epithelial cells of the eye, chondroblasts; organs and tissues are selected from the skin, cartilage tissue or human muscle tissue, which is a set of biologically active gene therapy substances, each of which is a gene therapy substance selected from a group of gene therapy substances, each being a genetic construct based on a vector plasmid, comprising the cDNA of the COL1A1 gene, with the alpha-1 protein coding sequence of the type I collagen chain, with deletions of 5' and 3' non-translated regions, in combination with a transport molecule or without it. Group of inventions also concerns the use of said means for correcting the pathological conditions of the cells of organs and tissues and/or human organs and tissues associated with a quantitative reduction in the alpha-1 protein of the collagen type I chain.;EFFECT: group of inventions provides a high and stable level of the target protein in the cells.;5 cl, 18 ex, 20 dwg