A RANGE OF BIOLOGICALLY ACTIVE GENE THERAPEUTIC SUBSTANCES BASED ON THE COL1A1 GENE FOR CORRECTION OF PATHOLOGICAL STATES OF HUMAN BODIES AND TISSUES AND HUMAN BODIES AND HUMAN TISSUES

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